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 : 292 / 薬物数 : 151 - (DrugBank : 49) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 108
Showing 1 to 10 of 292 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 | 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) A Phase II, Open Label, Multi Dose Study of NM8074 in Soliris-Treated Subjects With Paroxysmal Noctu ... | 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 Study of Efficacy and Safety of NM8074 in Adult PNHPatients Who Are Naive to Complement Inhibitor Th ... | 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) A Phase II, Open-Label Study of NM8074 in Soliris®-Treated Patients With Paroxysmal Nocturnal Hemogl ... | 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. This is a Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable ... | 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 A Multicenter, Single Arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Ipt ... | 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 A Study to Examine the Long-term Safety, Tolerability, and Effectiveness of Pozelimab and Cemdisiran ... | 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 An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozeli ... | 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 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. A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on ... | 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] Patients with Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code ... | 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 | 95 | Phase 3 | United States;Czechia;Greece;Thailand;Spain;Israel;United Kingdom;Italy;France;Canada;Brazil;Malaysia;Poland;Japan;Korea, Republic of United States;Czechia;Greece;Thailand;Spain;Israel;United Kingdom;Italy;France;Canada;Brazil;Malaysi ... | ||
| 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 An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects With Paroxys ... | 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 A study to examine the long-term safety, tolerability, and effectiveness of Pozelimab and Cemdisiran ... | 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 An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozeli ... | 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] Paroxysmal nocturnal hemoglobinuria(PNH) MedDRA version: 21.1;Level: LLT;Classification code 1005562 ... | 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 Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran P ... | 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 United States;Philippines;Taiwan;Greece;Thailand;Spain;Colombia;Italy;India;France;Jordan;Malaysia;P ... | ||
| 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 Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ra ... | 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 A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SA ... | 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] Paroxysmal nocturnal hemoglobinuria(PNH) MedDRA version: 21.1;Level: LLT;Classification code 1005562 ... | 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 Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran P ... | 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 Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;M ... | ||
| 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) An Open-label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic ... | Paroxysmal Nocturnal Hemoglobinuria | Drug: KP104 | Kira Pharmacenticals (US), LLC. | NULL | Recruiting | 18 Years | N/A | All | 35 | Phase 2 | China |