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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 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.

Interventions

DRUG

ALXN2350

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Conditions

BAG3 Mutation Associated Dilated Cardiomyopathy

Locations

Research Site

Birmingham, Alabama 35233

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