Clinical Course Of Disease In Participants With FA-CM
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age6+
SponsorLexeo Therapeutics
About this trial
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Eligibility criteria
Qualifiers
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%
Disqualifiers
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
Trial design
Treatments tested in this trial
- Not listed
Trial groups
65 Participants
are grouped into 2 trial groupsSponsors and collaborators
Source ClinicalTrials.gov