Clinical trial NCT06865482: Clinical Course Of Disease In Participants With FA-CM
Official title: Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Friedreich Ataxia
Cardiomyopathy
Keywords: Friedreich Ataxia
FA-CM
Cardiomyopathy
FA
Cardiac Disease
Lead sponsor: Lexeo Therapeutics
Sponsor class: INDUSTRY
First posted: 2025-03-07
Enrollment: 65
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Up to 65 participants with genetically confirmed FA-CM will be enrolled in the study with allocation by age categories and cohorts

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

Detailed 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.

Eligibility criteria: 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), 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
* 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

Primary outcomes:
measure: Characterize cardiac disease presentation and progression among participants
timeFrame: 52 weeks
Secondary outcomes:
measure: Describe progression of left ventricular wall thickness (LVWT) among this population
timeFrame: 52 weeks
measure: Describe progression of relative wall thickness (RWT) among this population
timeFrame: 52 weeks
measure: Describe progression of relative wall mass (RWM) among this population
timeFrame: 52 weeks
measure: Describe progression of high sensitivity troponin I among this population
timeFrame: 52 weeks
measure: Describe participant perception of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)
timeFrame: 52 weeks
measure: Describe participant perception of illness in modified Friedreich Ataxia Rating Scale (mFARS)
timeFrame: 52 weeks
measure: Describe participant perception of illness in Patient Global Impression of Severity (PGI-S)
timeFrame: 52 weeks
measure: Describe participant perception of illness in Patient Global Impression of Change (PGI-C)
timeFrame: 52 weeks
measure: Describe patterns of concomitant medication use among this population
timeFrame: 12 months
measure: Describe changes to medication use among this population
timeFrame: 12 months
measure: Evaluate all-cause mortality
timeFrame: 12 months
measure: Evaluate major adverse cardiovascular events (MACE)
timeFrame: 12 months
measure: Evaluate healthcare resource utilization (HRU)
timeFrame: 12 months
Other outcomes:
Locations:
Study officials:
affiliation: Lexeo Therapeutics
name: Lexeo Clinical Trials
role: STUDY_DIRECTOR
References: