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Can a single gene fix a fatal heart condition? a trial aims to find out

NCT ID NCT07721025

Recruiting now ⭐️ CURE ⭐️ Sponsor: Lexeo Therapeutics Source: ClinicalTrials.gov ↗

First seen Jul 22, 2026 · Last updated Jul 23, 2026 · Updated 1 time

Summary

This phase 2 trial tests whether a single dose of LX2006 gene therapy can improve heart muscle thickening and damage in people with Friedreich ataxia, a genetic disorder that often leads to life-threatening cardiomyopathy. Participants aged 6 and older with confirmed heart enlargement will receive either the gene therapy or usual care, with the option to switch groups after six months. The study measures changes in heart structure and function, as well as neurological symptoms, to see if delivering a working copy of the FXN gene can slow or reverse disease progression.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
a gene therapy called LX2006 that delivers a working copy of the FXN gene via a harmless virus
What this could lead to
If it works, this could become the first treatment to address the underlying genetic cause of heart damage in Friedreich ataxia, potentially slowing or reversing disease progression.
What could go wrong
This is an early phase 2 trial with only 26 participants, so results may not apply to everyone. Gene therapies carry risks such as immune reactions or liver inflammation, and long-term effects are still 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.

cardiomyopathy extrinsic cardiomyopathy Friedreich ataxia heart disorder hypertrophic cardiomyopathy Hypertrophy, Left Ventricular

As listed by the trial registrant

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

CARDIOMYOPATHY, SECONDARY FRIEDREICH ATAXIA

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • University of South Florida

    RECRUITING

    Tampa, Florida, 33612, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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