Study of Pozelimab and Cemdisiran Combination Therapy for Adults with Paroxysmal Nocturnal Hemoglobinuria Not Controlled by Current C5 Inhibitor Treatments

What this Trial Is About

Researchers are studying a combination of two experimental drugs, pozelimab and cemdisiran, to find better treatment options for adults with Paroxysmal Nocturnal Hemoglobinuria PNH whose condition has not improved despite current treatments with complement component 5 C5 inhibitors like eculizumab, ravulizumab, or crovalimab. This Phase 3 study aims to evaluate how well the drug combination lowers hemolysis and to monitor potential side effects, drug levels in the blood, and antibody responses against the study drugs. The treatment involves two periods a 28-week initial Treatment Period TP and a 52-week Extension Period EP. Participants receive the pozelimab and cemdisiran combination therapy according to the study protocol during these times. The study is designed as a single-group trial where all participants receive this combination treatment. Participants will be involved in regular clinic or remote visits throughout the study to monitor their health and treatment effects. Assessments include measuring lactate dehydrogenase LDH levels to track hemolysis, monitoring hemoglobin levels, fatigue, and transfusion needs, as well as checking for adverse events and antibody development against the drugs. The study also measures drug concentrations and complement activity. The total participation duration spans up to 80 weeks, including both treatment periods.

A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently