Clinical course of cardiomyopathy in people with Friedreich ataxia
Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
This study follows people with Friedreich ataxia who have cardiomyopathy to see how their heart disease changes over time and to identify who may later be eligible for a gene therapy study.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 65 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | Lexeo Therapeutics Industry-sponsored |
| Locations | 14 sites (La Jolla, California and 13 other locations) |
| Trial ID | NCT06865482 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are people aged 6 or older with genetically confirmed Friedreich ataxia (symptom onset ≤25 years) who have left ventricular hypertrophy, an ejection fraction of at least 40%, and no other major structural or ischemic heart disease.
Not a fit: Patients with other causes of cardiomyopathy, significant coronary or primary valvular disease, current IV inotrope therapy, ventricular assist devices, prior heart transplant, or advanced heart failure are unlikely to benefit from this natural-history study.
Why it matters
Potential benefit: If successful, the study could improve understanding of how heart disease progresses in FA-CM and help identify patients who might benefit from gene therapy earlier.
How similar studies have performed: Previous natural-history studies in Friedreich ataxia have described cardiac involvement, but applying a prospective multicenter protocol specifically to select patients for a gene therapy program is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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 ≥40% 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 this trial is running
La Jolla, California and 13 other locations
- 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)
- Cincinnati Children's Hospital — Cincinnati, Ohio, United States (Recruiting)
- Instituto D'or de Pesquisa e Ensino - Hospital São Rafael — Salvador, Estado de Bahia, Brazil (Recruiting)
- Campinas State University (UNICAMP) — Campinas, São Paulo, Brazil (Recruiting)
- Centre Hospitalier de Universite de Montreal (CHUM) — Montreal, Quebec, Canada (Recruiting)
- Motol University Hospital and the Second Faculty of Medicine — Prague, Czechia (Recruiting)
- Institut de Cardiologie Hopital Pitie Salpetriere — Paris, France (Recruiting)
- University Hospital Tübingen — Tübingen, Baden-Wurttemberg, Germany (Recruiting)
- Hospital Univeritario Puerta de Hierro de Majadahonda — Majadahonda, Madrid, Spain (Recruiting)
Study contacts
- Study coordinator: Lexeo Clinical Trials
- Email: clinicaltrials@lexeotx.com
- Phone: 212-547-9879
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.