One-Time gene infusion aims to repair hearts in rare genetic condition

NCT ID NCT07426419

Recruiting now Disease control Sponsor: Affinia Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jul 10, 2026 · Updated 6 times

Summary

This early-stage trial tests a single intravenous dose of AFTX-201, a gene therapy designed to deliver a working copy of the BAG3 gene to heart cells. It enrolls 22 adults aged 18–55 with dilated cardiomyopathy and a BAG3 mutation. The study primarily checks safety and tolerability over 12 months, while also measuring changes in heart function and biomarkers.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: AFTX-201 (gene therapy)
What this could lead to: If successful, this could point toward a one-time gene therapy treatment to improve heart function in people with BAG3-related dilated cardiomyopathy.
What could go wrong: This is an early Phase 1/2 trial with only 22 participants, so safety and effectiveness are not yet proven. Gene therapies can have unexpected side effects, and long-term benefits are uncertain.

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Conditions

The condition(s) this trial relates to.

dilated cardiomyopathy 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 DILATED CARDIOMYOPATHY (DCM)

Contacts and locations

Locations

Houston Methodist Hospital
RECRUITING

Houston, Texas, 77030, United States
St. Lukes Hospital
RECRUITING

Kansas City, Missouri, 64111, United States
University of Miami Hospital and Clinics
RECRUITING

Miami, Florida, 33136, United States

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