Phase 12 Study of ALXN2350 Gene Therapy for Adults With BAG3 Mutation Associated Dilated Cardiomyopathy

What this Trial Is About

Researchers are conducting a Phase 12 open-label study to evaluate the safety, tolerability, and efficacy of ALXN2350, a gene therapy, in adults with BAG3 mutation associated dilated cardiomyopathy DCM. This study focuses on adults aged 18 to 70 years who have this specific genetic form of DCM and aims to understand the effects of a single intravenous infusion of ALXN2350. Participants receive one of three dose levels of ALXN2350 depending on their cohort. ALXN2350 consists of an AAV9 capsid containing the BAG3 transgene and is given as a single intravenous infusion. The study includes dose finding and dose expansion phases to assess the therapys effects over time. During the study, participants are monitored for treatment emergent adverse events and serious adverse events from baseline up to Week 78. Cardiac events and time to first major cardiac outcomes such as death, heart transplant, mechanical support, or aborted sudden cardiac death are also tracked. The total study duration for each participant extends up to 78 weeks, with close follow-up to evaluate safety and efficacy.

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy