A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)
Lexeo Therapeutics
Summary
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
- Age range
- 6+ years
- Sex
- All
- Healthy volunteers
- No
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 us…
Interventions
- GeneticLX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
- OtherUsual Care
Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover).
Location
- University of South FloridaTampa, Florida