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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 |
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 |
| 11 | 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 |