NCT05302271View on ClinicalTrials.gov

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

PHASE1RecruitingINTERVENTIONAL
Enrollment

25

Participants

Timeline

Start Date

February 22, 2022

Primary Completion Date

December 31, 2028

Study Completion Date

December 31, 2029

Conditions
Friedreich AtaxiaCardiomyopathiesCardiac HypertrophyMyocardial Fibrosis
Interventions
BIOLOGICAL

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

AAVrh.10hFXN will be administered intravenously.

DRUG

Prednisone

All participants will remain immunosuppression therapy with prednisone for a total of 14 weeks.

Trial Locations (1)

10021

RECRUITING

Weill Cornell Medicine, New York

All Listed Sponsors
collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

lead

Weill Medical College of Cornell University

OTHER