62. Paroxysmal nocturnal hemoglobinuria Clinical trials / Disease details
Clinical trials : 271 / Drugs : 163 - (DrugBank : 49) / Drug target genes : 22 - Drug target pathways : 106
| 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 | EUCTR2018-003956-19-FI (EUCTR) | 13/02/2019 | 28/01/2019 | Study of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3, Multicenter, Open-Label, Single-Arm Study to Confirm the Safety and Efficacy of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria - Phase 3 Study in Treatment-Naive Paroxysmal Nocturnal Hemoglobinuria Patients | 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 Name: Zilucoplan Product Code: RA101495 INN or Proposed INN: ZILUCOPLAN Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;Hong Kong;Taiwan;Finland;Spain;Turkey;Russian Federation;Italy;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Malaysia;Belgium;Brazil;Australia;Georgia;Germany;New Zealand;Korea, Republic of | ||
| 2 | EUCTR2016-003522-16-FI (EUCTR) | 20/06/2017 | 27/12/2016 | Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH) | A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | ||
| 3 | EUCTR2016-003522-16-DK (EUCTR) | 01/05/2017 | 14/02/2017 | Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A PHASE 2 MULTICENTER, OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Canada;Finland;Australia;Denmark;Germany;New Zealand;United Kingdom | ||
| 4 | EUCTR2016-003523-34-DK (EUCTR) | 01/05/2017 | 14/02/2017 | Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | United States;Hungary;Canada;Finland;Australia;Denmark;Germany;United Kingdom;New Zealand | ||
| 5 | EUCTR2016-003523-34-FI (EUCTR) | 06/04/2017 | 27/12/2016 | Extension Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | 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: Zilucoplan Product Code: RA101495 INN or Proposed INN: Zilucoplan Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 palmitoylated linker | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | United States;Hungary;Finland;Denmark;Australia;Germany;United Kingdom;New Zealand | ||
| 6 | EUCTR2016-003523-34-HU (EUCTR) | 17/03/2017 | 31/01/2017 | Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | United States;Hungary;Finland;Denmark;Australia;Germany;United Kingdom;New Zealand | ||
| 7 | EUCTR2016-003522-16-HU (EUCTR) | 17/03/2017 | 31/01/2017 | Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | ||
| 8 | EUCTR2016-003523-34-GB (EUCTR) | 20/02/2017 | 26/09/2017 | Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | ||
| 9 | EUCTR2016-003522-16-GB (EUCTR) | 15/02/2017 | 03/01/2017 | Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH) | A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 | Ra Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom |