Following heart disease in people with Friedreich ataxia
Observes how heart muscle disease develops in people with Friedreich ataxia.
Plain-language title generated from the registry record
Registry title: Clinical Course Of Disease In Participants With FA-CM
Key facts
- Where
- Paris, FranceSee the site
Where in France
- Institut de Cardiologie Hopital Pitie SalpetriereParis, FranceTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Lexeo Therapeutics
- Trial code
- NCT06865482
- Record updated
- 19 October 2026
- Original description
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)Read full original descriptionShow less
Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Inclusion: 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: Currently receiving intermittent or continuous intravenous (IV) inotrope infusion, presence of a ventricular assist device, or history of prior heart transplantation; Prior organ transplantation; Initiation of cardiac resynchronization therapy (CRT) within 6 months prior to screening; History of prior gene transfer or cell therapy; 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; Active hematologic or solid organ malignancy; Poorly controlled diabetes (hemoglobin A1c ≥8%); 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.