New gene therapy trial hopes to strengthen hearts in rare genetic condition

NCT ID NCT07218887

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

First seen Nov 01, 2025 · Last updated May 25, 2026 · Updated 27 times

Summary

This early-stage study tests a one-time gene therapy called ALXN2350 for adults with a specific genetic heart condition (BAG3-related dilated cardiomyopathy). The heart muscle becomes weak and enlarged, making it hard to pump blood. The treatment aims to fix the faulty gene to improve heart function. About 6 participants will receive the therapy intravenously, and researchers will monitor safety and heart health over time.

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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
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Boston, Massachusetts, 02115, United States
Research Site
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Cincinnati, Ohio, 45219, United States
Research Site
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Portland, Oregon, 97239-3098, United States
Research Site
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Houston, Texas, 77030, United States
Research Site
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Barcelona, 08035, Spain
Research Site
RECRUITING

Majadahonda, 28222, Spain

Conditions

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BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY