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

NCT05302271 · clinicaltrials.gov ↗

PHASE1

Phase

RECRUITING

Status

25

Enrollment

OTHER

Sponsor class

Conditions

Friedreich Ataxia
Cardiomyopathies
Cardiac Hypertrophy
Myocardial Fibrosis

Interventions

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

Sponsor

Weill Medical College of Cornell University

Collaborators

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