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 Jun 27, 2026 · Last updated Jun 27, 2026

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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Conditions

The condition(s) this trial relates to.

dilated cardiomyopathy 1HH

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY

Contacts and locations

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Study contacts

Contact

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

Locations

Research Site
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Birmingham, Alabama, 35233, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
Research Site
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Boston, Massachusetts, 02115, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
Research Site
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Cincinnati, Ohio, 45219, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
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Portland, Oregon, 97239-3098, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
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Philadelphia, Pennsylvania, 19104, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
Research Site
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Houston, Texas, 77030, United States

Contact Phone: •••-•••-••••
Research Site
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Massa, 54100, Italy
Research Site
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Barcelona, 08035, Spain
Research Site
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Majadahonda, 28222, Spain
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London, SW3 6NP, United Kingdom

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