LX2006 Gene Therapy for Friedreich Ataxia Cardiomyopathy (FA-CM)

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, open-label, randomized, controlled Phase 2 trial evaluates the safety and potential benefit of LX2006 gene therapy in FA patients with cardiomyopathy. Participants must have FA with LV hypertrophy and an LV ejection fraction of at least 30%, and be eligible for AAVrh.10–based treatment. The intervention is LX2006 alongside usual care, with enrollment limited to institutions including University of South Florida under Lexeo Therapeutics leadership.

Why it matters

Who can join: the full rules

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.

Where it's running

• University of South Florida
  Tampa, Florida, United States Recruiting

Study contacts

• Study coordinator: Lexeo Clinical Trials
• Email: clinicaltrials@lexeotx.com
• Phone: 212-547-9879

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.