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Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

This study is testing a gene therapy called LX2006 for people with Friedreich ataxia (FA) cardiomyopathy (CM), a heart condition. LX2006 works by delivering a healthy copy of the FXN gene. The study aims to see how well LX2006 works and if it is safe. You might be able to join if you are at least 6 years old, have a confirmed diagnosis of FA with a specific genetic change (GAA expansion), and have heart problems like left ventricular hypertrophy and an abnormal left ventricular mass index. The main way researchers will measure success is by looking at changes in your heart muscle size (left ventricular mass index) after 26 weeks. The current status of this study is unclear.

Study design
This is a Phase 2, open-label, randomized, controlled study, meaning some participants will receive usual care before LX2006. It plans to enroll 26 participants.
What's involved
Participants in Cohort 1 will receive usual care for 26 weeks before getting a single treatment with LX2006. The primary endpoint is measured at Week 26.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint is measured at Week 26, suggesting follow-up for at least that duration.

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NCT07721025

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

Recruiting
PHASE2Ages 6+InterventionalTreatment
Lexeo Therapeutics
~26 participants
Updated 2026-07-22 on ClinicalTrials.gov
What's tested:LX2006Usual Care

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Percent change from baseline in left ventricular mass index by cardiac MRI
Measured over At Week 26

Conditions

Friedreich Ataxia
Cardiomyopathy, Secondary

Explore related trials

Cardiomyopathy in Friedreich's ataxiaAnti-AAVrh.10 antibodyFXN Gene

Where it's being run

1 sites across 1 states
Florida1

Study leadership

  • Lexeo Clinical Trials · STUDY_DIRECTOR · Lexeo Therapeutics, Inc.

Who to contact

Lexeo Clinical Trials
[email protected]
212-547-9879
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Percent change from baseline in left ventricular mass index by cardiac MRIAt Week 26