Regeneron Pharmaceuticals: Hemoglobinuria, Paroxysmal trials

The registry lists 10 Hemoglobinuria, Paroxysmal studies with Regeneron Pharmaceuticals as lead sponsor. 3 are open to participants now and 5 have results posted.

Phase 1 1Phase 2 4Phase 3 5No phase 1(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• 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 InhibitorRecruitingNCT07187401Phase 1/2
• A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working EfficientlyRecruitingNCT07154745Phase 3
• A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it WorksRecruitingNCT05744921Phase 3
• Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal HemoglobinuriaTerminatedNCT05131204Phase 3Results posted
• Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab TherapyCompletedNCT04888507Phase 2Results posted
• Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab MonotherapyCompletedNCT04811716Phase 2Results posted
• REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.TerminatedNCT04162470Phase 3Results posted
• Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)CompletedNCT03946748Phase 2Results posted
• Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal HemoglobinuriaNo longer availableNCT06028594
• A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor TreatmentActive, not recruitingNCT05133531Phase 3