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
Alexion Pharmaceuticals, Inc.
6 participants
Oct 24, 2025
INTERVENTIONAL
Conditions
Summary
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 Criteria4
- 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 Criteria3
- 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
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Interventions
ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.
Locations(7)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07218887