Overview
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Description
Study LX2006-02 is a prospective, longitudinal, low-intervention, multicenter, global study aimed at characterizing the nature and rate of cardiac disease progression in participants with genetically confirmed FA-CM. After completing at least 26 weeks in Study LX2006-02, participants who meet the eligibility criteria may have the opportunity to participate in an LX2006 interventional study and receive gene therapy.
Eligibility
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
