Long-Term Safety and Effectiveness Study of Pozelimab and Cemdisiran Combination Therapy in Adults with Paroxysmal Nocturnal Hemoglobinuria

What this Trial Is About

Researchers are studying an experimental treatment combining two drugs, pozelimab and cemdisiran, to evaluate their long-term safety and effectiveness for adults with paroxysmal nocturnal hemoglobinuria PNH. This Phase 3 study aims to answer key questions about how well this combination works, potential side effects, drug levels in the blood, and whether the body develops antibodies against the drugs that could affect treatment. Participants include those who have completed treatment in a related parent study and those with a specific C5 genetic variation making them resistant to other treatments. The study involves administering the study drugs per protocol, including a loading dose of pozelimab given intravenously on Day 1 for some participants. The study is open-label and non-randomized, with two groups based on prior treatment history or genetic markers. During the study, participants will attend clinic visits to receive treatments and undergo various assessments such as blood tests to monitor hemolysis and hemoglobin levels, measure drug concentrations, and check for antibodies. Researchers will track serious and other adverse events, treatment discontinuation, and changes in quality of life. The study lasts up to around 108 weeks, with ongoing safety and effectiveness monitoring throughout this period.

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