OncoMatch/Clinical Trials/NCT05744921
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 Works
Is NCT05744921 recruiting? Yes, currently enrolling (May 2026). This Phase 3 trial studies multiple treatments including Pozelimab and Cemdisiran for paroxysmal nocturnal hemoglobinuria.
Treatment: Pozelimab · Cemdisiran — This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section. This study is looking at several other research questions, including: * How effective is the pozelimab + cemdisiran combination? * What side effects may happen from taking the study drugs? * How much of each study drug is in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)
Check if I qualifyExtracted eligibility criteria
Biomarker criteria
Required: C5 polymorphism rendering refractory to eculizumab or ravulizumab (e.g., p.Arg885His, p.Arg885Cys)
Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol
Prior therapy
Must have received: eculizumab or ravulizumab (eculizumab, ravulizumab) — parent study completion (for parent study cohort)
Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021[NCT05133531]), including the post-Open-label treatment period (OLTP) transition period, if applicable.
Cannot have received: complement inhibitor
Exception: except for prior eculizumab or ravulizumab which are not exclusionary
Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary
Structured fields extracted by AI. May contain errors — verify against the official protocol.
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