Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

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

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.

Conditions3

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