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Study identifier: NCT05302271 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Friedreich Ataxia · Cardiomyopathies · Cardiac Hypertrophy  ·  Sponsor: Weill Medical College of Cornell University

PhasePhase 1
Planned participants25
Who can joinAll sexes, 12 Years to 50 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Maddie Galbraith, BS  ·  646-962-2672  ·  meg4013@med.cornell.edu

Niamh Savage, BS  ·  646-962-5527  ·  nis2049@med.cornell.edu

Always discuss trial participation with your own doctor first.

Locations (1)

Weill Cornell MedicineNew York, New York, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT05302271