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 study will evaluate up to three 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 1/Phase 2

Eligibility

Gender

All

Healthy Volunteers

No

Minimum Age

18 Years

Maximum Age

50 Years

Inclusion Criteria:

Confirmed genetic diagnosis of FA, with onset being before 25 years of age
Protocol specified ranges for antibodies
Protocol specified measures of FA cardiomyopathy

Exclusion Criteria:

Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured by cardiac ECHO
Uncontrolled diabetes
Abnormal liver function
Active infection of any type, including hepatitis virus (A, B or C) or human immunodeficiency virus (HIV-1 and HIV-2)
Contraindication to cardiac MRI
Contraindications to cardiac biopsies
Participants who are receiving systemic corticosteroids or other immunosuppressive medications
History of significant coronary artery disease or any structural heart or vascular disease other than FA cardiomyopathy
Presence of clinically significant, hemodynamically unstable arrhythmias, requiring physician intervention
Presence of clinically significant abnormalities as determined by the investigator, other than ECG abnormalities related to FA
Uncontrolled psychiatric disease