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Clinical Course Of Disease In Participants With FA-CM

ConditionsFriedreich AtaxiaCardiomyopathy
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

This trial does not accept healthy volunteers

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 ≥30%

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 population

Up to 65 participants with genetically confirmed FA-CM will be enrolled in the study with allocation by age categories and cohorts

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

65 Participants
are grouped into 2 trial groups
Group A: Cohort 1: Participants ≥16 years of age with FA-CM
Group B: Cohort 2: Participants ≥6 to <16 years of age with FA-CM

Trial outcomes

Primary outcomes

1

Characterize cardiac disease presentation and progression among participants

Change from baseline in left ventricular mass index (LVMi)

Time frame
52 weeks

Secondary outcomes

1

Describe progression of maximal wall thickness (MWT) among this population

Change from baseline

Time frame
52 weeks
2

Describe progression of high sensitivity troponin I among this population

Change from baseline

Time frame
52 weeks
3

Describe participant perception and clinician assessment of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)

Change from baseline

Time frame
52 weeks
4

Describe clinician assessment of illness in modified Friedreich Ataxia Rating Scale (mFARS)

Change from baseline

Time frame
52 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Lexeo Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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