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 10 participants. Drug: Prednisone, all participants will remain immunosuppression therapy with prednisone for a total of 14 weeks.
Thursday, April 21, 2022
Phase IA Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
ClinicalTrials.gov Identifier: NCT05302271; Recruitment Status : Recruiting; First Posted : March 31, 2022
Sponsor: Weill Medical College of Cornell University
