Gene therapy trial aims to fix heart damage in rare disease
NCT ID NCT05302271
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This early-stage trial tests a gene therapy called AAVrh.10hFXN for heart problems caused by Friedreich's ataxia, a rare genetic disease. The therapy delivers a working copy of the frataxin gene to help heart cells function better. 25 people aged 12 to 50 with heart involvement will receive a single IV dose, and all will take prednisone for 14 weeks to manage immune reactions. The main goal is to check safety over 5 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AAVrh.10hFXN gene therapy
- What this could lead to
- If successful, this could point toward a treatment that slows or reverses heart damage in Friedreich's ataxia, potentially improving heart function and quality of life.
- What could go wrong
- This is a very early Phase 1 trial with only 25 people, focused on safety. It may not work, and participants must take steroids for 14 weeks, which can have side effects. Long-term risks are unknown.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
-
Weill Cornell Medicine
RECRUITINGNew York, New York, 10021, United States
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