NCT07721025PHASE2Recruiting
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Lexeo Therapeutics
Start Date
6/25/2026
Completion Date
6/1/2032
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 Criteria
Age Range: 6 years to No maximum
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.
Interventions
GENETIC
LX2006
OTHER
Usual Care
Conditions
Friedreich AtaxiaCardiomyopathy, Secondary
Locations
University of South Florida
Tampa, Florida 33612
United States