Gene therapy trial aims to repair hearts in rare neurological disease

NCT ID NCT05445323

Ongoing Disease control Sponsor: Lexeo Therapeutics Source: ClinicalTrials.gov ↗

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.

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.

cardiomyopathy extrinsic cardiomyopathy Friedreich ataxia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CARDIOMYOPATHY, SECONDARY FRIEDREICH ATAXIA

Contacts and locations

Locations

Ataxia Center and HD Center of Excellence, University of California

Los Angeles, California, 90095, United States
Mayo Clinic

Rochester, Minnesota, 55905, United States
University of South Florida

Tampa, Florida, 33612, United States

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