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Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

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Brief Summary

This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The first portion will evaluate two doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase I/II

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
18 Years
Maximum Age
40 Years

Inclusion Criteria:

  • Confirmed genetic diagnosis of FA, with onset before 25 years of age
  • No contraindications to cardiac biopsies
  • Normal liver and kidney function
  • Protocol specified ranges for cardiopulmonary exercise testing (CPET) arm ergometry
  • Protocol specified ranges for antibodies
  • Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured by cardiac MRI
  • Protocol specified ranges for left ventricular hypertrophy (LVH) on cardiac MRI, defined as left ventricular mass index (LVMi)
  • Protocol specified ranges for focal fibrosis on cardiac MRI
  • Protocol specified ranges for stroke volume index and/or global longitudinal left ventricular strain on cardiac MRI
  • No evidence of active infection of any type, including hepatitis virus (A, B or C) or human immunodeficiency virus (HIV-1 and HIV-2)

Exclusion Criteria:

  • Uncontrolled diabetes
  • History of clinically significant lung function abnormality
  • Contraindication to cardiac MRI
  • Participants who are receiving systemic corticosteroids or other immunosuppressive medications
  • History of coronary artery disease or any structural heart or vascular disease, including but not limited to aortic stenosis and hypertrophic obstructive cardiomyopathy, other than FA cardiomyopathy
  • History of hemodynamically unstable arrhythmias requiring physician intervention; the presence of clinically significant abnormalities as determined by the investigator, other than ECG abnormalities related to FA
  • Uncontrolled psychiatric disease

Other Inclusion/Exclusion criteria to be applied as per protocol.

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Study Stats
Protocol No.
22-000494
Category
Neurology
Principal Investigator
Contact
Aaron Fisher
Location
  • UCLA Westwood
For Providers
NCT No.
NCT05445323
For detailed technical eligibility, visit ClinicalTrials.gov.