A Study to Examine the Long-term Safety, Tolerability, and Effectiveness of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

• Status: Not yet recruiting
• Trial end date: 2028-03-27

Summary

The primary objective of the study is to describe the long-term safety, tolerability, and efficacy of pozelimab and cemdisiran combination therapy in patients with PNH

The secondary objectives of the study are to describe the long-term effect of the combination of pozelimab and cemdisiran on:

• Measures of intravascular hemolysis

• Transfusion parameters

• Hemoglobin levels

• Fatigue as assessed by a Patient Reported Outcome (PRO)

• Physical Function (PF) as assessed by a PRO

• Change in Global Health Status (GHS) as assessed by a PRO

• Complement activation

• Concentrations of total pozelimab in serum and cemdisiran and total complement component 5 (C5) protein in plasma

• Immunogenicity of pozelimab & cemdisiran

• Phase: Phase 3

Details

• Lead Sponsor: Regeneron Pharmaceuticals