Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

Recruiting

Phase 1

AAVrh.10hFXNPhase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

Sponsor:

Weill Medical College of Cornell University

Code:

NCT05302271

Conditions

Friedreich Ataxia

Cardiomyopathies

Cardiac Hypertrophy

Myocardial Fibrosis

Eligibility Criteria

Sex: All

Age: 18 - 50

Healthy Volunteers: Not accepted

Interventions

AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN

Prednisone

Study Details

Eligibility Criteria

Study Design

Interventions and Outcome Measures

Central Contacts and Locations

More Information

Trial information was received from ClinicalTrials.gov and was last updated on 2026-02-11. This information was provided to ClinicalTrials.gov by Weill Medical College of Cornell University on 2025-11-24.