Study design of A phase 3, open-label trial for pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria with inadequate control of intravascular hemolysis
Open-label trial assesses combination therapy for paroxysmal nocturnal hemoglobinuria, suggesting improved control of intravascular hemolysis with monoclonal antibody treatment.
Key Points
The trial aims to evaluate the effectiveness of the combination therapy for paroxysmal nocturnal hemoglobinuria.
Primary endpoint is the percentage change in lactate dehydrogenase from baseline to week 28.
Observational approach includes transitioning patients from their current C5 inhibitor therapy to the combination treatment regimen.
The findings may highlight the potential benefits of new treatments for managing hemolysis in patients with PNH.