This study examines LX2006 gene therapy in patients with cardiomyopathy caused by Friedreich ataxia. The treatment aims to deliver a working copy of the gene encoding the frataxin protein to heart tissue. It is currently recruiting.
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: * Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable). * Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene) * Onset of FA on or before 25 years of age * Confirmed left ventricular hypertrophy and abnormal left ventricular mass index * Left ventricular ejection fraction at least 30% * Anti-AAVrh.10 total antibody titer less than the protocol-specified maximum level Exclusion Criteria: * Presence of other forms of cardiomyopathy that contribute to heart failure * Current use of inotrope infusion or presence of a ventricular assist device * Contraindication to cardiac MRI * Prior organ transplant * Previous gene transfer or cell therapy * Poorly controlled diabetes (hemoglobin A1c ≥8%) * Active hematologic or solid organ cancer Other inclusion/exclusion criteria to be applied as per protocol.