Phase IA Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
Status:
Recruiting
Trial end date:
2029-12-31
Target enrollment:
Participant gender:
Summary
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.