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ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
RecruitingPhase 1/2
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
Who can join
Ages 18 to 70 · All sexes
Full eligibility criteria
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.
About the study
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.
What is being tested
- ALXN2350 (drug)
Sponsor: Alexion Pharmaceuticals, Inc. · Participants: 6 · Started: Oct 24, 2025
Contact the study team
- Alexion Pharmaceuticals, Inc. (Sponsor) · Phone: 1-855-752-2356
Official record on ClinicalTrials.gov — NCT07218887
Locations in the U.S.
| Alabama | Research Site, Birmingham |
| California | Research Site, Palo Alto |
| Massachusetts | Research Site, Boston |
| Ohio | Research Site, Cincinnati |
| Oregon | Research Site, Portland |
| Pennsylvania | Research Site, Philadelphia |
| Texas | Research Site, Houston |
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.