One-Time gene infusion aims to repair hearts in rare genetic condition
NCT ID NCT07426419
First seen Jun 26, 2026 · Last updated Jul 01, 2026 · Updated 3 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.
Disclaimer
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the original study
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Houston Methodist Hospital
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••