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Pozelimab and Cemdisiran Combination Therapy in PNH
NCT05133531Phase 3
Key facts
- Status
- Active, not recruiting
- Start
- Aug 2022
- Primary completion
- Oct 2026
- Countries
- 24
- Sponsor
- Regeneron Pharmaceuticals
- Modality
- Antisense DNA, Recombinant Proteins and Enzymes, Conjugated siRNA
- Drugs
- pozelimabcemdisiran
- Conditions
- paroxysmal nocturnal hemoglobinuria (PNH)
01
Overview
Phase 3, randomised, open-label trial of pozelimab and cemdisiran in paroxysmal nocturnal hemoglobinuria (PNH) (2 cohorts), sponsored by Regeneron Pharmaceuticals. Primary endpoints: Percent change in lactate dehydrogenase at 26 weeks, Transfusion avoidance at 26 weeks and 1 more. Started Aug 2022; status: active, not recruiting.
02
Design
2 arms- Study design
- RandomizedOpen-label
Cohort A
Randomized 1:1 to Pozelimab + Cemdisiran or Ravulizumab
Cohort B
Randomized 1:1 to Pozelimab + Cemdisiran or Eculizumab
03
Who it enrols
Patients with confirmed PNH, active disease (LDH >= 2x ULN), weight >= 40 kg, and no recent complement inhibitor therapy.
04
Endpoints
Primary
Percent change in lactate dehydrogenase
Transfusion avoidance
Adequate control of hemolysis
Secondary
Maintenance of adequate control of hemolysis
Breakthrough hemolysis
Hemoglobin stabilization
Normalization of LDH
Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy-Fatigue Scale
Change in physical function scores on the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire
Change in global health status/QoL scale score on the EORTC-QLC-C30
Rate of RBC transfused
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