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 | EUCTR2020-005005-17-HU (EUCTR) | 07/05/2021 | 05/03/2021 | Pozelimab and Cemdisiran Combination Treatment in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | Paroxysmal Nocturnal Hemoglobinuria 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: 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 | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | Taiwan;Hong Kong;Hungary;Malaysia;United Kingdom;Korea, Republic of | ||
| 2 | EUCTR2016-002943-40-ES (EUCTR) | 21/11/2016 | 30/09/2016 | A Clinical Study with Investigational Drug, ALN-CC5, in patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab | A Phase 2, Open-label, Single Dose, Study of Subcutaneously Administered ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALN-CC5 INN or Proposed INN: ALN-62643 | Alnylam Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Taiwan;Finland;Spain;Turkey;Austria;Russian Federation;Israel;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Denmark;Bulgaria;Norway;Netherlands;Germany;Korea, Republic of;Sweden | ||
| 3 | NCT02352493 (ClinicalTrials.gov) | January 2015 | 23/1/2015 | A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH | A Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ALN-CC5;Drug: Sterile Normal Saline (0.9% NaCl) | Alnylam Pharmaceuticals | NULL | Completed | 18 Years | 45 Years | All | 62 | Phase 1/Phase 2 | Spain;United Kingdom |