Apply to trial NCT05302271

A few quick questions so the study team can decide if you might be a fit.

RecruitingPhase 1Biological intervention

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

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.

How this works

Answer a few questions
About 5 to 10 minutes. Skip-friendly where possible.
We connect you with the study team
When a site picks up your application, we introduce you to their team directly — with you on the thread.
The team reaches out to schedule screening
Usually within a few business days, via the contact you give.

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

How this works

Answer a few questions

We connect you with the study team

The team reaches out to schedule screening