ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

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

This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.

Inclusion Criteria:

• Pathogenic or likely pathogenic mutation in BAG3
• Medical history of diagnosis of DCM
• Stable combination of HF SoC medications
• Adequate acoustic windows for echocardiography
• LVEF > 15% and ≤ 45% at Screening by echocardiography
• Diagnosis of chronic HF for at least 3 months
• NYHA Class I/II/III at Screening

Exclusion Criteria:

• Presence of antibodies to AAV9
• Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM
• Decompensated HF
• Cardiac ventricular arrhythmia that requires treatment
• History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency
• NYHA Class IV
• History of substance abuse that could contribute to cardiac dysfunction

Note: Other inclusion and exclusion criteria may apply.