Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia


About this study

The primary purpose of this dose-ranging study is to assess the safety and tolerability of 2 ascending doses of LX2006 gene therapy for the purpose of selecting the appropriate dose for further clinical development. In addition, assessments of biomarkers and preliminary efficacy are included in this study.

The primary purpose of the LTFU is to assess the long-term safety and tolerability of LX2006 up to 5 years post-treatment. Additionally, efficacy assessments will be evaluated quarterly during Year 2 and annually up to 5 years post-treatment.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Confirmed genetic diagnosis of Friedreich’s Ataxia (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.

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

Eligibility last updated 7/13/23. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Ralitza Gavrilova, M.D.

Contact us for the latest status

Contact information:

Clinical Genomics Research

(507) 293-9114

More information


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