New gene therapy aims to fix inherited heart condition

NCT ID NCT07218887

Recruiting now Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 25 times

Summary

This early-stage study tests a single-dose gene therapy called ALXN2350 in 6 adults with dilated cardiomyopathy caused by a BAG3 gene mutation. The goal is to see if the treatment is safe and can improve heart function. Participants must have stable heart failure treatment and no antibodies to the virus used to deliver the therapy.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Research Site
RECRUITING

Birmingham, Alabama, 35233, United States
Research Site
RECRUITING

Boston, Massachusetts, 02115, United States
Research Site
RECRUITING

Cincinnati, Ohio, 45219, United States
Research Site
RECRUITING

Portland, Oregon, 97239-3098, United States
Research Site
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Houston, Texas, 77030, United States
Research Site
RECRUITING

Barcelona, 08035, Spain
Research Site
RECRUITING

Majadahonda, 28222, Spain

Conditions

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BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY