Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

About

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

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 focal fibrosis on cardiac MRI
Protocol specified ranges for stroke volume index and 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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