This study examines a gene therapy called LX2006 (at different doses) in patients with heart muscle disease (cardiomyopathy) associated with Friedreich's ataxia. It enrolls patients with a genetically confirmed diagnosis of Friedreich's ataxia whose symptoms began before the age of 25. The study is in Phase 1/Phase 2 and is currently active but not recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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 Other Inclusion/Exclusion criteria to be applied as per protocol.