RecruitingPhase 1NCT05302271

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

Sponsor

Weill Medical College of Cornell University

Enrollment

25 participants

Start Date

Feb 22, 2022

Study Type

INTERVENTIONAL

Conditions

Cardiomyopathies Friedreich Ataxia Myocardial Fibrosis Cardiac Hypertrophy

Summary

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.

Eligibility

Min Age: 12 YearsMax Age: 50 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN and a drug called Prednisone for people with cardiac hypertrophy, cardiomyopathies, and other related conditions. The study is currently recruiting participants at 1 location. People eligible for this study include aged 12 Years to 50 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALAAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN

AAVrh.10hFXN will be administered intravenously.

DRUGPrednisone

All participants will remain immunosuppression therapy with prednisone for a total of 14 weeks.

Locations(1)

Weill Cornell Medicine

New York, New York, United States

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NCT05302271

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