Overview
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
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.
