RecruitingNCT06865482

Clinical Course Of Disease In Participants With FA-CM

Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)

Sponsor

Lexeo Therapeutics

Enrollment

65 participants

Start Date

Sep 30, 2025

Study Type

OBSERVATIONAL

Conditions

Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)

Min Age: 6 Years

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%

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

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