Plain-English translation of NCT07721025 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing a new gene therapy called LX2006 to see if it can help people whose hearts have been damaged by Friedreich ataxia, a rare genetic condition that affects movement and heart function. Researchers want to learn whether this medication is safe and whether it can improve heart health in both teenagers and adults with this condition. The study will compare people who receive the treatment right away with those who receive usual care first, then the medication later.
Friedreich ataxia often damages the heart muscle, leading to serious complications that current treatments cannot fully address. This trial exists to find out if this new gene therapy approach could offer patients a way to slow or improve the heart damage caused by this disease.
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If you join this study, you will be placed into one of two groups based on your age. Depending on which group you're in, you may either receive the medication right away, or you may receive your usual medical care for 26 weeks and then receive the medication. Throughout the study, you'll have regular visits for heart imaging, blood tests, and checkups to see how the treatment is working and to monitor for any side effects.
AI-generated summary from trial data · Sep 2, 2026 · Not medical advice
United States
Phase
Testing effectiveness
Sponsor
Lexeo Therapeutics
Enrollment target
~26 participants
Started
June 2026
Primary completion
June 2032
Age range
6 Years and older
Last updated on clinicaltrials.gov in September 2026.
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Central contact
Lexeo Clinical Trials
Lexeo Therapeutics, Inc.
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