This is a multicenter, Phase 2, open-label, randomized, controlled study. The purpose of the study is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM). LX2006 is an adeno-associated vector encoding the FXN gene (AAVrh.10hFXN).
Cohort one will enroll individuals over 16 years. Individuals in cohort one will be assigned to receive LX2006 or receive usual care for 26 weeks. Individuals in the usual care group will receive LX2006 after 26 weeks. After safety is assessed in a group of participants in cohort 1, cohort 2 will enroll individuals between the ages of 6 and 15 years. All individuals in cohort 2 will receive LX2006.
The primary outcome measure for this study is percent change from baseline in left ventricular mass index, measured by cardiac MRI, at week 26. The study will also collect other cardiac and neurological measures over five years.
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:
Key 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
Key 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
Additional inclusion and exclusion criteria apply and will be evaluated by a study doctor.