NCT05302271

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 25 (estimated)
• Sponsor: Weill Medical College of Cornell University · Academic / Other
• Sex: All
• Age: 12 Years – 50 Years
• Healthy volunteers: Not accepted

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 25 participants.

Conditions

• Friedreich Ataxia
• Cardiomyopathies
• Cardiac Hypertrophy
• Myocardial Fibrosis

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2022-02-22
• Primary completion: 2028-12-31
• Completion: 2029-12-31
• First posted: 2022-03-31
• Last updated: 2026-03-31

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study