Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

Plain-language summary

This study examines LX2006 gene therapy in patients with cardiomyopathy caused by Friedreich ataxia. The treatment aims to deliver a working copy of the gene encoding the frataxin protein to heart tissue. It is currently recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Recruiting

Sponsor

Lexeo Therapeutics

Intervention

LX2006, Usual Care

Start date

6/25/2026

Estimated completion

6/1/2032

Countries

United States

Related genes

Locations

• Tampa, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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.