Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia (NCT05302271)
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). AAVrh.10hFXN is a serotype rh.10 adeno-associated virus gene transfer vector coding for Frataxin (FXN). The drug is administered intravenously. This is a phase 1, open label, dose escalation study with a total of 25 participants.
Who can join: Review the eligibility criteria below.
Location: Weill Cornell Medicine — New York, New York
Age range: 12 Years to 50 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (1 Sites)
- Weill Cornell Medicine — New York, New York
📞 Contact the Study Coordinator
Phone: (646) 962-2672
Email: meg4013@med.cornell.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Friedreich Ataxia
- A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old — Philadelphia, Pennsylvania
- A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA) — Gainesville, Florida
- FA Clinical Outcome Measures — Los Angeles, California
- A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia — Los Angeles, California
- Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia — Los Angeles, California
- Clinical Course Of Disease In Participants With FA-CM — La Jolla, California
Explore by condition: All Friedreich Ataxia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.