62. Paroxysmal nocturnal hemoglobinuria Clinical trials / Disease details
Clinical trials : 292 / Drugs : 151 - (DrugBank : 49) / Drug target genes : 22 - Drug target pathways : 108
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05731050 (ClinicalTrials.gov) | June 2025 | 1/2/2023 | Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase II, Open Label, Multi Dose Study of NM8074 in Soliris-Treated Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | PNH - Paroxysmal Nocturnal Hemoglobinuria | Drug: NM8074 | NovelMed Therapeutics | NULL | Not yet recruiting | 18 Years | 65 Years | All | 6 | Phase 2 | NULL |
| 2 | NCT05646524 (ClinicalTrials.gov) | November 2023 | 30/11/2022 | Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy | A Phase II, Open-Label Study of NM8074 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: NM8074 | NovelMed Therapeutics | NULL | Not yet recruiting | 18 Years | N/A | All | 12 | Phase 2 | NULL |
| 3 | NCT05646563 (ClinicalTrials.gov) | September 2023 | 30/11/2022 | Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris | A Phase II, Open-Label Study of NM8074 in Soliris®-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: NM8074;Drug: Soliris | NovelMed Therapeutics | NULL | Not yet recruiting | 18 Years | N/A | All | 12 | Phase 2 | NULL |
| 4 | NCT05630001 (ClinicalTrials.gov) | May 30, 2023 | 18/11/2022 | This is a Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. | A Multicenter, Single Arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Have Hb=10 g/dL in Response to Anti-C5 Antibody and Switch to Iptacopan | Paroxysmal Nocturnal Hemoglobinuria | Drug: Iptacopan | Novartis Pharmaceuticals | NULL | Not yet recruiting | 18 Years | N/A | All | 50 | Phase 3 | NULL |
| 5 | NCT05744921 (ClinicalTrials.gov) | March 9, 2023 | 6/2/2023 | A Study to Examine the Long-term Safety, Tolerability, and Effectiveness of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Not yet recruiting | 18 Years | N/A | All | 300 | Phase 3 | NULL |
| 6 | EUCTR2021-004253-22-GR (EUCTR) | 23/01/2023 | 29/11/2022 | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to C5i in Patients with PNH. | A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated with Danicopan in an Alexion-sponsored Clinical Study. | Patients with Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 95 | Phase 3 | United States;Czechia;Greece;Thailand;Spain;Israel;United Kingdom;Italy;France;Canada;Brazil;Malaysia;Poland;Japan;Korea, Republic of | ||
| 7 | NCT05741346 (ClinicalTrials.gov) | January 18, 2023 | 14/2/2023 | Long-term Safety of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria | An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Previously Received BCX9930 in a BioCryst-sponsored Study | Paroxysmal Nocturnal Hemoglobinuria | Drug: BCX9930 | BioCryst Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 30 | Phase 2 | Hungary;Malaysia |
| 8 | EUCTR2021-004931-10-GR (EUCTR) | 04/01/2023 | 22/11/2022 | A study to examine the long-term safety, tolerability, and effectiveness of Pozelimab and Cemdisiran combination therapy in adult patients with Paroxysmal Nocturnal Hemoglobinuria | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria - ACCESS-EXTENSION | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: Cemdisiran Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 288 | Phase 3 | United States;Philippines;Taiwan;Greece;Thailand;Spain;Colombia;Italy;India;France;Jordan;Malaysia;Peru;South Africa;China;Korea, Democratic People's Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Singapore;Romania;Germany;Japan | ||
| 9 | EUCTR2020-002761-33-FR (EUCTR) | 06/12/2022 | 21/03/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 140 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Netherlands;Germany;Japan;Korea, Republic of | ||
| 10 | NCT05476887 (ClinicalTrials.gov) | November 25, 2022 | 25/7/2022 | To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 | An Open-label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 in Complement Inhibitor-naïve Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: KP104 | Kira Pharmacenticals (US), LLC. | NULL | Recruiting | 18 Years | N/A | All | 35 | Phase 2 | China |
| 11 | NCT05389449 (ClinicalTrials.gov) | October 28, 2022 | 20/5/2022 | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH | A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study | Paroxysmal Nocturnal Hemoglobinuria | Drug: Danicopan | Alexion Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 100 | Phase 3 | United States;Italy;United Kingdom |
| 12 | NCT05131204 (ClinicalTrials.gov) | October 6, 2022 | 12/11/2021 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | A Randomized, Open-Label, Eculizumab and Ravulizumab Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Currently Treated With Eculizumab or Ravulizumab | Paroxysmal Nocturnal Hemoglobinuria | Drug: Cemdisiran;Drug: Eculizumab;Drug: Pozelimab;Drug: Ravulizumab | Regeneron Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 2 | Phase 3 | United States |
| 13 | EUCTR2020-004438-39-NL (EUCTR) | 04/10/2022 | 05/01/2022 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BioCryst Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Slovakia;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;Netherlands;China;Japan;Korea, Republic of | ||
| 14 | NCT05133531 (ClinicalTrials.gov) | August 1, 2022 | 12/11/2021 | Ravulizumab-Controlled Non-Inferiority Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy | A Randomized, Open-Label, Ravulizumab-Controlled,Non-Inferiority Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy | Paroxysmal Nocturnal Hemoglobinuria | Drug: Ravulizumab;Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 148 | Phase 3 | United States;Canada;Greece;Italy;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Romania;Singapore;Spain;Taiwan;Turkey |
| 15 | EUCTR2020-002761-33-DE (EUCTR) | 20/06/2022 | 03/03/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 140 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Brazil;Poland;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of | ||
| 16 | EUCTR2020-004486-40-GR (EUCTR) | 17/06/2022 | 08/04/2022 | Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy | A RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 124 | Phase 3 | Portugal;United States;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Canada;Jordan;Poland;Brazil;Malaysia;Singapore;Romania;Peru;South Africa;Netherlands;China;Japan;Korea, Republic of | ||
| 17 | EUCTR2020-002761-33-GR (EUCTR) | 17/06/2022 | 12/04/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED, NON-INFERIORITY STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB - ACCESS-2 | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 134 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Thailand;Turkey;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Brazil;Poland;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of | ||
| 18 | NCT05642585 (ClinicalTrials.gov) | May 27, 2022 | 30/11/2022 | A Study of Single-dose MY008211A in Healthy Adults | A Phase I, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Doses Clinical Study to Evaluate the Safety, Tolerability and Pharmacokinetic/Pharmacodynamics Characteristics of MY008211A Tablets in Healthy Adult Volunteers | Paroxysmal Nocturnal Hemoglobinuria | Drug: MY008211A tablets;Drug: Placebo | Wuhan Createrna Science and Technology Co., Ltd | NULL | Active, not recruiting | 18 Years | 45 Years | All | 46 | Phase 1 | China |
| 19 | EUCTR2020-004486-40-ES (EUCTR) | 09/05/2022 | 11/03/2022 | Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy | A RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 124 | Phase 3 | Portugal;United States;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Canada;Jordan;Poland;Brazil;Malaysia;Singapore;Romania;Peru;South Africa;Netherlands;China;Japan;Korea, Republic of | ||
| 20 | EUCTR2020-002761-33-ES (EUCTR) | 09/05/2022 | 11/03/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Netherlands;Germany;Japan;Korea, Republic of | ||
| 21 | JPRN-jRCT2031220046 | 28/04/2022 | 28/04/2022 | Pegcetacoplan Long-term Safety and Efficacy Extension Study | AN OPEN-LABEL, NONRANDOMIZED, MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF PEGCETACOPLAN IN THE TREATMENT OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - APL2-307 | Paroxysmal Nocturnal Hemoglobinuria (PNH) | A subcutaneous dose of pegcetacoplan (APL-2) 1080 mg twice a week, 3 times a week or every 3 days | Machaidze Zurab | NULL | Not Recruiting | >= 18age old | Not applicable | Both | 10 | Phase 3 | the United States;South Korea;Australia;Malaysia;Hong Kong;Philippines;Singapore;Thailand;Peru;Colombia;Mexico;Canada;Bulgaria;Serbia;Belgium;France;Germany;Italy;the Netherlands;Russia;Spain;the United Kingdom;Japan |
| 22 | NCT04965597 (ClinicalTrials.gov) | April 19, 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
| 23 | EUCTR2020-002761-33-PL (EUCTR) | 14/04/2022 | 22/03/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 140 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of | ||
| 24 | EUCTR2020-004403-14-CZ (EUCTR) | 31/03/2022 | 24/09/2021 | BCX9930 compared to placebo in the treatment of PNH | A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria - REDEEM 2 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 57 | Phase 2 | United States;Serbia;Czechia;Spain;Turkey;Lithuania;Azerbaijan;Italy;Czech Republic;Canada;Argentina;Brazil;Poland;Romania;South Africa;Albania;China;Sweden | ||
| 25 | EUCTR2019-003829-18-GR (EUCTR) | 08/03/2022 | 14/01/2022 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Czechia;Taiwan;Greece;Thailand;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Canada;Argentina;Poland;Brazil;Malaysia;Netherlands;Germany;Japan;Korea, Republic of | ||
| 26 | JPRN-jRCT2031210645 | 04/03/2022 | 04/03/2022 | A Phase 3, Randomized, Multicenter, Open-label, Active-comparator Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Randomized, Multicenter, Open-label, Active-comparator Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | Paroxysmal Nocturnal Hemoglobinuria (PNH) | A subcutaneous dose of pegcetacoplan (APL-2) 1080 mg twice weekly (or change to a dose of 1080 mg every 3 days as necessary). | Machaidze Zurab | NULL | Complete | >= 18age old | Not applicable | Both | 8 | Phase 3 | the U.S.;Korea;Australia;Canada;Belgium;France;Germany;Russia;Spain;the United Kingdom;Japan |
| 27 | EUCTR2019-004931-21-RO (EUCTR) | 14/02/2022 | 19/05/2022 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | Portugal;United States;Philippines;Hong Kong;Taiwan;Estonia;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;Austria;United Kingdom;Hungary;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Romania;Germany;Japan;Sweden | ||
| 28 | NCT05083364 (ClinicalTrials.gov) | February 2, 2022 | 8/10/2021 | Study of ARO-C3 in Adult Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria and Complement-Mediated Renal Disease | A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Pharmacodynamics of ARO-C3 in Adult Healthy Volunteers and in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Adult Patients With Complement-Mediated Renal Disease | Paroxysmal Nocturnal Hemoglobinuria;C3 Glomerulopathy;IgA Nephropathy | Drug: ARO-C3;Drug: Placebo | Arrowhead Pharmaceuticals | NULL | Recruiting | 18 Years | 70 Years | All | 84 | Phase 1 | Australia;Korea, Republic of;New Zealand;Thailand |
| 29 | EUCTR2020-004385-19-NL (EUCTR) | 11/01/2022 | 17/11/2021 | Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria | An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 165 | Phase 3 | United States;Taiwan;Spain;Lithuania;Italy;France;Brazil;Malaysia;Singapore;Netherlands;China;Japan;Korea, Republic of | ||
| 30 | EUCTR2020-004385-19-CZ (EUCTR) | 16/12/2021 | 27/07/2021 | Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria | An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 165 | Phase 3 | United States;Taiwan;Spain;Lithuania;Italy;France;Czech Republic;Brazil;Malaysia;Singapore;Netherlands;China;Japan;Korea, Republic of | ||
| 31 | JPRN-jRCT2021210059 | 09/12/2021 | 17/12/2021 | Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria | An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. | Paroxysmal Nocturnal Hemoglobinuria | Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral | Suzuki Kazuyuki | NULL | Recruiting | >= 18age old | Not applicable | Both | 19 | Phase 3 | Italy;Republic of Korea;Taiwan;Japan |
| 32 | NCT05116774 (ClinicalTrials.gov) | December 6, 2021 | 2/11/2021 | BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: BCX9930;Drug: Eculizumab;Drug: Ravulizumab | BioCryst Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 81 | Phase 2 | France;Hungary;Italy;Spain;United Kingdom;Taiwan;United States |
| 33 | EUCTR2020-004438-39-SK (EUCTR) | 29/11/2021 | 29/07/2021 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BioCryst Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Slovakia;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of | ||
| 34 | EUCTR2020-004403-14-ES (EUCTR) | 11/11/2021 | 14/07/2021 | BCX9930 compared to placebo in the treatment of PNH | A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria - REDEEM 2 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 57 | Phase 2 | Serbia;United States;Spain;Turkey;Lithuania;Azerbaijan;European Union;Canada;Argentina;Brazil;Albania;South Africa;China | ||
| 35 | EUCTR2020-004438-39-IT (EUCTR) | 10/11/2021 | 27/01/2022 | BCX9930 treatment in PNH patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: ULTOMIRIS - 300 MG - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO (VETRO) 3 ML (100 MG / ML) - 1 FLACONCINO Product Name: ravulizumab Product Code: [ravulizumab] INN or Proposed INN: Ravulizumab Other descriptive name: Ultomiris Trade Name: SOLIRIS - 300 MG CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO 1 FLACONCINO (VETRO) 30 ML (10 MG/ML) Product Name: Eculizumab Product Code: [Eculizumab] INN or Proposed INN: ECULIZUMAB Other descriptive name: Soliris Product Name: BCX9930 Product Code: [BCX9930] INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BIOCRYST PHARMACEUTICALS INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of | ||
| 36 | EUCTR2019-003830-17-ES (EUCTR) | 02/11/2021 | 14/07/2021 | Study of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy | A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy - Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: ALXN2050 Product Code: ALXN2050 INN or Proposed INN: N/A Other descriptive name: ACH-0145228 | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 26 | Phase 2 | France;Canada;Spain;Turkey;Germany;United Kingdom;Italy;New Zealand;Korea, Republic of | ||
| 37 | EUCTR2020-004438-39-ES (EUCTR) | 02/11/2021 | 23/07/2021 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Spain;Turkey;Colombia;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of | ||
| 38 | NCT05116787 (ClinicalTrials.gov) | October 26, 2021 | 2/11/2021 | BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy | A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of PNH | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: BCX9930 monotherapy;Drug: Placebo | BioCryst Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 57 | Phase 2 | Malaysia;South Africa;Spain;Taiwan;United States |
| 39 | EUCTR2020-004403-14-IT (EUCTR) | 20/10/2021 | 22/10/2021 | BCX9930 compared to placebo in the treatment of PNH | A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria - REDEEM 2 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: [BCX9930] INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BIOCRYST PHARMACEUTICALS INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 57 | Phase 2 | Serbia;United States;Spain;Lithuania;Turkey;Italy;Azerbaijan;European Union;Canada;Argentina;Brazil;Albania;South Africa;China | ||
| 40 | EUCTR2020-004403-14-LT (EUCTR) | 14/10/2021 | 18/08/2021 | BCX9930 compared to placebo in the treatment of PNH | A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria - REDEEM 2 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 57 | Phase 2 | United States;Serbia;European Union;Canada;Argentina;Brazil;Turkey;Lithuania;South Africa;Albania;China;Azerbaijan | ||
| 41 | EUCTR2019-003830-17-IT (EUCTR) | 13/10/2021 | 23/06/2020 | Study of the Oral Factor D (fD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with background use of an Approved C5 Inhibitor | A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with an Approved C5 Inhibitor as Background Therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: ALXN2050 Product Code: ACH-0145228 INN or Proposed INN: N/A Other descriptive name: ACH-0145228 Product Name: ALXN2050 Product Code: ACH-0145228 INN or Proposed INN: N/A Other descriptive name: ACH-0145228 | Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 26 | Phase 2 | United States;Spain;United Kingdom;New Zealand;Italy;Korea, Republic of | ||
| 42 | EUCTR2020-000597-26-IE (EUCTR) | 23/09/2021 | 13/01/2021 | A Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and efficacy of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 190 | Phase 3 | Portugal;United States;Philippines;Taiwan;Hong Kong;Estonia;Greece;Thailand;Spain;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Belgium;Brazil;Poland;Singapore;Romania;Germany;Japan;Sweden | ||
| 43 | EUCTR2020-004438-39-HU (EUCTR) | 01/09/2021 | 02/07/2021 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB | BioCryst Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Slovakia;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;Netherlands;China;Japan;Korea, Republic of | ||
| 44 | EUCTR2020-004385-19-FR (EUCTR) | 31/08/2021 | 15/07/2021 | Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria | An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 165 | Phase 3 | United States;France;Taiwan;Spain;Malaysia;Brazil;Singapore;Lithuania;Italy;Japan;China;Korea, Republic of | ||
| 45 | EUCTR2020-004438-39-FR (EUCTR) | 26/08/2021 | 16/06/2021 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Turkey;Colombia;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of | ||
| 46 | EUCTR2020-003172-41-CZ (EUCTR) | 12/08/2021 | 10/06/2021 | Study of Efficacy and Safety of Twice Daily Oral iptacopan in Adult PNH Patients who are naïve to complement inhibitor therapy | A multicenter, single-arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who are naive to complement inhibitor therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | France;United States;Czechia;Czech Republic;Malaysia;Singapore;Germany;United Kingdom;Italy;Japan;China;Korea, Republic of | ||
| 47 | EUCTR2020-004385-19-ES (EUCTR) | 30/07/2021 | 23/06/2021 | Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria | An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Farmacéutica, S.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 165 | Phase 3 | United States;Taiwan;Malaysia;Brazil;Spain;Singapore;Japan;China;Korea, Republic of | ||
| 48 | NCT04811716 (ClinicalTrials.gov) | July 28, 2021 | 19/3/2021 | Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | Paroxysmal Nocturnal Hemoglobinuria | Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 24 | Phase 2 | Hong Kong;Hungary;Korea, Republic of;Malaysia;Taiwan;United Kingdom |
| 49 | NCT04747613 (ClinicalTrials.gov) | July 27, 2021 | 5/2/2021 | Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria | An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan | Paroxysmal Nocturnal Hemoglobinuria | Drug: Iptacopan | Novartis Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 167 | Phase 3 | United States;Brazil;China;Czechia;France;Germany;Italy;Japan;Korea, Republic of;Lithuania;Malaysia;Netherlands;Singapore;Spain;Taiwan;United Kingdom |
| 50 | EUCTR2020-003172-41-IT (EUCTR) | 20/07/2021 | 17/08/2021 | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients who are naïve to complement inhibitor therapy. | A multicenter, single-arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who are naive to complement inhibitor therapy. - - | Paroxysmal Nocturnal Hemoglobinuria (PNH). MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: HIBERIX - POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE PER USO INTRAMUSCOLARE 1 FLACONCINO DI POLVERE + 1 SIRINGA PRERIEMPITA DI SOLVENTE DA 0.5 ML Product Name: - Product Code: [-] INN or Proposed INN: VACCINO HAEMOPHILUS INFLUENZAE B CONIUGATO CON TOSSOIDETETANICO Trade Name: MENVEO – Polvere e soluzione per soluzione iniettabile Product Name: - Product Code: [-] INN or Proposed INN: VACCINO POLISACCARIDICO ANTI-MENINGOCOCCICO (A.C.Y E W-135) Trade Name: PNEUMOVAX – Soluzione iniettabile in siringa preriempita da 0,5 ml Product Name: - Product Code: [-] INN or Proposed INN: VACCINO PNEUMOCOCCICO Trade Name: BEXSERO – Sospensione iniettabile – Siringa preriempita Product Name: - Product Code: [-] INN or Proposed INN: VACCINO CONTRO IL MEMINGOCOCCO DI GRUPPO B Trade Name: ACT-HIB Polvere e solvente per soluzione iniettabile in siringa preriempita 0,5 ml Product Name: - Product Code: [-] INN or Proposed INN: HAEMOPHILUS INFLUENZAE | NOVARTIS PHARMA AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | France;United States;Czechia;Malaysia;Singapore;Germany;United Kingdom;Japan;China;Italy;Korea, Republic of | ||
| 51 | NCT04820530 (ClinicalTrials.gov) | July 19, 2021 | 25/3/2021 | Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy | A Multicenter, Single-arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Iptacopan (LNP023) | Novartis Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 40 | Phase 3 | China;France;Germany;Italy;Korea, Republic of;Malaysia;Singapore;United Kingdom;Czechia;Japan |
| 52 | EUCTR2019-004931-21-LT (EUCTR) | 15/07/2021 | 19/05/2021 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | Portugal;United States;Philippines;Hong Kong;Taiwan;Estonia;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;Austria;United Kingdom;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Romania;Germany;Japan;Sweden | ||
| 53 | EUCTR2020-004385-19-LT (EUCTR) | 15/07/2021 | 20/04/2021 | Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria | An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 165 | Phase 3 | United States;Taiwan;Spain;Lithuania;Italy;France;Brazil;Malaysia;Singapore;Netherlands;China;Japan;Korea, Republic of | ||
| 54 | NCT04888507 (ClinicalTrials.gov) | July 8, 2021 | 23/4/2021 | Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy | A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy | Paroxysmal Nocturnal Hemoglobinuria | Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 6 | Phase 2 | United Kingdom |
| 55 | EUCTR2020-000597-26-EE (EUCTR) | 30/06/2021 | 24/04/2021 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | Portugal;United States;Philippines;Taiwan;Hong Kong;Estonia;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Poland;Brazil;Belgium;Singapore;Romania;Germany;Japan;Sweden | ||
| 56 | EUCTR2019-004931-21-EE (EUCTR) | 30/06/2021 | 24/04/2021 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | Portugal;United States;Philippines;Hong Kong;Estonia;Taiwan;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;Austria;United Kingdom;Hungary;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Romania;Germany;Japan;Sweden | ||
| 57 | JPRN-jRCT2021210014 | 28/06/2021 | 28/06/2021 | Pivotal study of danicopan as add-on therapy to a C5 inhibitor (eculizumab or ravulizumab) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have Clinically Evident EVH | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria;D006457 | Additional Danicopan (ALXN2040) given to patients diagnosed with paroxysmal nocturnal hemoglobinuria and receiving complement C5 inhibitors | Kamata Takeo | NULL | Recruiting | >= 18age old | Not applicable | Both | 84 | Phase 3 | America;Japan |
| 58 | EUCTR2020-004385-19-IT (EUCTR) | 15/06/2021 | 08/06/2021 | Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria | An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. - - | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: PNEUMOVAX – Soluzione iniettabile in siringa preriempita da 0,5 ml Product Name: - Product Code: [-] INN or Proposed INN: VACCINO PNEUMOCOCCICO Product Name: iptacopan Product Code: [LNP023] INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: [LNP023] INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: ACTHIB - POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE 1 FLACONCINO POLVERE+1 SIRINGA PRERIEMPITA SOLVENTE 0.5 ML Product Name: - Product Code: [-] INN or Proposed INN: HAEMOPHILUS INFLUENZAE Trade Name: MENVEO - POLV.E SOLUZ.PER SOLUZ. INIETT.-USO INTRAMUSCOLARE-COMP. CONIUG.LIOFILIZZ.MENA:FLAC.(VETRO) COMP. GONIUG.LIQUI.MENCWY:FLAC.(VETRO)-1 FLAC+1FLAC Product Name: - Product Code: [-] INN or Proposed INN: VACCINO POLISACCARIDICO ANTI-MENINGOCOCCICO (A.C.Y E W-135) Trade Name: BEXSERO - SOSPENSIONE INIETTABILE - USO INTRAMUSCOLARE - SIRINGA P | NOVARTIS PHARMA AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 165 | Phase 3 | United States;Taiwan;Spain;Malaysia;Brazil;Singapore;Korea, Democratic People's Republic of;Lithuania;Japan;China;Italy | ||
| 59 | EUCTR2020-003172-41-FR (EUCTR) | 03/06/2021 | 15/04/2021 | Study of Efficacy and Safety of Twice Daily Oral iptacopan in Adult PNH Patients who are naïve to complement inhibitor therapy | A multicenter, single-arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who are naive to complement inhibitor therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | United States;France;Czechia;Malaysia;Singapore;Germany;United Kingdom;Italy;Japan;China;Korea, Republic of | ||
| 60 | EUCTR2019-003829-18-NL (EUCTR) | 21/05/2021 | 07/10/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Finland;Thailand;Spain;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 61 | EUCTR2019-004931-21-PT (EUCTR) | 19/05/2021 | 28/10/2020 | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Soliris 300 mg concentrate for solution for infusion INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Portugal;Philippines;Estonia;Taiwan;Hong Kong;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Peru;Australia;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;Austria;United Kingdom;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Germany;Japan;Sweden | ||
| 62 | EUCTR2020-000597-26-PT (EUCTR) | 18/05/2021 | 23/12/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689 INN or Proposed INN: CROVALIMAB Trade Name: SOLIRIS® (eculizumab) injection, for intravenous use Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | Portugal;United States;Estonia;Taiwan;Hong Kong;Greece;Thailand;Spain;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden | ||
| 63 | EUCTR2020-005005-17-HU (EUCTR) | 07/05/2021 | 05/03/2021 | Pozelimab and Cemdisiran Combination Treatment in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: Cemdisiran Other descriptive name: CEMDISIRAN | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | Taiwan;Hong Kong;Hungary;Malaysia;United Kingdom;Korea, Republic of | ||
| 64 | EUCTR2020-000597-26-CZ (EUCTR) | 28/04/2021 | 22/12/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | Portugal;United States;Philippines;Taiwan;Hong Kong;Estonia;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Poland;Brazil;Belgium;Singapore;Romania;Germany;Japan;Sweden | ||
| 65 | EUCTR2019-003829-18-PL (EUCTR) | 08/04/2021 | 04/11/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Spain;Thailand;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 66 | NCT04729062 (ClinicalTrials.gov) | April 5, 2021 | 25/1/2021 | Pegcetacoplan Early Access Program for PNH | Early Access Program (EAP) For The Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) With Pegcetacoplan | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Pegcetacoplan | Apellis Pharmaceuticals, Inc. | NULL | No longer available | 18 Years | N/A | All | United States | ||
| 67 | EUCTR2019-004665-40-CZ (EUCTR) | 31/03/2021 | 03/11/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 91 | Phase 3 | United States;Czechia;Taiwan;Spain;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of | ||
| 68 | NCT04320602 (ClinicalTrials.gov) | March 26, 2021 | 23/3/2020 | Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab | Phase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab | Paroxysmal Nocturnal Hemoglobinuria | Biological: Eculizumab;Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 18 | Phase 4 | United Kingdom |
| 69 | JPRN-jRCT2051200132 | 26/03/2021 | 09/02/2021 | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment - APPLY-PNH | Paroxysmal nocturnal hemoglobinuria PNH | Arm A : LNP023 monotherapy Arm B : Anti-C5 antibody treatment(Eculizumab or Ravulizumab) | Suzuki Kazuyuki | NULL | Not Recruiting | >= 18age old | Not applicable | Both | 91 | Phase 3 | Netherlands;Japan |
| 70 | NCT04654468 (ClinicalTrials.gov) | March 17, 2021 | 30/11/2020 | A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition | A Phase III, Multicenter, Single Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition | Paroxysmal Nocturnal Hemoglobinuria | Drug: Crovalimab | Hoffmann-La Roche | NULL | Active, not recruiting | 12 Years | N/A | All | 51 | Phase 3 | China |
| 71 | EUCTR2019-003829-18-DE (EUCTR) | 03/02/2021 | 23/01/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Thailand;Turkey;Israel;United Kingdom;Italy;France;Canada;Argentina;Brazil;Malaysia;Poland;Netherlands;Germany;Japan;Korea, Republic of | ||
| 72 | EUCTR2019-004665-40-DE (EUCTR) | 29/01/2021 | 22/09/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris 300mg/30ml Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris 300mg/30ml Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 91 | Phase 3 | United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of | ||
| 73 | EUCTR2019-004931-21-FR (EUCTR) | 26/01/2021 | 18/09/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Portugal;Taiwan;Hong Kong;Greece;Thailand;Spain;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden | ||
| 74 | NCT04558918 (ClinicalTrials.gov) | January 25, 2021 | 27/8/2020 | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment | A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody. | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: LNP023;Drug: Eculizumab;Drug: Ravulizumab | Novartis Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 97 | Phase 3 | United States;Brazil;Czechia;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Spain;Taiwan;United Kingdom;Hungary |
| 75 | EUCTR2019-004931-21-BE (EUCTR) | 22/01/2021 | 23/11/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | Portugal;United States;Philippines;Hong Kong;Taiwan;Estonia;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;Austria;United Kingdom;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Romania;Germany;Japan;Sweden | ||
| 76 | EUCTR2020-000597-26-BE (EUCTR) | 11/01/2021 | 15/10/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | United States;Portugal;Philippines;Taiwan;Estonia;Hong Kong;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Brazil;Belgium;Poland;Singapore;Romania;Germany;Japan;Sweden | ||
| 77 | ChiCTR2000039675 | 2021-01-01 | 2020-11-05 | Phase II clinical trial of the superiority of all-trans retinoic acid in the treament of paroxysmal nocturnal hemoglobinuria | Phase II clinical trial of the superiority of all-trans retinoic acid in the treament of paroxysmal nocturnal hemoglobinuria | paroxysmal nocturnal hemoglobinuria | experimental group:Tretinoin 0.33mg/kg.d; methylprednisolone 0.8mg/kg.d;Control test:methylprednisolone 0.8mg/kg.d; | Department of Hematology, Second Medical Center, Chinese PLA General Hospital | NULL | Recruiting | 14 | 80 | Both | experimental group:10;Control test:10; | Phase 2 | Chinese |
| 78 | NCT05490017 (ClinicalTrials.gov) | December 30, 2020 | 4/8/2022 | To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 | SYNERGY-1: A Phase 1 First-in-human, Randomized, Double Blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 in Healthy Subjects | Paroxysmal Nocturnal Hemoglobinuria | Drug: KP104;Drug: Placebo | Kira Pharmacenticals (US), LLC. | NULL | Completed | 18 Years | 55 Years | All | 80 | Phase 1 | Australia |
| 79 | EUCTR2019-004665-40-IT (EUCTR) | 23/12/2020 | 15/06/2021 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody - Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia d | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Iptacopan Product Code: [LNP023] INN or Proposed INN: Iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: Iptacopan Product Code: [LNP023] INN or Proposed INN: Iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris Product Name: Eculizumab Product Code: [-] INN or Proposed INN: ECULIZUMAB Trade Name: ACT-HIB Polvere e solvente per soluzione iniettabile in siringa preriempita 0,5 ml Product Name: - Product Code: [-] INN or Proposed INN: HAEMOPHILUS INFLUENZAE Trade Name: MENVEO – Polvere e soluzione per soluzione iniettabile Product Name: - Product Code: [-] INN or Proposed INN: VACCINO POLISACCARIDICO ANTI-MENINGOCOCCICO (A.C.Y E W-135) Trade Name: PNEUMOVAX – Soluzione iniettabile in siringa preriempita da 0,5 ml Product Name: - Product Code: [-] INN or Proposed INN: VACCINO PNEUMOCOCCICO | NOVARTIS PHARMA AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 91 | Phase 3 | United States;Czechia;Taiwan;Spain;Korea, Democratic People's Republic of;Italy;United Kingdom;France;Hungary;Canada;Brazil;Germany;Netherlands;Japan | ||
| 80 | NCT04702568 (ClinicalTrials.gov) | December 18, 2020 | 7/1/2021 | A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria;PNH | Drug: BCX9930 | BioCryst Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 200 | Phase 2 | Austria;South Africa;United Kingdom |
| 81 | NCT04469465 (ClinicalTrials.gov) | December 16, 2020 | 9/7/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: Danicopan;Drug: Placebo;Drug: C5 Inhibitor | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 86 | Phase 3 | United States;Brazil;Canada;Czechia;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Malaysia;Netherlands;Poland;Spain;Taiwan;Thailand;United Kingdom |
| 82 | EUCTR2019-004931-21-IT (EUCTR) | 10/12/2020 | 24/05/2021 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS - N/A | Paroxysmal Nocturnal Hemoglobinuria (PNH);Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: [RO7112689] INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab Product Code: [NA] INN or Proposed INN: Eculizumab | F. HOFFMANN - LA ROCHE LTD. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Portugal;Estonia;Hong Kong;Taiwan;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Peru;Australia;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;United Kingdom;Hungary;Mexico;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden | ||
| 83 | EUCTR2020-000597-26-IT (EUCTR) | 10/12/2020 | 22/06/2021 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS - na | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: crovalimab Product Code: [RO7112689] INN or Proposed INN: crovalimab Trade Name: Eculizumab Product Name: eculizumab Product Code: [na] INN or Proposed INN: eculizumab Other descriptive name: NA | F. HOFFMANN - LA ROCHE LTD. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | United States;Portugal;Estonia;Taiwan;Hong Kong;Greece;Thailand;Spain;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;United Kingdom;Czech Republic;Hungary;Mexico;Brazil;Belgium;Poland;Germany;Japan;Sweden | ||
| 84 | EUCTR2020-000597-26-GR (EUCTR) | 04/12/2020 | 16/10/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689 INN or Proposed INN: CROVALIMAB Trade Name: Eculizumab Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | United States;Portugal;Hong Kong;Taiwan;Greece;Spain;Thailand;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden | ||
| 85 | EUCTR2019-004931-21-GR (EUCTR) | 04/12/2020 | 16/10/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Portugal;Taiwan;Hong Kong;Greece;Thailand;Spain;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Romania;Germany;Japan;Sweden | ||
| 86 | EUCTR2019-004931-21-DE (EUCTR) | 01/12/2020 | 14/07/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITH COMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | Portugal;United States;Philippines;Estonia;Hong Kong;Taiwan;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Peru;Australia;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;United Kingdom;Hungary;Mexico;Argentina;Poland;Brazil;Belgium;Germany;Japan;Sweden | ||
| 87 | EUCTR2020-000597-26-DE (EUCTR) | 01/12/2020 | 14/07/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | United States;Portugal;Philippines;Estonia;Taiwan;Hong Kong;Greece;Thailand;Spain;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;United Kingdom;Czech Republic;Hungary;Mexico;Brazil;Belgium;Poland;Germany;Japan;Sweden | ||
| 88 | EUCTR2020-000501-93-AT (EUCTR) | 27/11/2020 | 08/09/2020 | A long term study of BCX9930 in patients with PNH | A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 2 | European Union;Denmark;Austria;South Africa;Italy;United Kingdom | ||
| 89 | EUCTR2019-004931-21-PL (EUCTR) | 24/11/2020 | 18/09/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | Portugal;United States;Philippines;Estonia;Hong Kong;Taiwan;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Peru;Australia;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;United Kingdom;Hungary;Mexico;Argentina;Brazil;Poland;Belgium;Germany;Japan;Sweden | ||
| 90 | EUCTR2020-000597-26-PL (EUCTR) | 23/11/2020 | 16/09/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 250 | Phase 3 | United States;Portugal;Philippines;Estonia;Taiwan;Hong Kong;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;United Kingdom;Czech Republic;Hungary;Mexico;Brazil;Poland;Belgium;Germany;Japan;Sweden | ||
| 91 | EUCTR2019-004665-40-NL (EUCTR) | 20/11/2020 | 09/11/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris 300mg/30ml Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris 300mg/30ml Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 91 | Phase 3 | United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Brazil;Netherlands;Germany;Japan;Korea, Republic of | ||
| 92 | EUCTR2019-004665-40-HU (EUCTR) | 17/11/2020 | 23/09/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 91 | Phase 3 | United States;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of | ||
| 93 | EUCTR2019-004931-21-HU (EUCTR) | 05/11/2020 | 08/09/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 3 | Hungary;United States;Portugal;Taiwan;Hong Kong;Greece;Thailand;Spain;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden | ||
| 94 | EUCTR2019-003829-18-FR (EUCTR) | 04/11/2020 | 22/01/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Finland;Spain;Thailand;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 95 | EUCTR2019-003829-18-IT (EUCTR) | 03/11/2020 | 24/05/2021 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) - . | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: [ACH-0144471] Product Name: Danicopan (ALXN2040) Product Code: [ACH-0144471] | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Finland;Spain;Thailand;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 96 | EUCTR2019-004665-40-FR (EUCTR) | 03/11/2020 | 18/09/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 91 | Phase 3 | United States;Taiwan;Spain;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of | ||
| 97 | EUCTR2020-000501-93-IT (EUCTR) | 02/11/2020 | 25/01/2021 | A long term study of BCX9930 in patients with HPN | A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) - A long term study of BCX9930 in patients with HPN | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: BCX9930 Product Code: [BCX9930] INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BIOCRYST PHARMACEUTICALS INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 2 | European Union;Denmark;Austria;South Africa;United Kingdom;Italy | ||
| 98 | EUCTR2020-000501-93-DK (EUCTR) | 27/10/2020 | 11/09/2020 | A long term study of BCX9930 in patients with PNH | A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 2 | European Union;Austria;Denmark;South Africa;Italy;United Kingdom | ||
| 99 | EUCTR2019-004931-21-SE (EUCTR) | 26/10/2020 | 03/06/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Portugal;Philippines;Taiwan;Estonia;Hong Kong;Greece;Thailand;Spain;Ireland;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;Austria;United Kingdom;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Romania;Germany;Japan;Sweden | ||
| 100 | JPRN-JapicCTI-205425 | 08/10/2020 | 21/08/2020 | A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. ( COMMODORE 2 ) | A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. | Paroxysmal Nocturnal Hemoglobinuria | Intervention name : Crovalimab INN of the intervention : crovalimab Dosage And administration of the intervention : Participants will receive an initial intravenous (IV) loading dose on Week 1 Day 1, followed by 4 weekly crovalimab subcutaneous (SC) doses on Week 1 Day 2, then on Weeks 2, 3 and 4. Maintenance dosing will begin at Week 5 and will continue Q4W (every 4 weeks) thereafter, for a total of at least 24 weeks of study treatment. Crovalimab will be administered at a dose of 1000 mg IV (for participants with body weight between 40 and 100kg) or 1500 mg IV (for participants with body weight >=100kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, it will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight between 40 and 100kg) or 1020 mg SC (for participants with body weight >=100kg). Control intervention name : Eculizumab INN of the control intervention : eculizumab Dosage And administration of the control intervention : Participants will receive initial IV weekly doses for 4 weeks which will be followed by Q2W (every 2 weeks) IV administrations starting on Week 5. Eculizumab will be administered at a dose of 600 mg for the first 4 weeks, followed by maintenance doses of 900 mg starting on Week 5. | Chugai Pharmaceutical Co., Ltd. | NULL | recruiting | BOTH | 200 | Phase 3 | Japan, Asia except Japan, North America, South America, Europe, Oceania, Africa | ||
| 101 | NCT04434092 (ClinicalTrials.gov) | October 8, 2020 | 3/6/2020 | A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. | A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. | Paroxysmal Nocturnal Hemoglobinuria | Drug: Crovalimab;Drug: Eculizumab | Hoffmann-La Roche | Chugai Pharmaceutical | Active, not recruiting | N/A | N/A | All | 214 | Phase 3 | Argentina;Australia;Brazil;China;France;Germany;Greece;Hong Kong;Japan;Korea, Republic of;Lithuania;Malaysia;Mexico;Netherlands;Philippines;Poland;Portugal;Romania;Singapore;Spain;Sweden;Taiwan;Thailand;Turkey;Ukraine;United Kingdom;Belgium;Canada;Chile;Colombia;Czechia;Estonia;Hungary;Ireland;Israel;Italy;Peru;Saudi Arabia;South Africa;United States |
| 102 | NCT04432584 (ClinicalTrials.gov) | September 30, 2020 | 3/6/2020 | A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors | A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Safety, Pharmacokinetics, Pharmacodynamic And Efficacy of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors | Paroxysmal Nocturnal Hemoglobinuria | Drug: Crovalimab;Drug: Eculizumab | Hoffmann-La Roche | Chugai Pharmaceutical | Recruiting | N/A | N/A | All | 190 | Phase 3 | United States;Australia;Belgium;Brazil;Canada;Czechia;Estonia;France;Germany;Greece;Hong Kong;Hungary;Ireland;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Saudi Arabia;Singapore;Spain;Sweden;Taiwan;Turkey;United Kingdom;Colombia;Israel;Mexico;Slovakia |
| 103 | JPRN-JapicCTI-205430 | 29/9/2020 | 24/08/2020 | A Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors. (COMMODORE 1) | A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors. | Paroxysmal Nocturnal Hemoglobinuria | Intervention name : Crovalimab INN of the intervention : crovalimab Dosage And administration of the intervention : Participants will receive a loading series of crovalimab comprised of an intravenous (IV) dose on Day 1, followed by weekly crovalimab subcutaneous (SC) doses for 4 weeks on Week 1 Day 2, then on Weeks 2, 3, and 4. Maintenance SC dosing will begin at Week 5 and will continue Q4W (every 4 weeks) thereafter for a total of at least 24 weeks of study treatment. Crovalimab will be administered at a dose of 1000 mg IV (for participants with body weight between 40 and 100kg) or 1500 mg IV (for participants with body weight >=100kg) on Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, it will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight between 40 and 100kg) or 1020 mg SC (for participants with body weight >=100kg). Control intervention name : Eculizumab INN of the control intervention : eculizumab Dosage And administration of the control intervention : Participants will receive an approved maintenance dose of eculizumab starting on Day 1 and Q2W (every 2 weeks) thereafter for a total of 24 weeks of study treatment. After 24 weeks of study eculizumab treatment, participants will have the option to switch to crovalimab or to discontinue from the study after completion of 10 weeks of safety follow-up. Eculizumab will be administered at a dose of 900 mg every 2 weeks. | Chugai Pharmaceutical Co., Ltd. | NULL | recruiting | BOTH | 250 | Phase 3 | Japan, Asia except Japan, North America, South America, Europe, Oceania | ||
| 104 | EUCTR2020-000501-93-GB (EUCTR) | 29/09/2020 | 09/07/2020 | A long term study of BCX9930 in patients with PNH | A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 2 | European Union;South Africa;United Kingdom | ||
| 105 | EUCTR2019-003829-18-GB (EUCTR) | 18/09/2020 | 05/02/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Finland;Thailand;Spain;Austria;Switzerland;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 106 | EUCTR2019-003830-17-GB (EUCTR) | 16/09/2020 | 16/06/2020 | Study of the Oral Factor D (fD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with background use of an Approved C5 Inhibitor | A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with an Approved C5 Inhibitor as Background Therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: ALXN2050 Product Code: ACH-0145228 INN or Proposed INN: N/A Other descriptive name: ACH-0145228 Product Name: ALXN2050 Product Code: ACH-0145228 INN or Proposed INN: N/A Other descriptive name: ACH-0145228 | Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 26 | Phase 2 | United States;New Zealand;Italy;United Kingdom;Korea, Republic of | ||
| 107 | EUCTR2018-002734-20-CZ (EUCTR) | 07/08/2020 | 15/03/2019 | A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor Therapy | An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 42 | Phase 2 | United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Czech Republic;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands | ||
| 108 | EUCTR2019-003440-74-GB (EUCTR) | 14/05/2020 | 17/03/2020 | Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab | Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab - ALXN1210-PNH-401 | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: ULTOMIRIS Product Name: ULTOMIRIS Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 4 | United Kingdom | ||
| 109 | EUCTR2019-000130-20-GB (EUCTR) | 23/04/2020 | 19/11/2019 | REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability. | An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 147 | Phase 3 | United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands | ||
| 110 | EUCTR2019-000130-20-NL (EUCTR) | 01/04/2020 | 16/10/2019 | A trial with REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability. | An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 147 | Phase 3 | United States;Hong Kong;Taiwan;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Netherlands;Germany | ||
| 111 | EUCTR2017-001418-27-FI (EUCTR) | 01/04/2020 | 08/10/2019 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Netherlands;Germany;Norway;Sweden | ||
| 112 | EUCTR2017-001418-27-PT (EUCTR) | 04/03/2020 | 28/11/2019 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Portugal;Czech Republic;Slovenia;Spain;Ireland;Norway;Netherlands;Germany;Italy;United Kingdom | ||
| 113 | NCT04330534 (ClinicalTrials.gov) | March 3, 2020 | 23/3/2020 | First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH | A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Drug: BCX9930;Drug: Placebo | BioCryst Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 168 | Phase 1/Phase 2 | Austria;South Africa;United Kingdom |
| 114 | EUCTR2017-001418-27-SE (EUCTR) | 20/02/2020 | 08/11/2019 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - proposed biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris | Amgen Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | United States;Portugal;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden | ||
| 115 | EUCTR2019-000130-20-CZ (EUCTR) | 12/02/2020 | 14/11/2019 | REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability. | An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 147 | Phase 3 | United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Czech Republic;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands | ||
| 116 | EUCTR2019-001106-23-FR (EUCTR) | 04/02/2020 | 15/10/2019 | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL-2 | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 119 | Phase 3 | United States;Serbia;Hong Kong;Thailand;Spain;Russian Federation;United Kingdom;France;Canada;Belgium;Malaysia;Poland;Australia;Bulgaria;Germany;Japan;Korea, Republic of | ||
| 117 | EUCTR2019-000130-20-HU (EUCTR) | 27/01/2020 | 25/11/2019 | REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability. | An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 147 | Phase 3 | United States;Hong Kong;Taiwan;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands | ||
| 118 | EUCTR2019-001106-23-ES (EUCTR) | 22/01/2020 | 14/01/2020 | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL-2 | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 119 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Poland;Malaysia;Belgium;Australia;Bulgaria;Germany;Japan;Korea, Republic of | ||
| 119 | EUCTR2017-001418-27-IE (EUCTR) | 15/01/2020 | 02/10/2019 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden | ||
| 120 | EUCTR2019-001106-23-GB (EUCTR) | 19/12/2019 | 30/09/2019 | An Open Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). | An Open Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pegcetacoplan (APL-2) Product Code: Pegcetacoplan (APL-2) INN or Proposed INN: pegcetacoplan Other descriptive name: Pegcetacoplan (APL-2) | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Malaysia;Australia;Bulgaria;Japan;Korea, Republic of | ||
| 121 | NCT04170023 (ClinicalTrials.gov) | December 16, 2019 | 6/11/2019 | Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy | A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ALXN2050 | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 26 | Phase 2 | Canada;Italy;Korea, Republic of;New Zealand;Spain;Turkey;United Kingdom;Germany;United States |
| 122 | EUCTR2019-000130-20-IT (EUCTR) | 12/12/2019 | 24/05/2021 | REGN3918 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to evaluate its long term safety, efficacy and tolerability. | An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria - R3918-PNH-1868 | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: [REGN3918] INN or Proposed INN: REGN3918 | REGENERON PHARMACEUTICALS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 147 | Phase 3 | United States;Hong Kong;Taiwan;United Kingdom;Italy;Czech Republic;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands;Korea, Republic of | ||
| 123 | NCT04162470 (ClinicalTrials.gov) | December 3, 2019 | 11/11/2019 | REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability. | An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Drug: REGN3918 | Regeneron Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 24 | Phase 3 | Hong Kong;Hungary;Korea, Republic of;Malaysia;Taiwan;United Kingdom |
| 124 | EUCTR2017-001418-27-CZ (EUCTR) | 22/11/2019 | 05/08/2019 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 3 | United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden | ||
| 125 | EUCTR2017-001418-27-SI (EUCTR) | 23/10/2019 | 10/09/2019 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Germany;Netherlands;Norway;Sweden | ||
| 126 | EUCTR2017-001418-27-NO (EUCTR) | 18/10/2019 | 22/08/2019 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;Portugal;Czechia;Taiwan;Slovenia;Finland;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Brazil;Norway;Germany;Netherlands;Sweden | ||
| 127 | EUCTR2019-001453-10-ES (EUCTR) | 16/09/2019 | 09/08/2019 | A Low-Interventional Study Documenting the Efficacy, Health-Related Quality of Life, and Safety of Standard-Of-Care Treatment with Eculizumab or Treatment with Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria | A MULTICENTER, LOW-INTERVENTIONAL STUDY DOCUMENTING THE EFFICACY, HEALTH-RELATED QUALITY OF LIFE, AND SAFETY OF STANDARD-OF-CARE TREATMENT WITH ECULIZUMAB OR TREATMENT WITH RAVULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal nocturnal hemoglobinuria (PNH) that is treated with either eculizumab or ravulizumab as per local label MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Soliris 300 Product Name: ECULIZUMAB INN or Proposed INN: ECULIZUMAB Trade Name: ULTOMIRIS Product Name: Ultomiris INN or Proposed INN: RAVULIZUMAB Other descriptive name: Ultomiris | Roche Farma S. A. U. que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 4 | United States;Taiwan;Greece;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Argentina;Poland;Brazil;Romania;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 128 | NCT04085601 (ClinicalTrials.gov) | August 27, 2019 | 9/9/2019 | A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH | A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 53 | Phase 3 | Colombia;Hong Kong;Malaysia;Mexico;Peru;Philippines;Poland;Serbia;Singapore;Thailand |
| 129 | EUCTR2019-001106-23-BG (EUCTR) | 09/08/2019 | 09/05/2019 | An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) | An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: pegcetacoplan Product Code: pegcetacoplan INN or Proposed INN: pegcetacoplan Other descriptive name: pegcetacoplan | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | Serbia;United States;Hong Kong;Spain;Thailand;Ireland;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Malaysia;Australia;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of | ||
| 130 | NCT04058158 (ClinicalTrials.gov) | August 7, 2019 | 12/8/2019 | A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria | A Phase III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity Between SB12 (Proposed Eculizumab Biosimilar) and Soliris® in Subjects With Paroxysmal Nocturnal Haemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Drug: SB12 (proposed eculizumab biosimilar);Drug: Soliris (eculizumab) | Samsung Bioepis Co., Ltd. | NULL | Completed | 18 Years | N/A | All | 50 | Phase 3 | India;Korea, Republic of;Malaysia;Mexico;Romania;Taiwan;Thailand;Ukraine |
| 131 | EUCTR2018-002734-20-NL (EUCTR) | 29/07/2019 | 02/04/2019 | A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor Therapy | An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2 | United States;Hong Kong;Taiwan;Korea, Democratic People's Republic of;United Kingdom;Italy;Czech Republic;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Netherlands;Germany | ||
| 132 | EUCTR2018-002734-20-GB (EUCTR) | 24/07/2019 | 22/03/2019 | A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor Therapy | An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2 | United States;Taiwan;Hong Kong;Korea, Democratic People's Republic of;United Kingdom;Italy;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands | ||
| 133 | EUCTR2018-004220-11-PL (EUCTR) | 23/07/2019 | 13/05/2019 | A Research Study to Gather Scientific Information About the Efficacy and Safety of the Investigational Drug APL-2 In Treating Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated with Anemia, In a Randomly Assigned Comparison with the Current Standard of Care Treatment Approved for PNH | A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PRINCE | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL-2 | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 54 | Phase 3 | Serbia;Philippines;Hungary;Hong Kong;Mexico;Argentina;Thailand;Malaysia;Brazil;Poland;Peru;Colombia | ||
| 134 | EUCTR2017-002370-39-DE (EUCTR) | 02/07/2019 | 15/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 105 | Phase 3 | Russian Federation;United States;United Kingdom;Spain;Canada;Austria;Czech Republic;Netherlands;Sweden;Turkey;Belgium;Finland;Brazil;Korea, Republic of;Italy;Australia;France;Germany | ||
| 135 | EUCTR2017-002370-39-NL (EUCTR) | 01/07/2019 | 27/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Brazil;Belgium;Australia;Netherlands;Germany;Sweden;Korea, Republic of | ||
| 136 | NCT04671810 (ClinicalTrials.gov) | June 13, 2019 | 1/12/2020 | Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® | Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Paroxysmal Nocturnal Hemoglobinuria. | Paroxysmal Nocturnal Hemoglobinuria | Biological: Elizaria® | AO GENERIUM | NULL | Active, not recruiting | 18 Years | N/A | All | 44 | Russian Federation | |
| 137 | EUCTR2017-004268-36-NL (EUCTR) | 23/05/2019 | 09/08/2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: n/a Other descriptive name: n/a Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Netherlands;Germany;Japan;Korea, Republic of | ||
| 138 | EUCTR2018-002734-20-IT (EUCTR) | 16/05/2019 | 25/05/2021 | A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor Therapy | An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy - - | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: [REGN3918] INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | REGENERON PHARMACEUTICALS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 42 | Phase 2 | United States;Hong Kong;Taiwan;Korea, Democratic People's Republic of;United Kingdom;Italy;Czech Republic;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands | ||
| 139 | NCT03946748 (ClinicalTrials.gov) | May 16, 2019 | 8/5/2019 | Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | An Open-Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor-Naive or Have Not Recently Received Complement Inhibitor Therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: REGN3918 | Regeneron Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 24 | Phase 2 | Hong Kong;Hungary;Korea, Republic of;Malaysia;United Kingdom |
| 140 | EUCTR2018-002734-20-HU (EUCTR) | 10/05/2019 | 25/03/2019 | A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor Therapy | An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 Product Name: REGN3918 Product Code: REGN3918 INN or Proposed INN: REGN3918 Other descriptive name: REGN3918 | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 42 | Phase 2 | United States;Hong Kong;Taiwan;Korea, Democratic People's Republic of;United Kingdom;Italy;Czech Republic;Hungary;Canada;Malaysia;Poland;Singapore;Romania;South Africa;Germany;Netherlands | ||
| 141 | NCT03818607 (ClinicalTrials.gov) | April 24, 2019 | 17/1/2019 | A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH | A Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: ABP 959;Drug: Eculizumab | Amgen | NULL | Completed | 18 Years | N/A | All | 42 | Phase 3 | United States;Czechia;Finland;France;Ireland;Italy;Netherlands;Norway;Portugal;Slovenia;Spain;Sweden;Turkey;United Kingdom;Germany;Taiwan |
| 142 | EUCTR2017-002370-39-CZ (EUCTR) | 09/04/2019 | 02/01/2019 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 105 | Phase 3 | Russian Federation;United States;United Kingdom;Spain;Canada;Czech Republic;Austria;Netherlands;Sweden;Turkey;Belgium;Finland;Brazil;Korea, Republic of;Italy;Australia;France;Germany | ||
| 143 | NCT03896152 (ClinicalTrials.gov) | April 5, 2019 | 28/3/2019 | Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | A Multi-center, Randomized, Open-label, Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis | Paroxysmal Nocturnal Hemoglobinuria | Drug: LNP023 | Novartis Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 13 | Phase 2 | Korea, Republic of;Malaysia;Singapore;Taiwan;Russian Federation |
| 144 | NCT04060264 (ClinicalTrials.gov) | April 4, 2019 | 13/8/2019 | Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria | Randomized, Open-Label, International, Multi-center, Comparative Study of Efficacy and Safety of BCD-148 (JSC BIOCAD, Russia) and Soliris® in Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Biological: BCD-148;Biological: Soliris | Biocad | NULL | Completed | 18 Years | 65 Years | All | 28 | Phase 3 | Russian Federation |
| 145 | EUCTR2017-002370-39-AT (EUCTR) | 01/04/2019 | 24/01/2019 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 105 | Phase 3 | United States;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Germany;Netherlands;Sweden;Korea, Republic of | ||
| 146 | NCT03866681 (ClinicalTrials.gov) | April 1, 2019 | 28/2/2019 | Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH | Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory Classic Paroxysmal Nocturnal Hemoglobinuria ,a Prospective Study | Paroxysmal Nocturnal Hemoglobinuria | Drug: sirolimus | Peking Union Medical College Hospital | NULL | Not yet recruiting | 18 Years | 70 Years | All | 40 | Phase 4 | China |
| 147 | EUCTR2017-002370-39-SE (EUCTR) | 27/03/2019 | 30/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Trade Name: ULTOMIRIS Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Sweden;Korea, Republic of | ||
| 148 | EUCTR2016-004129-18-LT (EUCTR) | 15/03/2019 | 29/01/2019 | An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study | CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study | Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: rVA576 INN or Proposed INN: rVA576 Other descriptive name: COVERSIN | Akari Therapeutics Plc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom;India | ||
| 149 | EUCTR2017-002370-39-BE (EUCTR) | 13/03/2019 | 23/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Germany;Netherlands;Korea, Republic of;Sweden | ||
| 150 | NCT03748823 (ClinicalTrials.gov) | March 5, 2019 | 19/11/2018 | Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria | Combination Product: Ravulizumab OBDS;Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 136 | Phase 3 | United States;Australia;Austria;Belgium;Brazil;Canada;Finland;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey |
| 151 | EUCTR2017-004268-36-IT (EUCTR) | 04/03/2019 | 26/09/2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: n/a Other descriptive name: n/a Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 152 | EUCTR2017-002370-39-IT (EUCTR) | 21/02/2019 | 18/01/2021 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab. | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab. - NA | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: NA Product Name: Ravulizumab Product Code: [ALXN1210] Other descriptive name: Ravulizumab Trade Name: NA Product Name: Ravulizumab Product Code: [ALXN1210] Other descriptive name: Ravulizumab | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Brazil;Belgium;Australia;Netherlands;Germany;Sweden;Korea, Republic of | ||
| 153 | EUCTR2017-002370-39-GB (EUCTR) | 19/02/2019 | 26/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden | ||
| 154 | NCT03520647 (ClinicalTrials.gov) | February 19, 2019 | 9/5/2018 | Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis | Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis | Severe Aplastic Anemia (SAA);Hypo-Plastic Myelodysplastic Syndrome (MDS);Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Cyclophosphamide;Other: Peripheral Blood Stem Cells | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Recruiting | 4 Years | 75 Years | All | 56 | Phase 2 | United States |
| 155 | EUCTR2018-003956-19-FI (EUCTR) | 13/02/2019 | 28/01/2019 | Study of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3, Multicenter, Open-Label, Single-Arm Study to Confirm the Safety and Efficacy of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria - Phase 3 Study in Treatment-Naive Paroxysmal Nocturnal Hemoglobinuria Patients | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Zilucoplan Product Code: RA101495 INN or Proposed INN: ZILUCOPLAN Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;Hong Kong;Taiwan;Finland;Spain;Turkey;Russian Federation;Italy;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Malaysia;Belgium;Brazil;Australia;Georgia;Germany;New Zealand;Korea, Republic of | ||
| 156 | EUCTR2017-002370-39-ES (EUCTR) | 08/02/2019 | 18/01/2019 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden | ||
| 157 | EUCTR2017-001418-27-DE (EUCTR) | 29/01/2019 | 27/07/2017 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Czech Republic;Slovenia;Spain;Ireland;Norway;Netherlands;Germany;Italy;United Kingdom | ||
| 158 | EUCTR2017-002370-39-FI (EUCTR) | 29/01/2019 | 21/12/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Brazil;Belgium;Australia;Netherlands;Germany;Sweden;Korea, Republic of | ||
| 159 | EUCTR2017-001418-27-NL (EUCTR) | 22/01/2019 | 02/11/2017 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Spain;Norway;Germany;Netherlands;Italy;United Kingdom;Korea, Republic of | ||
| 160 | EUCTR2017-004268-36-GB (EUCTR) | 08/01/2019 | 21/08/2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug Pegcetacoplan In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pegcetacoplan (APL-2) Product Code: Pegcetacoplan (APL-2) INN or Proposed INN: n/a Other descriptive name: n/a Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 161 | EUCTR2017-002370-39-FR (EUCTR) | 28/12/2018 | 20/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden | ||
| 162 | EUCTR2017-001418-27-GB (EUCTR) | 14/12/2018 | 11/12/2018 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - ABP 959 in PNH | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Spain;Norway;Netherlands;Germany;Italy;United Kingdom | ||
| 163 | EUCTR2017-002820-26-NO (EUCTR) | 07/12/2018 | 16/10/2018 | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: ALXN1210 Other descriptive name: ALXN1210 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 3 | United States;France;Netherlands;Norway;United Kingdom | ||
| 164 | EUCTR2017-004268-36-BE (EUCTR) | 25/10/2018 | 23/07/2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: n/a Other descriptive name: n/a Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 165 | EUCTR2017-004268-36-FR (EUCTR) | 05/10/2018 | 20/08/2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: n/a Other descriptive name: n/a Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 166 | EUCTR2017-004268-36-DE (EUCTR) | 01/10/2018 | 14/06/2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: n/a Other descriptive name: n/a Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 167 | EUCTR2017-004268-36-ES (EUCTR) | 19/09/2018 | 07/08/2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: n/a Other descriptive name: n/a Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 168 | NCT03531255 (ClinicalTrials.gov) | August 27, 2018 | 9/5/2018 | Pegcetacoplan Long Term Safety and Efficacy Extension Study | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) | PNH | Drug: Pegcetacoplan | Apellis Pharmaceuticals, Inc. | NULL | Active, not recruiting | 18 Years | N/A | All | 160 | Phase 3 | United States;Australia;Belgium;Bulgaria;Canada;France;Germany;Hong Kong;Japan;Korea, Republic of;Malaysia;Russian Federation;Serbia;Singapore;Spain;Thailand;United Kingdom |
| 169 | NCT03593200 (ClinicalTrials.gov) | August 16, 2018 | 28/6/2018 | A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With PNH | Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH). | PNH | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 4 | Phase 2 | Bulgaria;Serbia;Greece;Poland;Romania |
| 170 | EUCTR2017-000665-79-GB (EUCTR) | 23/07/2018 | 27/04/2018 | A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who Completed Clinical Study ACH471-100 | An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment with ACH-0144471 in Patients with PNH who Completed Clinical Study ACH471-100 | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ACH-0144471 INN or Proposed INN: Not available Other descriptive name: ACH-0144471 Product Code: ACH-0144471 INN or Proposed INN: Not available Other descriptive name: ACH-0144471 Product Code: ACH-0144471 INN or Proposed INN: Not available Other descriptive name: ACH-0144471 | Achillion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | New Zealand;Italy;United Kingdom;Korea, Republic of | ||
| 171 | EUCTR2016-003526-16-GB (EUCTR) | 20/07/2018 | 23/04/2018 | A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have poor Response to Eculizumab Monotherapy | A Phase 2 Open-label Study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Danicopan Product Code: ALXN2040 (ACH-0144471) INN or Proposed INN: Not available Other descriptive name: ACH-0144471 Product Name: Danicopan Product Code: ALXN2040 (ACH-0144471) INN or Proposed INN: Not available Other descriptive name: ACH-0144471 Product Name: Danicopan Product Code: ALXN2040 (ACH-0144471) INN or Proposed INN: Not available Other descriptive name: ACH-0144471 | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 14 | Phase 2 | United States;Italy;United Kingdom | ||
| 172 | EUCTR2017-005140-16-BG (EUCTR) | 18/07/2018 | 26/03/2018 | Phase IIa study of APL-2 in patients with PNH | A Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: APL-2 INN or Proposed INN: Not yet assigned | Apellis Pharmaceuticals | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Serbia;Greece;Poland;Romania;Bulgaria | ||
| 173 | NCT03500549 (ClinicalTrials.gov) | June 14, 2018 | 10/4/2018 | Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: APL-2;Drug: Soliris | Apellis Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 80 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Japan;Korea, Republic of;Russian Federation;Spain;United Kingdom |
| 174 | NCT03588026 (ClinicalTrials.gov) | June 7, 2018 | 1/6/2018 | Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 | Investigational Product ; Coversin. Phase III Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: rVA576;Other: Standard of care (SOC) | AKARI Therapeutics | NULL | Completed | 18 Years | N/A | All | 9 | Phase 3 | Kazakhstan;Lithuania;Sri Lanka |
| 175 | NCT03472885 (ClinicalTrials.gov) | May 8, 2018 | 15/3/2018 | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab | A Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Danicopan;Drug: Eculizumab | Alexion Pharmaceuticals | Achillion, a wholly owned subsidiary of Alexion | Completed | 18 Years | 65 Years | All | 12 | Phase 2 | United States;Italy;United Kingdom |
| 176 | EUCTR2017-005140-16-GR (EUCTR) | 04/05/2018 | 02/04/2018 | Phase IIa study of APL-2 in patients with PNH | A Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: APL-2 INN or Proposed INN: Not yet assigned | Apellis Pharmaceuticals | NULL | Not Recruiting | Female: yes Male: yes | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Serbia;Greece;Poland;Romania;Bulgaria | ||
| 177 | EUCTR2017-002820-26-NL (EUCTR) | 21/03/2018 | 10/01/2018 | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: ALXN1210 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 13 | Phase 3 | France;United States;Russian Federation;Norway;Netherlands;United Kingdom | ||
| 178 | EUCTR2016-002652-25-IT (EUCTR) | 16/03/2018 | 08/02/2021 | A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) - A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH | Untreated Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ACH-0144471 Product Code: ACH-0144471 Other descriptive name: ACH-0144471 Product Code: ACH-0144471 Other descriptive name: ACH-0144471 | ACHILLION PHARMACEUTICALS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2 | United Kingdom;New Zealand;Italy;Korea, Republic of | ||
| 179 | EUCTR2017-003847-39-LT (EUCTR) | 12/03/2018 | 23/01/2018 | A study to assess the safety and efficacy of rVA576 in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) | CAPSTONE: Phase III Confirmatory Assessment Protocol: rVA576 Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) - CAPSTONE | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: rVA576 INN or Proposed INN: NA | Akari Therapeutics Plc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | Ecuador;Romania;Turkey;Lithuania;Peru;Kazakhstan;Sri Lanka | ||
| 180 | NCT03406507 (ClinicalTrials.gov) | February 22, 2018 | 5/1/2018 | A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Completed | N/A | 17 Years | All | 13 | Phase 3 | United States;France;Netherlands;Norway;Russian Federation;United Kingdom |
| 181 | EUCTR2016-004129-18-NL (EUCTR) | 14/02/2018 | 16/11/2017 | An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial study | CONSERVE: Coversin Long Term Safety and Efficacy Surveillance Study | Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Coversin Product Code: rVA576 INN or Proposed INN: COVERSIN Other descriptive name: COVERSIN | Akari Therapeutics Plc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Argentina;Poland;Lithuania;Netherlands;United Kingdom | ||
| 182 | EUCTR2017-000888-33-DE (EUCTR) | 05/02/2018 | 17/10/2017 | A Study evaluating the safety and efficacy of LNP023 in patients with a blood disorder called paroxysmal nocturnal hemoglobinuria (PNH) | An open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of LNP023 when administered in addition to Standard of Care (SoC) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: HLGT;Classification code 10018911;Term: Haemolyses and related conditions;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | France;Germany;Italy | ||
| 183 | EUCTR2016-002652-25-GB (EUCTR) | 02/02/2018 | 27/10/2017 | A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Untreated Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ACH-0144471 INN or Proposed INN: Not available Other descriptive name: ACH-0144471 Product Code: ACH-0144471 INN or Proposed INN: Not available Other descriptive name: ACH-0144471 Product Code: ACH-0144471 INN or Proposed INN: Not available Other descriptive name: ACH-0144471 | Achillion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 12 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | New Zealand;Italy;United Kingdom;Korea, Republic of | ||
| 184 | EUCTR2017-000888-33-FR (EUCTR) | 15/01/2018 | 22/01/2018 | A Study evaluating the safety and efficacy of LNP023 in patients with a blood disorder called paroxysmal nocturnal hemoglobinuria (PNH) | An open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of LNP023 when administered in addition to Standard of Care (SoC) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: HLGT;Classification code 10018911;Term: Haemolyses and related conditions;System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Germany;Italy | ||
| 185 | NCT03333486 (ClinicalTrials.gov) | December 7, 2017 | 2/11/2017 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Roswell Park Cancer Institute | NULL | Active, not recruiting | 1 Year | 75 Years | All | 31 | Phase 2 | United States |
| 186 | NCT04463056 (ClinicalTrials.gov) | November 29, 2017 | 3/7/2020 | Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH | A Multicenter, Open Label, Randomized, Parallel-group Study of Efficacy and Safety of Eculizumab (JSC GENERIUM, Russia) vs. Soliris® (Alexion Pharma GmbH, Switzerland) in Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria;Marchiafava-Micheli Syndrome;Paroxysmal Hemoglobinuria | Biological: Elizaria®;Biological: Soliris® | AO GENERIUM | NULL | Completed | 18 Years | 75 Years | All | 32 | Phase 3 | Russian Federation |
| 187 | EUCTR2017-002820-26-GB (EUCTR) | 24/11/2017 | 26/10/2017 | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: ALXN1210 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 13 | Phase 3 | United States;France;Russian Federation;Norway;Netherlands;United Kingdom | ||
| 188 | JPRN-JapicCTI-173517 | 17/10/2017 | 17/02/2017 | An adaptive Phase I/II study of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) | An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Intervention name : RO7112689 INN of the intervention : Crovalimab Dosage And administration of the intervention : Intravenous and Subcutaneous Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : - | CHUGAI PHARMACEUTICAL CO., LTD. | NULL | complete | 18 | 75 | BOTH | 44 | Phase 1-2 | Japan, Asia except Japan, Europe |
| 189 | EUCTR2016-002128-10-IT (EUCTR) | 19/09/2017 | 06/02/2018 | Studio volto a valutare la sicurezza, l’efficacia, la farmacocinetica e la farmacodinamica di RO7112689 in volontari sani e in pazienti con Emoglobinuria Parossistica Notturna | An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) - An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: RO7112689/F01 Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 | F. HOFFMANN - LA ROCHE LTD. | NULL | Not Recruiting | Female: yes Male: yes | 49 | Phase 1;Phase 2 | France;Hungary;Brazil;Netherlands;Germany;New Zealand;Japan;Italy;Korea, Republic of | ||
| 190 | EUCTR2017-001418-27-ES (EUCTR) | 28/08/2017 | 31/07/2017 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH);Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 INN or Proposed INN: ECULIZUMAB Other descriptive name: ABP 959 - biosimilar to eculizumab Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Amgen Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | Portugal;Slovenia;Finland;Spain;Ireland;Russian Federation;Italy;United Kingdom;France;Czech Republic;Germany;Netherlands;Norway;Japan;Sweden;Korea, Republic of | ||
| 191 | EUCTR2016-002128-10-FR (EUCTR) | 17/08/2017 | 08/09/2017 | A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria | An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: RO7112689/F01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 49 | Phase 1;Phase 2 | France;Hungary;Netherlands;Germany;Japan;Italy;Korea, Republic of | ||
| 192 | EUCTR2016-002026-36-NL (EUCTR) | 27/07/2017 | 27/03/2017 | ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 197 | Phase 3 | United States;Spain;Germany;Netherlands;Italy;United Kingdom | ||
| 193 | EUCTR2016-002026-36-IT (EUCTR) | 24/07/2017 | 08/06/2021 | ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal NocturnalHemoglobinuria (PNH) Currently Treated With Eculizumab - ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 Trade Name: SOLIRIS - 300 MG CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO 1 FLACONCINO (VETRO) 30 ML (10 MG/ML) INN or Proposed INN: ECULIZUMAB | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 192 | Phase 3 | United States;Spain;Netherlands;Germany;United Kingdom;Italy | ||
| 194 | NCT03225287 (ClinicalTrials.gov) | July 17, 2017 | 17/7/2017 | Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Zilucoplan (RA101495) | Ra Pharmaceuticals, Inc. | NULL | Terminated | 18 Years | N/A | All | 19 | Phase 2 | United States;Australia;Canada;Finland;Germany;Hungary;New Zealand;United Kingdom;Denmark |
| 195 | NCT03181633 (ClinicalTrials.gov) | June 22, 2017 | 5/6/2017 | A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | An Open-Label Study to Evaluate Efficacy and Safety of Long-Term Treatment With ACH-0144471 in Participants Who Completed Clinical Study ACH471-100 | Paroxysmal Nocturnal Hemoglobinuria | Drug: ACH-0144471 | Alexion Pharmaceuticals | Achillion, a wholly owned subsidiary of Alexion | Completed | 18 Years | N/A | All | 8 | Phase 2 | Italy;Korea, Republic of;New Zealand |
| 196 | EUCTR2016-003522-16-FI (EUCTR) | 20/06/2017 | 27/12/2016 | Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH) | A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | ||
| 197 | EUCTR2016-002026-36-DE (EUCTR) | 14/06/2017 | 23/02/2017 | ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 192 | Phase 3 | France;United States;Canada;Spain;Australia;Netherlands;Germany;Japan;Italy;United Kingdom;Korea, Republic of | ||
| 198 | EUCTR2016-002026-36-ES (EUCTR) | 31/05/2017 | 22/03/2017 | ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 19.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 192 | Phase 3 | United States;Spain;Germany | ||
| 199 | EUCTR2016-002026-36-GB (EUCTR) | 30/05/2017 | 14/02/2017 | ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 192 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Netherlands;Germany;United Kingdom | ||
| 200 | EUCTR2016-003522-16-DK (EUCTR) | 01/05/2017 | 14/02/2017 | Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A PHASE 2 MULTICENTER, OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Canada;Finland;Australia;Denmark;Germany;New Zealand;United Kingdom | ||
| 201 | EUCTR2016-003523-34-DK (EUCTR) | 01/05/2017 | 14/02/2017 | Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | United States;Hungary;Canada;Finland;Australia;Denmark;Germany;United Kingdom;New Zealand | ||
| 202 | EUCTR2016-002025-11-FI (EUCTR) | 28/04/2017 | 01/11/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 214 | Phase 3 | United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Czech Republic;Poland;Belgium;Denmark;Germany;Netherlands;Korea, Republic of;Sweden | ||
| 203 | EUCTR2016-003523-34-FI (EUCTR) | 06/04/2017 | 27/12/2016 | Extension Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Zilucoplan Product Code: RA101495 INN or Proposed INN: Zilucoplan Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 palmitoylated linker | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | United States;Hungary;Finland;Denmark;Australia;Germany;United Kingdom;New Zealand | ||
| 204 | NCT03053102 (ClinicalTrials.gov) | March 31, 2017 | 1/2/2017 | A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ACH-0144471 | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 10 | Phase 2 | Italy;Korea, Republic of;New Zealand;United Kingdom |
| 205 | EUCTR2016-002025-11-NL (EUCTR) | 27/03/2017 | 08/11/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 214 | Phase 3 | United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Belgium;Poland;Denmark;Netherlands;Germany;Korea, Republic of | ||
| 206 | EUCTR2016-003523-34-HU (EUCTR) | 17/03/2017 | 31/01/2017 | Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | United States;Hungary;Finland;Denmark;Australia;Germany;United Kingdom;New Zealand | ||
| 207 | EUCTR2016-004129-18-GB (EUCTR) | 17/03/2017 | 21/12/2016 | An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study | CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study | Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: rVA576 INN or Proposed INN: rVA576 Other descriptive name: rVA576 | Akari Therapeutics Plc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom | ||
| 208 | EUCTR2016-003522-16-HU (EUCTR) | 17/03/2017 | 31/01/2017 | Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | ||
| 209 | EUCTR2016-002025-11-PL (EUCTR) | 16/03/2017 | 20/03/2017 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 301 | Phase 3 | Portugal;United States;Taiwan;Estonia;Thailand;Spain;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Finland;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Poland;Belgium;Singapore;Germany;Japan;Sweden | ||
| 210 | EUCTR2016-002025-11-BE (EUCTR) | 14/03/2017 | 17/10/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 301 | Phase 3 | Portugal;Taiwan;Estonia;Thailand;Spain;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Czechia;Finland;Korea, Democratic People's Republic of;Turkey;United States Minor Outlying Islands;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden | ||
| 211 | EUCTR2016-002128-10-DE (EUCTR) | 14/03/2017 | 24/10/2016 | A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria | An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: RO7112689/F01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 Product Code: RO7112689/F03-01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 Product Code: RO7112689/F03-10 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 49 | Phase 1;Phase 2 | France;Hungary;Netherlands;Germany;Italy;Japan;Korea, Republic of | ||
| 212 | NCT03829449 (ClinicalTrials.gov) | March 13, 2017 | 20/12/2018 | rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study | CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study | Paroxysmal Nocturnal Hemoglobinuria | Drug: rVA576 (Coversin) | AKARI Therapeutics | NULL | Terminated | 18 Years | N/A | All | 15 | Phase 3 | Poland |
| 213 | EUCTR2016-004129-18-PL (EUCTR) | 07/03/2017 | 18/01/2017 | An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial study | CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study | Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: rVA576 (Coversin) Product Code: rVA576 INN or Proposed INN: rVA576 (COVERSIN) Other descriptive name: rVA576 (COVERSIN) | Akari Therapeutics Plc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom | ||
| 214 | EUCTR2016-002025-11-EE (EUCTR) | 20/02/2017 | 23/01/2017 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Name: Ultomiris Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 301 | Phase 3 | Portugal;United States;Taiwan;Estonia;Thailand;Spain;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Finland;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden | ||
| 215 | EUCTR2016-003523-34-GB (EUCTR) | 20/02/2017 | 26/09/2017 | Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | ||
| 216 | EUCTR2016-003522-16-GB (EUCTR) | 15/02/2017 | 03/01/2017 | Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH) | A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | ||
| 217 | EUCTR2016-002025-11-IT (EUCTR) | 07/02/2017 | 17/06/2021 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Na¿ve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a compleme | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ALXN1210 Product Code: ALXN1210 Trade Name: Soliris Product Name: ALXN1210 Product Code: [ALXN1210] INN or Proposed INN: ECULIZUMAB | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 214 | Phase 3 | United States;Portugal;Estonia;Finland;Spain;Austria;Italy;United Kingdom;Czech Republic;Poland;Belgium;Denmark;Germany;Netherlands;Korea, Republic of;Sweden | ||
| 218 | EUCTR2016-002025-11-CZ (EUCTR) | 02/02/2017 | 17/10/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 214 | Phase 3 | United States;United Kingdom;Portugal;Spain;Czech Republic;Sweden;Netherlands;Austria;Belgium;Finland;Korea, Republic of;Denmark;Poland;Italy;Germany;Estonia | ||
| 219 | EUCTR2016-002025-11-PT (EUCTR) | 30/01/2017 | 21/10/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 214 | Phase 3 | United States;Portugal;Estonia;Poland;Spain;Austria;Denmark;Germany;United Kingdom;Korea, Republic of | ||
| 220 | EUCTR2016-002128-10-HU (EUCTR) | 24/01/2017 | 14/11/2016 | A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria | An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: RO7112689/F01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 49 | Phase 1;Phase 2 | France;Hungary;Brazil;Netherlands;Germany;New Zealand;Japan;Italy;Korea, Republic of | ||
| 221 | EUCTR2016-002025-11-ES (EUCTR) | 16/01/2017 | 04/11/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 214 | Phase 3 | United States;Spain;Austria;Denmark;Germany;Korea, Republic of | ||
| 222 | EUCTR2016-002025-11-AT (EUCTR) | 11/01/2017 | 14/12/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 214 | Phase 3 | Portugal;United States;Estonia;Taiwan;Spain;Thailand;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Korea, Republic of;Finland;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden | ||
| 223 | EUCTR2016-002025-11-DK (EUCTR) | 23/12/2016 | 20/10/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 214 | Phase 3 | United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Belgium;Poland;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 224 | NCT02946463 (ClinicalTrials.gov) | December 20, 2016 | 25/10/2016 | ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Biological: Ravulizumab;Biological: Eculizumab | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 246 | Phase 3 | United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Czechia;Estonia;France;Germany;Italy;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Russian Federation;Singapore;Spain;Sweden;Taiwan;Thailand;Turkey;United Kingdom;Colombia |
| 225 | EUCTR2016-002025-11-DE (EUCTR) | 20/12/2016 | 07/10/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 214 | Phase 3 | Portugal;United States;Taiwan;Estonia;Thailand;Spain;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Korea, Republic of;Finland;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden | ||
| 226 | EUCTR2016-002025-11-GB (EUCTR) | 02/12/2016 | 07/10/2016 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 214 | Phase 3 | United States;Portugal;Estonia;Finland;Spain;Austria;United Kingdom;Czech Republic;Poland;Belgium;Denmark;Germany;Netherlands;Korea, Republic of;Sweden | ||
| 227 | EUCTR2016-002943-40-ES (EUCTR) | 21/11/2016 | 30/09/2016 | A Clinical Study with Investigational Drug, ALN-CC5, in patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab | A Phase 2, Open-label, Single Dose, Study of Subcutaneously Administered ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALN-CC5 INN or Proposed INN: ALN-62643 | Alnylam Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Taiwan;Finland;Spain;Turkey;Austria;Russian Federation;Israel;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Denmark;Bulgaria;Norway;Netherlands;Germany;Korea, Republic of;Sweden | ||
| 228 | NCT03157635 (ClinicalTrials.gov) | November 14, 2016 | 1/11/2016 | Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria | An Adaptive Phase I/II Study to Assess Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Crovalimab in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Hemoglobinuria, Nocturnal | Drug: Crovalimab;Drug: Placebo | Hoffmann-La Roche | NULL | Active, not recruiting | 18 Years | 75 Years | All | 59 | Phase 1/Phase 2 | France;Germany;Hungary;Italy;Japan;Korea, Republic of;Netherlands |
| 229 | EUCTR2016-002128-10-NL (EUCTR) | 14/09/2016 | 05/09/2016 | A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria | An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: RO7112689/F01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: no Male: yes | 74 | Phase 1;Phase 2 | France;Hungary;Czech Republic;Brazil;Germany;Netherlands;Japan;Italy;United Kingdom | ||
| 230 | EUCTR2016-002067-33-GB (EUCTR) | 18/07/2016 | 16/08/2016 | An Open Clincal Trial of Coversin in Patients with PNH | COBALT: Coversin Global Study: An Open-Label, Safety and Efficacy Trial in PNH Patients | Paroxysmal nocturnal haemoglobinuria (PNH) MedDRA version: 20.0;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Coversin Product Code: rVA576 INN or Proposed INN: COVERSIN Other descriptive name: COVERSIN | Akari Therapeutics Plc | NULL | Not Recruiting | Female: yes Male: yes | 10 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 231 | NCT02591862 (ClinicalTrials.gov) | February 2016 | 10/9/2015 | Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) | Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms | Paroxysmal Nocturnal Haemoglobinuria (PNH) | Drug: Coversin | AKARI Therapeutics | Radboud University | Completed | 18 Years | 80 Years | All | 6 | Phase 2 | Netherlands |
| 232 | EUCTR2015-003778-34-NL (EUCTR) | 13/01/2016 | 10/12/2015 | Coversin in Paroxysmal Nocturnal Haemoglobulinuria (PNH) in patients with resistance to Eculizumab due to complement C5 Polymorphisms | Coversin in Paroxysmal Nocturnal Haemoglobulinuria (PNH) in patients with resistance to Eculizumab due to complement C5 Polymorphisms - Coversin in PNH patients with resistance to Eculizumab | Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms. MedDRA version: 18.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Coversin Product Code: rVA576 INN or Proposed INN: Not applicable Other descriptive name: coversin | Volution Immuno Pharmaceuticals (UK) Ltd | NULL | Not Recruiting | Female: yes Male: yes | 6 | Phase 2 | Netherlands | ||
| 233 | NCT02605993 (ClinicalTrials.gov) | January 4, 2016 | 11/11/2015 | Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 2, Open-label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria;PNH | Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 26 | Phase 2 | Canada;France;Germany;Korea, Republic of;Spain;Taiwan;United Kingdom;Sweden |
| 234 | EUCTR2015-002674-20-DE (EUCTR) | 04/12/2015 | 11/08/2015 | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: ALXN1210 Product Code: ALXN1210 INN or Proposed INN: ALXN1210 | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 26 | Phase 2 | France;United States;Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Sweden;Korea, Republic of | ||
| 235 | EUCTR2015-002674-20-SE (EUCTR) | 30/11/2015 | 02/10/2015 | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 18.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: ALXN1210 | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | Germany;United Kingdom;Sweden;France;Taiwan;Canada;Finland;Spain;Russian Federation | ||
| 236 | EUCTR2015-002674-20-ES (EUCTR) | 27/11/2015 | 05/10/2015 | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 18.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: ALXN1210 | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Phase 2 | France;Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Sweden | ||
| 237 | NCT02598583 (ClinicalTrials.gov) | November 2015 | 2/11/2015 | Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | An Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria | PNH | Biological: ALXN1210 | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 13 | Phase 1/Phase 2 | Australia;Korea, Republic of |
| 238 | NCT02588833 (ClinicalTrials.gov) | November 2015 | 27/10/2015 | Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. APL-2 in PNH Subjects | A Phase Ib, Open Label, Multiple Ascending Dose, Pilot Study to Assess the Safety, Preliminary Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 20 | Phase 1 | Hong Kong;Malaysia;New Zealand;Thailand |
| 239 | EUCTR2015-002674-20-GB (EUCTR) | 28/10/2015 | 01/10/2015 | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: ALXN1210 Product Code: ALXN1210 INN or Proposed INN: ALXN1210 | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 26 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Korea, Republic of;Sweden | ||
| 240 | NCT02534909 (ClinicalTrials.gov) | September 9, 2015 | 7/8/2015 | Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria | An Open-label Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316, an Anti-C5 Monoclonal Antibody in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria PNH | Biological: LFG316;Drug: LNP023 | Novartis Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 10 | Phase 2 | Czechia;Japan;Lithuania;Czech Republic;Moldova, Republic of |
| 241 | EUCTR2014-005338-74-LT (EUCTR) | 07/07/2015 | 09/06/2015 | To find out whether LFG316 is able to reduce the destruction of red blood cells in patients with PNH | An open-label proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316, an anti-C5 monoclonal antibody in patients with paroxysmal nocturnal hemoglobinuria (PNH) - CLFG316X2201 | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: LFG316 INN or Proposed INN: No Other descriptive name: human monoclonal antibody directed against complement 5 protein Product Code: LFG316 INN or Proposed INN: No Other descriptive name: human monoclonal antibody directed against complement 5 protein Product Name: iptacopan Product Code: LNP023 Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2 | Czechia;Czech Republic;Lithuania;Japan | ||
| 242 | EUCTR2014-005338-74-CZ (EUCTR) | 17/06/2015 | 23/04/2015 | To find out whether LFG316 is able to reduce the destruction of red blood cells in patients with PNH | An open-label proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316, an anti-C5 monoclonal antibody in patients with paroxysmal nocturnal hemoglobinuria (PNH) - CLFG316X2201 | Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: LFG316 INN or Proposed INN: No Other descriptive name: human monoclonal antibody directed against complement 5 protein Product Code: LFG316 INN or Proposed INN: No Other descriptive name: human monoclonal antibody directed against complement 5 protein Product Name: iptacopan Product Code: LNP023 Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 Other descriptive name: LNP023 HYDROCHLORIDE SALT | Novartis Pharma Services AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Phase 2 | Czechia;Czech Republic;Lithuania;Japan | ||
| 243 | NCT02264639 (ClinicalTrials.gov) | February 23, 2015 | 8/10/2014 | A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects With PNH | An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH). | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 9 | Phase 1 | United States |
| 244 | NCT02352493 (ClinicalTrials.gov) | January 2015 | 23/1/2015 | A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH | A Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ALN-CC5;Drug: Sterile Normal Saline (0.9% NaCl) | Alnylam Pharmaceuticals | NULL | Completed | 18 Years | 45 Years | All | 62 | Phase 1/Phase 2 | Spain;United Kingdom |
| 245 | NCT02179359 (ClinicalTrials.gov) | September 2, 2014 | 27/6/2014 | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders | Sickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic Disorders | Drug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative Regimen | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 25 | N/A | United States |
| 246 | ChiCTR-TRC-14004219 | 2014-01-24 | 2014-01-24 | A prospective, randomized, open label, single-center trial of comparing intravenous iron sucrose versus iron dextran tablet in the treatment of iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria | A prospective, randomized, open label, single-center trial of comparing intravenous iron sucrose versus iron dextran tablet in the treatment of iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria | iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria | intravenous iron sucrose:intravenous iron sucrose;iron dextran tablet:Oral iron dextran tablet; | Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical Sciences | NULL | Recruiting | 18 | 65 | Both | intravenous iron sucrose:24;iron dextran tablet:24; | China | |
| 247 | ChiCTR-ONC-13003994 | 2014-01-01 | 2013-11-06 | Chemotherapy plus hematopoietic growth factors (DAG) for refractory paroxysmal nocturnal hemoglobinuria: diminishing PNH clone and stimulating hematopoisis | Chemotherapy plus hematopoietic growth factors (DAG) for refractory paroxysmal nocturnal hemoglobinuria: diminishing PNH clone and stimulating hematopoisis | paroxymal nocturnal hemoglobinuria | A1:DA chemotherapy regimen plus G-CSF ; | Tianjin Medical University General Hospital | NULL | Recruiting | 16 | 75 | Both | A1:50; | China | |
| 248 | NCT01760096 (ClinicalTrials.gov) | January 2013 | 1/1/2013 | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) | Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) | Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic | Drug: Levamisole+cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids | Institute of Hematology & Blood Diseases Hospital | NULL | Active, not recruiting | 18 Years | N/A | Both | 50 | Phase 2 | China |
| 249 | NCT01642979 (ClinicalTrials.gov) | July 2012 | 14/7/2012 | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria | Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic | Drug: Levamisole+cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids | Institute of Hematology & Blood Diseases Hospital | NULL | Active, not recruiting | 18 Years | N/A | Both | 120 | Phase 2 | China |
| 250 | NCT01529827 (ClinicalTrials.gov) | February 28, 2012 | 6/2/2012 | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies | A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation | Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation | Roswell Park Cancer Institute | NULL | Completed | 3 Years | 75 Years | All | 94 | Phase 2 | United States |
| 251 | NCT01335165 (ClinicalTrials.gov) | April 2011 | 12/4/2011 | Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ALXN1102;Drug: ALXN1103 | Alexion Pharmaceuticals | NULL | Terminated | 18 Years | N/A | Both | 10 | Phase 1 | United States;Czech Republic;Italy;Poland;United Kingdom |
| 252 | NCT00867932 (ClinicalTrials.gov) | October 2, 2009 | 23/3/2009 | Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH) | An Open-Label Multi-Center Study of Eculizumab in Children and Adolescents With a Diagnosis of Paroxysmal Nocturnal Hemoglobinuria | Hemoglobinuria, Paroxysmal | Drug: Eculizumab | Alexion Pharmaceuticals | NULL | Completed | 2 Years | 17 Years | All | 7 | Phase 4 | United States |
| 253 | NCT00856388 (ClinicalTrials.gov) | January 14, 2009 | 4/3/2009 | Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders | A Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body Irradiation | Accelerated Phase Chronic Myelogenous Leukemia;Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Eosinophilic Leukemia;Chronic Myelomonocytic Leukemia;Chronic Neutrophilic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;de Novo Myelodysplastic Syndromes;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Fanconi Anemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Noncontiguous Stage II Adult Burkitt Lymphoma;Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma;Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma;Noncontiguous Stage II Adult Lymphoblastic Lymphoma;Noncontiguous Stage II Grade 1 Follicular Lymphoma;Noncontiguous Stage II Grade 2 Follicular Lymphoma;Noncontiguous Stage II Grade 3 Follicular Lymphoma;Noncontiguous Stage II Mantle Cell Lymphoma;Noncontiguous Stage II Marginal Zone Lymphoma;Noncontiguous Stage II Small Lymphocytic Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Multiple Myeloma;Relapsing Chronic Myelogenous Leukemia;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Splenic Marginal Zone Lymphoma;Stage III Adult Diffuse Small Cleaved Cell Lymphoma;Stage III Adult Immunoblastic Large Cell Lymphoma;Stage III Adult Lymphoblastic Lymphoma;Stage III Grade 1 Follicular Lymphoma;Stage III Grade 2 Follicular Lymphoma;Stage III Grade 3 Follicular Lymphoma;Stage III Mantle Cell Lymphoma;Stage III Marginal Zone Lymphoma;Stage III Small Lymphocytic Lymphoma;Stage IV Adult Burkitt Lymphoma;Stage IV Adult Diffuse Small Cleaved Cell Lymphoma;Stage IV Adult Immunoblastic Large Cell Lymphoma;Stage IV Adult Lymphoblastic Lymphoma;Stage IV Grade 1 Follicular Lymphoma;Stage IV Grade 2 Follicular Lymphoma;Stage IV Grade 3 Follicular Lymphoma;Stage IV Mantle Cell Lymphoma;Stage IV Marginal Zone Lymphoma;Stage IV Small Lymphocytic Lymphoma;Waldenström Macroglobulinemia | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Procedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulin | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Completed | 3 Years | 75 Years | All | 62 | N/A | United States |
| 254 | NCT01194804 (ClinicalTrials.gov) | April 2008 | 30/8/2010 | E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol | Paroxysmal Nocturnal Hemoglobinuria | Drug: Eculizumab | Alexion Pharmaceuticals | CMIC Co, Ltd. Japan | Completed | 12 Years | N/A | All | 27 | Phase 2 | NULL |
| 255 | NCT01192399 (ClinicalTrials.gov) | November 2007 | 30/8/2010 | Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients | Paroxysmal Nocturnal Hemoglobinuria | Biological: Eculizumab | Alexion Pharmaceuticals | NULL | Completed | 12 Years | N/A | All | 29 | Phase 2 | NULL |
| 256 | NCT00438789 (ClinicalTrials.gov) | February 2007 | 20/2/2007 | The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol | Hemoglobinuria, Paroxysmal | Drug: eculizumab | Alexion Pharmaceuticals | NULL | Approved for marketing | 18 Years | N/A | Both | Phase 3 | United States | ||
| 257 | EUCTR2005-000043-28-ES (EUCTR) | 07/04/2006 | 21/10/2005 | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | Germany;United Kingdom;Ireland;Spain;Italy;Sweden | ||
| 258 | NCT00397813 (ClinicalTrials.gov) | January 2006 | 9/11/2006 | Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders | Low-Dose TBI Dose Escalation to Decrease Risks of Progression and Graft Rejection After Hematopoietic Cell Transplantation With Nonmyeloablative Conditioning as Treatment for Untreated Myelodysplastic Syndrome or Myeloproliferative Disorders - A Multi-Center Trial | Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Chronic Myelomonocytic Leukemia;de Novo Myelodysplastic Syndrome;Essential Thrombocythemia;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Polycythemia Vera;Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase;Primary Myelofibrosis;Refractory Anemia;Refractory Anemia With Excess Blasts;Refractory Anemia With Ring Sideroblasts;Refractory Cytopenia With Multilineage Dysplasia;Refractory Cytopenia With Multilineage Dysplasia and Ring Sideroblasts | Drug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Completed | 50 Years | 75 Years | All | 77 | Phase 2 | United States;Denmark;Germany;Italy |
| 259 | EUCTR2005-000043-28-IT (EUCTR) | 01/09/2005 | 06/02/2007 | A Phase III Open label extension study of Eculizumab in patients with transfusion dependent hemolytic paroxysmal nocturnal hemoglobinuria PNH who have participated in Triumph C04-001 , Shepherd C04-002 or X03-001 studies | A Phase III Open label extension study of Eculizumab in patients with transfusion dependent hemolytic paroxysmal nocturnal hemoglobinuria PNH who have participated in Triumph C04-001 , Shepherd C04-002 or X03-001 studies | paroxymal nocturnal hemoglobinuria MedDRA version: 6.1;Level: PT;Classification code 10034042 | Product Name: Eculizumab Product Code: h5G1.1-mAb Product Name: Eculizumab Product Code: h5G1.1-mAb | ALEXION PHARMACEUTICALS INC | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | Germany;United Kingdom;Spain;Ireland;Italy;Sweden | ||
| 260 | NCT00130000 (ClinicalTrials.gov) | August 9, 2005 | 11/8/2005 | Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria | SHEPHERD: Safety in Hemolytic PNH Patients Treated With Eculizumab: A Multi-Center Open-Label Research Design | Paroxysmal Hemoglobinuria, Nocturnal | Drug: Eculizumab | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 18 Years | N/A | All | 87 | Phase 3 | United States |
| 261 | NCT00143559 (ClinicalTrials.gov) | August 2005 | 1/9/2005 | Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies | Haploidentical Hematopoietic Stem Cell Transplantation Utilizing Partial T-Cell Depletion as Immunotherapy for Hematologic Malignancies | Leukemia;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic Syndrome;Paroxysmal Nocturnal Hemoglobinuria;Hodgkin's Lymphoma;Non-Hodgkin Lymphoma | Drug: Systematic chemotherapy and antibodies;Procedure: Allogeneic stem cell transplantation;Device: Miltenyi CliniMACS | St. Jude Children's Research Hospital | NULL | Completed | 2 Years | 21 Years | Both | 17 | Phase 2 | United States |
| 262 | EUCTR2005-000043-28-DE (EUCTR) | 06/07/2005 | 08/04/2005 | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05-001 Extension | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05-001 Extension | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 MedDRA version: 9.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria | Product Name: eculizumab Product Code: L04AA25 INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | United Kingdom;Germany;Spain;Ireland;Italy;Sweden | ||
| 263 | EUCTR2005-000043-28-IE (EUCTR) | 27/05/2005 | 06/04/2005 | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | Spain;Ireland;Germany;Italy;United Kingdom;Sweden | ||
| 264 | EUCTR2004-002795-42-ES (EUCTR) | 24/05/2005 | 22/02/2006 | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 85 | Phase 3 | Spain;Ireland;Germany;Italy;United Kingdom;Sweden | ||
| 265 | EUCTR2005-000043-28-SE (EUCTR) | 24/05/2005 | 07/04/2005 | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05-001 Extension | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05-001 Extension | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 MedDRA version: 9.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | Germany;United Kingdom;Spain;Ireland;Italy;Sweden | ||
| 266 | NCT00122317 (ClinicalTrials.gov) | May 2005 | 20/7/2005 | Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) | Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH | Paroxysmal Hemoglobinuria, Nocturnal | Drug: eculizumab | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 187 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;United Kingdom |
| 267 | EUCTR2004-002795-42-IT (EUCTR) | 29/04/2005 | 06/07/2005 | SHEPHERD: Safety in hemolytic PNH patients treated with Eculizumab: a multi-center open label research design study | SHEPHERD: Safety in hemolytic PNH patients treated with Eculizumab: a multi-center open label research design study | paroxysmal nocturnal hemoglobinuria MedDRA version: 6.1;Level: PT;Classification code 10034042 | Product Name: Eculizumab | ALEXION PHARMACEUTICALS INC | NULL | Not Recruiting | Female: yes Male: yes | 85 | United Kingdom;Germany;Spain;Ireland;Italy;Sweden | |||
| 268 | EUCTR2004-002795-42-GB (EUCTR) | 20/04/2005 | 23/02/2005 | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 85 | Phase 3 | Spain;Ireland;Germany;Italy;United Kingdom;Sweden | ||
| 269 | EUCTR2005-000043-28-GB (EUCTR) | 19/04/2005 | 16/03/2005 | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 170 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Spain;Ireland;Germany;Italy;United Kingdom;Sweden | ||
| 270 | EUCTR2004-002795-42-DE (EUCTR) | 24/03/2005 | 23/12/2004 | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 85 | United Kingdom;Germany;Spain;Ireland;Italy;Sweden | |||
| 271 | EUCTR2004-002795-42-SE (EUCTR) | 16/02/2005 | 09/12/2004 | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 85 | United Kingdom;Germany;Spain;Ireland;Italy;Sweden | |||
| 272 | EUCTR2004-002795-42-IE (EUCTR) | 03/02/2005 | 09/12/2004 | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 85 | Phase 3 | Spain;Ireland;Germany;Italy;United Kingdom;Sweden | ||
| 273 | EUCTR2004-000646-20-GB (EUCTR) | 02/12/2004 | 14/02/2005 | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 75 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): | Ireland;Italy;United Kingdom;Sweden | ||
| 274 | NCT00122304 (ClinicalTrials.gov) | December 2004 | 19/7/2005 | Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab | Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study | Hemoglobinuria, Paroxysmal | Drug: eculizumab | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | Both | 85 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom |
| 275 | NCT00098280 (ClinicalTrials.gov) | December 2004 | 3/12/2004 | Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Hemoglobinuria, Paroxysmal | Drug: Eculizumab | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | N/A | N/A | Both | 75 | Phase 3 | United States |
| 276 | EUCTR2004-000646-20-IT (EUCTR) | 01/11/2004 | 23/12/2005 | TRIUMPH A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | TRIUMPH A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | paaroxysmal nocturnal hemoglobinuria MedDRA version: 6.1;Level: PT;Classification code 10034042 | Product Name: Eculizumab INN or Proposed INN: VARI | ALEXION PHARMACEUTICALS INC | NULL | Not Recruiting | Female: yes Male: yes | 75 | Ireland;Italy;Sweden | |||
| 277 | NCT00112983 (ClinicalTrials.gov) | November 2004 | 2/6/2005 | Eculizumab in Treating Patients With Paroxysmal Nocturnal Hemoglobinuria | TRIUMPH: A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Leukemia | Biological: eculizumab | Jonsson Comprehensive Cancer Center | NULL | Completed | 18 Years | N/A | Both | Phase 3 | United States | |
| 278 | EUCTR2004-000646-20-IE (EUCTR) | 26/10/2004 | 16/08/2004 | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 75 | Phase 3 | Ireland;Italy;United Kingdom;Sweden | ||
| 279 | NCT00122330 (ClinicalTrials.gov) | October 2004 | 18/7/2005 | Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients | Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients | Hemoglobinuria, Paroxysmal | Drug: eculizumab | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | Both | 75 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Sweden;Switzerland;United Kingdom |
| 280 | EUCTR2004-000646-20-SE (EUCTR) | 21/09/2004 | 19/07/2004 | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0;Level: PT;Classification code 10034042 | Product Name: eculizumab INN or Proposed INN: eculizumab Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 75 | Ireland;Italy;Sweden | |||
| 281 | NCT00145613 (ClinicalTrials.gov) | June 2003 | 1/9/2005 | Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies | Haploidentical Stem Cell Transplantation Utilizing T-Cell Depletion as Therapy for Patients With Refractory Hematological Malignancies | Acute Lymphoblastic Leukemia (ALL);Acute Myeloid Leukemia (AML);Secondary AML;Myelodysplastic Syndrome (MDS);Secondary MDS;Chronic Myeloid Leukemia;Juvenile Myelomonocytic Leukemia (JMML);Paroxysmal Nocturnal Hemoglobinuria (PNH);Lymphoma, Non-Hodgkin;Hodgkin Disease | Procedure: Stem Cell Transplantation;Device: Miltenyi Biotec CliniMACS;Drug: Systemic chemotherapy and antibodies | St. Jude Children's Research Hospital | NULL | Completed | 2 Years | 21 Years | Both | 25 | Phase 2 | United States |
| 282 | NCT00587054 (ClinicalTrials.gov) | June 2001 | 21/12/2007 | Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders | Phase II Trial of Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Treatment of Adult Patients (>18 Years) With Lymphohematopoietic Disorders | Allogeneic Stem Cell Transplant;Leukemia;Non-Hodgkins;Lymphoblastic Lymphoma;Myelodysplastic Syndrome;Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant | Memorial Sloan Kettering Cancer Center | National Cancer Institute (NCI) | Completed | 18 Years | 55 Years | All | 129 | Phase 2 | United States |
| 283 | NCT00012376 (ClinicalTrials.gov) | March 2001 | 3/3/2001 | Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer | Dose Finding Study of Bryostatin-1 and GM-CSF in Refractory Myeloid Malignancies | Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Blastic Phase Chronic Myelogenous Leukemia;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Recurrent Adult Acute Myeloid Leukemia;Refractory Anemia;Refractory Anemia With Ringed Sideroblasts;Relapsing Chronic Myelogenous Leukemia;Thrombocytopenia;Untreated Adult Acute Myeloid Leukemia | Drug: bryostatin 1;Biological: sargramostim;Other: laboratory biomarker analysis;Other: pharmacological study | National Cancer Institute (NCI) | NULL | Completed | 18 Years | N/A | Both | 35 | Phase 1 | United States |
| 284 | NCT00004143 (ClinicalTrials.gov) | September 1999 | 10/12/1999 | Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes | Allogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure Syndromes | Sickle Cell Anemia;Severe Aplastic Anemia;Paroxysmal Nocturnal Hemoglobinuria (PNH);Pure Red Cell Aplasia | Drug: Campath, Chemo and/or TBI Allo SCT | David Rizzieri, MD | NULL | Completed | 18 Years | N/A | All | 2 | Phase 2 | United States |
| 285 | NCT00004464 (ClinicalTrials.gov) | February 1996 | 18/10/1999 | Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria | High Dose Cyclophosphamide for the Treatment of Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria | Aplastic Anemia;Paroxysmal Hemoglobinuria, Nocturnal | Drug: cyclophosphamide;Drug: filgrastim | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | NULL | Completed | N/A | 70 Years | All | 69 | Phase 2 | United States |
| 286 | EUCTR2009-010402-11-Outside-EU/EEA (EUCTR) | 30/04/2012 | Not Applicable | AN OPEN-LABEL MULTI-CENTER STUDY OF ECULIZUMAB IN CHILDREN AND ADOLESCENTS WITH A DIAGNOSIS OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA MedDRA version: 14.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Trade Name: SOLIRIS INN or Proposed INN: ECULIZUMAB Other descriptive name: Anti-C5 antibody | ALEXION PHARMACEUTICALS, INC. | NULL | NA | Female: yes Male: yes | 6 | United States | ||||
| 287 | EUCTR2020-001350-21-NL (EUCTR) | 11/07/2022 | A single arm study to evaluate the safety and biological activity of pegcetacoplan in patients 12- 17 years old with paroxysmal nocturnal hemoglobinuria | An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Aspaveli Product Name: pegcetacoplan Product Code: pegcetacoplan INN or Proposed INN: Pegcetacoplan Other descriptive name: pegcetacoplan | Apellis Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 12 | Phase 2 | Serbia;United States;Czechia;Thailand;Spain;Ireland;United Kingdom;Italy;France;Czech Republic;Hungary;Malaysia;Poland;Romania;Australia;Netherlands;Germany | |||
| 288 | EUCTR2020-000597-26-SE (EUCTR) | 02/06/2020 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors. | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F.Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 250 | Phase 3 | United States;Portugal;Philippines;Taiwan;Hong Kong;Estonia;Greece;Thailand;Spain;Ireland;Israel;Colombia;Italy;France;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Brazil;Belgium;Poland;Singapore;Romania;Germany;Japan;Sweden | |||
| 289 | EUCTR2017-002820-26-FR (EUCTR) | 12/01/2018 | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: ALXN1210 Other descriptive name: ALXN1210 | Alexion Pharmaceuticals Incorporated | NULL | NA | Female: yes Male: yes | 12 | Phase 3 | United States;France;Netherlands | |||
| 290 | EUCTR2019-004931-21-IE (EUCTR) | 13/01/2021 | A Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously Treated with Complement Inhibitors | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Portugal;Philippines;Estonia;Taiwan;Hong Kong;Greece;Thailand;Spain;Ireland;Israel;Colombia;Italy;France;Malaysia;Australia;Peru;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;Austria;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Romania;Germany;Japan;Sweden | |||
| 291 | EUCTR2020-001350-21-CZ (EUCTR) | 08/12/2020 | A single arm study to evaluate the safety and biological activity of pegcetacoplan in patients 12- 17 years old with paroxysmal nocturnal hemoglobinuria | An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: pegcetacoplan Product Code: pegcetacoplan INN or Proposed INN: Pegcetacoplan Other descriptive name: pegcetacoplan | Apellis Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 12 | Phase 2 | Serbia;United States;Czechia;Spain;Thailand;Ireland;United Kingdom;Italy;France;Czech Republic;Hungary;Malaysia;Poland;Romania;Australia;Germany;Netherlands | |||
| 292 | EUCTR2019-001106-23-DE (EUCTR) | 30/09/2019 | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Aspaveli Product Name: Pegcetacoplan (APL-2) Product Code: Pegcetacoplan (APL-2) INN or Proposed INN: pegcetacoplan Other descriptive name: Pegcetacoplan (APL-2) | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | United States;Serbia;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Malaysia;Australia;Bulgaria;Germany;Japan;Korea, Republic of |