Overview
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.
Eligibility
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
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
Note: Other inclusion and exclusion criteria may apply.