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Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
Sponsor: Lexeo Therapeutics
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.
Official title: A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants With Cardiomyopathy Associated With Friedreich's Ataxia
Key Details
Gender
All
Age Range
18 Years - 50 Years
Study Type
INTERVENTIONAL
Enrollment
8
Start Date
2022-08-24
Completion Date
2029-09
Last Updated
2025-12-23
Healthy Volunteers
No
Interventions
Low dose LX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
Mid Dose LX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
High Dose LX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
Locations (3)
Ataxia Center and HD Center of Excellence, University of California
Los Angeles, California, United States
University of South Florida
Tampa, Florida, United States
Mayo Clinic
Rochester, Minnesota, United States