Overview

Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Status:
Enrolling by invitation
Trial end date:
2024-01-28
Target enrollment:
Participant gender:
Summary
The primary objective of the study is to evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP) The secondary objectives of the study are: - To evaluate the effect of the combination treatment on the following parameters of intravascular hemolysis: lactate dehydrogenase (LDH) control, breakthrough hemolysis, and inhibition of total complement hemolysis activity (CH50) - To evaluate the effect of the combination treatment on hemoglobin levels - To evaluate the effect of the combination treatment on red blood cell (RBC) transfusion requirements - To evaluate the effect of the combination treatment on clinical outcome assessments (COAs) measuring fatigue and health related quality of life - To assess the concentrations of total pozelimab in serum and total complement component (C) 5 and cemdisiran in plasma - To assess immunogenicity to pozelimab and cemdisiran - To evaluate the long-term safety and efficacy of pozelimab and cemdisiran in an optional open-label extension period (OLEP) - To assess safety after treatment intensification with pozelimab and cemdisiran
Phase:
Phase 2
Details
Lead Sponsor:
Regeneron Pharmaceuticals