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

> **NCT05302271** · PHASE1 · RECRUITING · sponsor: **Weill Medical College of Cornell University** · enrollment: 25 (estimated)

## Conditions studied

- Friedreich Ataxia
- Cardiomyopathies
- Cardiac Hypertrophy
- Myocardial Fibrosis

## Interventions

- **BIOLOGICAL:** AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN
- **DRUG:** Prednisone

## Key facts

- **NCT ID:** NCT05302271
- **Lead sponsor:** Weill Medical College of Cornell University
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2022-02-22
- **Primary completion:** 2028-12-31
- **Final completion:** 2029-12-31
- **Target enrollment:** 25 (ESTIMATED)
- **Last updated:** 2026-03-31

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05302271

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05302271, "Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT05302271. Licensed CC0.

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