62. Paroxysmal nocturnal hemoglobinuria
202 clinical trials, 120 drugs (DrugBank: 25 drugs), 15 drug target genes, 90 drug target pathways
Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT04558918 (ClinicalTrials.gov) | December 15, 2020 | 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 | Not yet recruiting | 18 Years | N/A | All | 91 | Phase 3 | NULL |
| 2 | 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: 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;Italy;United Kingdom;France;Hungary;Canada;Brazil;Netherlands;Germany;Japan;Korea, Republic of | ||
| 3 | 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 | Authorised-recruitment may be ongoing or finished | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 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 |
| 6 | 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 | ||
| 7 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 85 | United Kingdom;Germany;Ireland;Spain;Italy;Sweden | |||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 | |||
| 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 |
| 11 | 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 | |||
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | 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 | |||
| 15 | 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 |