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

Cardiomyopathies Clinical Trial

Official title:
Phase IA Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05302271
Study type Interventional
Source Weill Medical College of Cornell University
Contact Maddie Galbraith, BS
Phone 646-962-4580
Email meg4013@med.cornell.edu
Status Recruiting
Phase Phase 1
Start date February 22, 2022
Completion date December 31, 2029
View Details
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