Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Co

• Status: Not yet recruiting
• Trial end date: 2027-07-16

Summary

The primary objective of the study is:

To evaluate the effect on hemolysis and red blood cells (RBC) transfusions over a 24-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with active Paroxysmal Nocturnal Hemoglobinuria (PNH) who are complement inhibitor treatment-naive or have not recently received complement inhibitor therapy

The secondary objectives of the study are to:

• Evaluate the effect of pozelimab and cemdisiran combination treatment versus ravulizumab treatment on the following:

• Measures of hemolysis

• Transfusion parameters

• Hemoglobin levels

• Fatigue as assessed by Clinical Outcome Assessments (COAs)

• Health-related quality of life (HRQoL) as assessed by COAs

• Safety and tolerability

• Complement activation

• To assess the concentrations of total pozelimab and total ravulizumab in serum and total cemdisiran and total complement factor 5 (C5) protein in plasma

• To assess the immunogenicity of pozelimab and cemdisiran

• Phase: Phase 3

Details

• Lead Sponsor: Regeneron Pharmaceuticals
• Treatments: Ravulizumab