Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
Public ClinicalTrials.gov record NCT05302271. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Study identification
- NCT ID
- NCT05302271
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Enrollment
- 25 participants
Conditions and interventions
Interventions
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN Biological
- Prednisone Drug
Biological · Drug
Eligibility (public fields only)
- Age range
- 12 Years to 50 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 21, 2022
- Primary completion
- Dec 30, 2028
- Completion
- Dec 30, 2029
- Last update posted
- Mar 30, 2026
2022 – 2029
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Weill Cornell Medicine | New York | New York | 10021 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05302271, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 30, 2026 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05302271 live on ClinicalTrials.gov.