Clinical Course Of Disease In Participants With FA-CM

Not yet recruiting

• Conditions: Friedreich Ataxia; Cardiomyopathy

• Registration Number: NCT06865482
• Lead Sponsor: Lexeo Therapeutics

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.

Study Timeline

• Study First Submitted: 2025-02-18
• Status Verified: 2025-03
• Study First Submitted QC: 2025-03-04
• Last Update Submitted: 2025-03-04
• Study First Posted: 2025-03-07
• Last Update Posted: 2025-03-07
• Study Start: 2025-04
• Primary Completion: 2027-04
• Study Completion: 2027-04

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 65

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Characterize cardiac disease presentation and progression among participants	52 weeks	Change from baseline in left ventricular mass index (LVMi)

Secondary Outcome Measures

Name	Time	Method
Describe progression of left ventricular wall thickness (LVWT) among this population	52 weeks	Change from baseline
Describe progression of relative wall thickness (RWT) among this population	52 weeks	Change from baseline
Describe progression of relative wall mass (RWM) among this population	52 weeks	Change from baseline
Describe progression of high sensitivity troponin I among this population	52 weeks	Change from baseline
Describe participant perception of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)	52 weeks	Change from baseline
Describe participant perception of illness in modified Friedreich Ataxia Rating Scale (mFARS)	52 weeks	Change from baseline
Describe participant perception of illness in Patient Global Impression of Severity (PGI-S)	52 weeks	Change from baseline
Describe participant perception of illness in Patient Global Impression of Change (PGI-C)	52 weeks	Change from baseline
Describe patterns of concomitant medication use among this population	12 months	Change from baseline in concomitant medication use, assessed using medication logs, electronic health records and patient self-report.
Describe changes to medication use among this population	12 months	Change from baseline in medication use, evaluated using prescription records, patient-reported medication, or clinician-reported changes.
Evaluate all-cause mortality	12 months	Time from baseline to the first occurrence of any event of death due to any cause
Evaluate major adverse cardiovascular events (MACE)	12 months	Time from baseline to the first occurrence of any MACE, defined as cardiovascular hospitalization/ambulatory visit, non-fatal stroke, non-fatal life-threatening arrhythmia, heart transplant, implantation of left ventricular assisted device (LVAD)
Evaluate healthcare resource utilization (HRU)	12 months