NCT07218887PHASE1, PHASE2Recruiting
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
Alexion Pharmaceuticals, Inc.
Start Date
10/24/2025
Completion Date
1/27/2032
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 Criteria
Age Range: 18 years to 70 years
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.
Interventions
DRUG
ALXN2350
Conditions
BAG3 Mutation Associated Dilated Cardiomyopathy
Locations
Research Site
Birmingham, Alabama 35233
United States
Research Site
Palo Alto, California 94304
United States
Research Site
Boston, Massachusetts 02115
United States
Research Site
Cincinnati, Ohio 45219
United States
Research Site
Portland, Oregon 97239-3098
United States
Research Site
Philadelphia, Pennsylvania 19104
United States
Research Site
Houston, Texas 77030
United States
Research Site
Massa, 54100
Italy
Research Site
Barcelona, 08035
Spain
Research Site
Majadahonda, 28222
Spain
Research Site
London, SW3 6NP
United Kingdom