Clinical Trial

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

Active, Not Recruiting Phase 1/2
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Summary
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.
Protocol Amendment History 14 amendments
This ClinicalTrials.gov record has been amended 14 times since 2022-06-30; most recent amendment 2025-12-19.
Status change: Recruiting → Active, Not Recruiting 2024-11-13
Status change: Not Yet Recruiting → Recruiting 2022-08-23
Trial Details
NCT Number NCT05445323
Lead Sponsor Lexeo Therapeutics
Conditions Friedreich Ataxia, Cardiomyopathy, Secondary
Enrollment 8 participants
Start Date 2022-08-24
Primary Completion 2029-09 (estimated)
Study Completion 2029-09 (estimated)
Updated on ClinicalTrials.gov 2025-12-23