Clinical Course Of Disease In Participants With FA-CM
Purpose
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Conditions
- Friedreich Ataxia
- Cardiomyopathy
Eligibility
- Eligible Ages
- Over 6 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| Cohort 1: Participants ≥16 years of age with FA-CM | Participants ≥16 years of age with FA-CM | |
| Cohort 2: Participants 6 to <16 years of age with FA-CM | Participants 6 to <16 years of age with FA-CM |
Recruiting Locations
Washington University in St. Louis and nearby locations
Washington University School of Medicine in St. Louis
St Louis 4407066, Missouri 4398678 63110
St Louis 4407066, Missouri 4398678 63110
More Details
- NCT ID
- NCT06865482
- Status
- Recruiting
- Sponsor
- Lexeo Therapeutics
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.