drugset / Trial / 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

NCT05744921

Phase 3 Recruiting 202 enrolled Regeneron Pharmaceuticals
Non-randomizedParallel-groupOpen-labelTreatment

Summary

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)

Timeline

Start
2023-03-07
Primary completion
2029-02-26
Completion
2029-02-26

Drugs

EvaluationDrugModalityDoseRoute
Subject Cemdisiran Small interfering RNA (siRNA)
Subject Pozelimab Monoclonal antibody Intravenous