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Medical Condition
paroxysmal nocturnal hemoglobinuria pnh
Total Trials
2
Recruiting Now
2
Trial Phases
Phase 2, Phase 1, Phase 2
NCT03520647 Phase 2
Recruiting
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Enrollment
56 pts
Location
United States
Sponsor
National Heart, Lung, and Bloo...
NCT07187401 Phase 1, Phase 2
Recruiting
A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
Enrollment
24 pts
Location
Canada, South Korea,...
Sponsor
Regeneron Pharmaceuticals