Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

• Status: Not yet recruiting
• Trial end date: 2026-09-18

Summary

The primary objective of the study is:

To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue their eculizumab or ravulizumab therapy

The secondary objectives of the study are to:

• Evaluate the effect of pozelimab and cemdisiran combination treatment versus anti-C5 standard-of-care treatment (eculizumab or ravulizumab) on the following:

• Transfusion requirements and transfusion parameters

• Measures of hemolysis: LDH control, breakthrough hemolysis, and inhibition of CH50

• Hemoglobin levels

• Fatigue as assessed by Clinical Outcome Assessments (COAs)

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

• Safety and tolerability

• To assess the concentrations of total pozelimab and either total eculizumab or total ravulizumab in serum and total cemdisiran and total C5 protein in plasma

• To assess the immunogenicity of pozelimab and cemdisiran

• Phase: Phase 3

Details

• Lead Sponsor: Regeneron Pharmaceuticals
• Treatments: Eculizumab; Ravulizumab