Study Overview:

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.

Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.

Study Details:

Friedreich’s ataxia (FA) is a rare, autosomal recessive disease caused by a mutation in the autosomal frataxin (FXN) gene. Progressive cardiomyopathy with cardiac hypertrophy and fibrosis is observed in most individuals with FA. The disease is more severe in those with earlier onset. Presently, there is no therapy that alters the progression of cardiomyopathy in FA, which is responsible for 59% of FA-related deaths.

The primary objective of this dose escalation study is to assess the safety and tolerability of three ascending doses of LX2006 in patients with FA-associated cardiomyopathy. LX2006 is designed to restore hFXN levels in order to improve mitochondrial function. Assessments of cardiac function, biomarkers and other preliminary efficacy endpoints are also included in this study.

Key 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

Key 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

Additional inclusion and exclusion criteria may apply and will be evaluated by a study doctor.

Length of Study Commitment:

  • Five years

Participating Study Locations

Institution Name and LocationStudy Coordinator Contact InformationStatus

Lexeo Clinical Trials



Ataxia Center and Hd Center of Excellence, University of California
Los Angeles, California

Aaron Fisher


University of South Florida
Tampa, Florida

Lucretia Campbell


Mayo Clinic
Rochester, Minnesota

Michaela Kolarova
Clinical Genomics Research Team


Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania

David Lynch, MD, PhD

Not yet recruiting

Explore the FA Drug Development Pipeline

FARA believes that there are many different approaches to treating Friedreich’s ataxia, and that it will require a cocktail approach of two or more treatments to slow, stop, reverse, and cure FA. Learn more about the approach behind this potential treatment and explore the other approaches that are in the FA Drug Development pipeline.