Gene therapy trial aims to repair hearts in rare neurological disease
NCT ID NCT05445323
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The therapy delivers a healthy copy of the FXN gene to heart cells using a modified virus. The main goal is to check safety over 5 years, while also looking for signs that the treatment improves heart function.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
LX2006 gene therapy (a modified virus carrying a healthy FXN gene)
What this could lead to
If successful, this could point toward a treatment that slows or stops heart damage in people with Friedreich's ataxia.
What could go wrong
This is a very early, small trial with only 8 participants. It is designed mainly to check safety, not to prove the therapy works. Gene therapies can have unexpected side effects.
Disclaimer
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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.
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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Locations
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Ataxia Center and HD Center of Excellence, University of California
Los Angeles, California, 90095, United States
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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University of South Florida
Tampa, Florida, 33612, United States