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

NCT05302271 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2026-03-31

No results posted yet for this study

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.

Conditions

  • 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

AAVrh.10hFXN will be administered intravenously.

DRUG

Prednisone

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

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Weill Medical College of Cornell University

    lead OTHER

Principal Investigators

  • Ronald G Crystal, MD · Weill Medical College of Cornell University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
12 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-22
Primary Completion
2028-12-31
Completion
2029-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05302271 on ClinicalTrials.gov