Gene therapy to treat heart problems in Friedreich's ataxia
Phase IA/IB Study of AAVrh.10hFXN Therapy to Treat the Cardiomyopathy of Friedreich's Ataxia
['FUNDING_OTHER'] · WEILL MEDICAL COLL OF CORNELL UNIV · NIH-10914836
This study is testing a new gene therapy called AAVrh.10hFXN to help improve heart health for people with Friedreich's ataxia by delivering a helpful gene directly to the heart, and it's designed for those who experience heart problems due to this condition.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | WEILL MEDICAL COLL OF CORNELL UNIV (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-10914836 on ClinicalTrials.gov |
What this research studies
This research investigates the use of a gene therapy called AAVrh.10hFXN to address heart issues associated with Friedreich's ataxia, a genetic disorder that affects movement and can lead to serious heart complications. The therapy involves delivering a modified virus that carries the human frataxin gene directly to the heart through an intravenous injection. By restoring the levels of frataxin, the therapy aims to reverse heart damage and improve heart function in affected patients. The study will assess the safety and effectiveness of this approach in a clinical setting.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Friedreich's ataxia who are experiencing cardiac symptoms.
Not a fit: Patients without Friedreich's ataxia or those who do not have cardiac manifestations of the disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve heart health and quality of life for patients with Friedreich's ataxia.
How similar studies have performed: Previous research has shown promising results with similar gene therapy approaches in animal models, indicating potential for success in human trials.
Where this research is happening
NEW YORK, UNITED STATES
- WEILL MEDICAL COLL OF CORNELL UNIV — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CRYSTAL, RONALD G — WEILL MEDICAL COLL OF CORNELL UNIV
- Study coordinator: CRYSTAL, RONALD G
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.