Phase 12 Study of AFTX-201 Gene Therapy for Adults 18 to 55 With BAG3 Mutation-Related Dilated Cardiomyopathy

What this Trial Is About

Researchers are evaluating the safety, tolerability, pharmacodynamics, and preliminary effects of a single intravenous infusion of AFTX-201 gene therapy in adults aged 18 to 55 with dilated cardiomyopathy caused by a BAG3 gene mutation. This Phase 12 open-label trial focuses on this specific genetic condition affecting heart function. Participants will receive one dose of AFTX-201, which is a gene therapy containing the BAG3 transgene delivered through a single intravenous infusion. After the infusion, participants will be monitored closely for safety and clinical outcomes, including heart function and symptom changes. The study includes short-term safety tracking and a long-term follow-up lasting up to 60 months to observe ongoing effects and any adverse events. During the study, participants will undergo various assessments such as measuring cardiac function through tests like left ventricular ejection fraction and peak oxygen consumption, monitoring heart failure symptoms and quality of life questionnaires. Safety will be closely followed for one year after treatment, with additional follow-up visits extending up to five years. The main outcomes include tracking adverse events and changes in heart-related biomarkers and functional status over time.

An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)