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.
Disclaimer
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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: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Weill Cornell Medicine
RECRUITINGNew York, New York, 10021, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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