A Phase 1/2, Open-Label, Dose Finding Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants with Symptomatic BAG3 Mutation-Associated Dilated Cardiomyopathy

Condition studied

BAG3 Mutation Associated Dilated Cardiomyopathy

Investigational medicinal product(s)

Eligibility

Primary endpoint

Part A : Incidence and severity of TEAEs and SAEs, and clinically significant safety findings (physical examinations, vital sign measurements, clinical laboratory assessments, and ECG results).

Endpoint detail

Part B : Incidence and severity of TEAEs and SAEs, and clinically significant safety findings (physical examinations, vital sign measurements, clinical laboratory assessments, and ECG results)., Part A and B : CFB in cardiac serum biomarker NT‑proBNP through Week 52 and 78., Part A and B : Incidence of cardiac events including heart failure hospitalization, sustained ventricular arrhythmias, appropriate implantable cardioverter defibrillator shock through Week 52 and 78., Part A and B : Time to the first event of death, heart transplant, mechanical circulating support, or aborted sudden cardiac death through Week 52 and 78., Part A and B : CFB of cardiac serum biomarker hs-TnT Week 52 and 78., Part A and B : Amount of virus secreted in blood, feces, saliva, semen, and urine through Week 52 and 78., Part A and B : Incidence of anti-AAV9 and anti BAG3 antibodies, response categories, and titer, in participants with BAG3-associated DCM treated with ALXN2350 through Week 52 and 78., Part A&B: CFB in LVEF at week 52 and 78, Part A & B: CFB in LVEDVI, CFB in LVESVI at Week 52 and 78., Part A & B: CFB in CPET: - VE/VCO2 slope and VE/VCO2 anerobic threshold at Week 52 and 78., Part A & B : CFB in KCCQ through Week 52 and 78., CFB in LVEDD, GLS through Week 52 and 78.