Clinical course of cardiomyopathy in people with Friedreich ataxia

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

LX2006-02 is a prospective, longitudinal, low-intervention, multicenter study designed to define the nature and rate of cardiac disease progression in genetically confirmed FA-CM. Participants aged 6 years and older with confirmed GAA expansions, left ventricular hypertrophy, and an ejection fraction ≥40% undergo serial cardiac imaging (cMRI or echocardiography) and standardized clinical assessments. The protocol runs for at least 26 weeks at multiple U.S. centers to map changes in cardiac structure and function and capture markers of progression. Participants who meet eligibility criteria after this period may have the opportunity to enroll in a subsequent LX2006 interventional gene therapy study.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Male or female, ages ≥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 both alleles or compound heterozygous), with onset of FA occurring at ≤25 years of age
* Confirmed left ventricular hypertrophy (LVH)
* Left ventricular ejection fraction ≥30%

Exclusion Criteria:

* Presence of other form(s) of CM contributing to heart failure (HF), clinically significant cardiac anatomic abnormality or congenital cardiac malformation, clinically significant coronary artery, uncorrected, hemodynamically significant primary structural valvular disease not due to CM
* Currently receiving intermittent or continuous intravenous (IV) inotrope infusion, presence of a ventricular assist device, or history of prior heart transplantation
* Contraindication to cMRI, participants \<12 years of age who cannot complete the cMRI without sedation will instead undergo ECHOs and are exempt from this criterion.
* Prior organ transplantation
* Initiation of cardiac resynchronization therapy (CRT) within 6 months prior to screening.
* History of prior gene transfer or cell therapy.
* Poorly controlled diabetes (hemoglobin A1c ≥8%)
* Active hematologic or solid organ malignancy

Where it's running

• University of California San Diego
  La Jolla, California, United States Recruiting
• University of South Florida
  Tampa, Florida, United States Recruiting
• Indiana University - Riley Children's Health
  Indianapolis, Indiana, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting
• Mayo Clinic
  Rochester, Minnesota, United States Recruiting
• Washington University School of Medicine in St. Louis
  St Louis, Missouri, United States Recruiting

Study contacts

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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Nomlabofusp by injection for Friedreich's Ataxia: efficacy and safety

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

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• Genetic testing for AF-triggered cardiomyopathy

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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.