62. 発作性夜間ヘモグロビン尿症 Paroxysmal nocturnal hemoglobinuria Clinical trials / Disease details
臨床試験数 : 271 / 薬物数 : 163 - (DrugBank : 49) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 106
Showing 1 to 10 of 271 diseases
| 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 | NCT05133531 (ClinicalTrials.gov) | July 31, 2022 | 12/11/2021 | 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 Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combina ... | 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 A Randomized, Open-Label, Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozeli ... | Paroxysmal Nocturnal Hemoglobinuria | Drug: Ravulizumab;Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 124 | Phase 3 | United States |
| 2 | NCT05274633 (ClinicalTrials.gov) | March 31, 2022 | 2/3/2022 | Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria | Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria a Multicenter Observational Retrospective and Prospective Cohort Study. Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuriaa Multicent ... | Paroxysmal Nocturnal Hemoglobinuria | Drug: Ravulizumab | Alexion Pharmaceuticals | NULL | Not yet recruiting | 18 Years | N/A | All | 120 | NULL | |
| 3 | 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) Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria(PNH) Participan ... | 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) A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizum ... | 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] Patients with Paroxysmal Nocturnal HemoglobinuriaWho Have Clinically Evident Extravascular Hemolysis ... | 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 Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: D ... | 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 United States;Czechia;Taiwan;Greece;Thailand;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;Franc ... | ||
| 4 | NCT04965597 (ClinicalTrials.gov) | March 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) Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of ... | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Mar ... | 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 Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Ap ... | 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 Drug: Treosulfan;Drug: FludarabinePhosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T ... | Fred Hutchinson Cancer Research Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow ... | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
| 5 | 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 Study of ARO-C3 in Adult Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuriaand ... | 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 A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Ph ... | Paroxysmal Nocturnal Hemoglobinuria;C3 Glomerulopathy;IgA Nephropathy | Drug: ARO-C3;Drug: Placebo | Arrowhead Pharmaceuticals | NULL | Recruiting | 18 Years | 70 Years | All | 62 | Phase 1 | New Zealand |
| 6 | 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. An open label, multicenter roll-over extension program (REP) to characterize the long-term safety an ... | 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] Paroxysmal Nocturnal Hemoglobinuria(PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042 ... | 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 Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LN ... | 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 United States;Taiwan;Spain;Lithuania;Italy;France;Brazil;Malaysia;Singapore;Netherlands;China;Japan; ... | ||
| 7 | 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. An open label, multicenter roll-over extension program (REP) to characterize the long-term safety an ... | Paroxysmal Nocturnal Hemoglobinuria | Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatincapsule Route of administration ... | Suzuki Kazuyuki | NULL | Recruiting | >= 18age old | Not applicable | Both | 19 | Phase 3 | Italy;Republic of Korea;Taiwan;Japan |
| 8 | 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 A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and To ... | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: BCX9930;Drug: Eculizumab;Drug: Ravulizumab | BioCryst Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 81 | Phase 2 | United States;France;Hungary;Spain;Taiwan;United Kingdom |
| 9 | 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 A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Ef ... | 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] Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term ... | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX993 ... | 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 Serbia;United States;Spain;Turkey;Lithuania;Azerbaijan;European Union;Canada;Argentina;Brazil;Albani ... | ||
| 10 | 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 A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and To ... | 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] Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term ... | 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 Trade Name: ULTOMIRIS- 300 MG - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCIN ... | 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 United States;Taiwan;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada ... |