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 145 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 | 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 Eculizumabor Rav ... | 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 ECULIZUMABAND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAF ... | 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 100556 ... | 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 ... | ||
| 2 | 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 Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumabor Rav ... | 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, Eculizumaband Ravulizumab Controlled Study to Evaluate the Efficacy and Sa ... | 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 |
| 3 | 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 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 ... | 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 Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX993 ... | 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 United States;Taiwan;Slovakia;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Uni ... | ||
| 4 | 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 Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumabor Rav ... | 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 ECULIZUMABAND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAF ... | 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 100556 ... | 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;Brazil;Poland;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;M ... | ||
| 5 | 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 Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumabor Rav ... | 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 A RANDOMIZED, OPEN-LABEL ECULIZUMABAND RAVULIZUMAB CONTROLLED, NON-INFERIORITY STUDY TO EVALUATE THE ... | 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 100556 ... | 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 | 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 Portugal;United States;Philippines;Taiwan;Greece;Spain;Thailand;Turkey;Colombia;United Kingdom;Italy ... | ||
| 6 | 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 Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumabor Rav ... | 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 ECULIZUMABAND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAF ... | 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 100556 ... | 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 | 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 Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;M ... | ||
| 7 | 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 Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumabor Rav ... | 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 ECULIZUMABAND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAF ... | 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 100556 ... | 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;Germany;Netherlands;Japan;Korea, Republic of Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;M ... | ||
| 8 | 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) Participa ... | 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 (Eculizumabor Ravulizuma ... | 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 Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysi ... | 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 ... | ||
| 9 | 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 Study to Evaluate the Efficacy and Safety of Crovalimab versus Eculizumabin Adult and Adolescent P ... | 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 AN ... | 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] Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 100556 ... | 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 Product Name: Crovalimab Product Code: RO7112689/F03-01 INN or Proposed INN: CROVALIMAB Trade Name: Sol ... | 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 Portugal;United States;Philippines;Hong Kong;Taiwan;Estonia;Greece;Spain;Thailand;Ireland;Israel;Col ... | ||
| 10 | 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 | Active, not recruiting | 18 Years | N/A | All | 81 | Phase 2 | France;Hungary;Italy;Spain;United Kingdom;Taiwan;United States |