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 or compound heterozygous), 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, 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 - 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
University of California San Diego
La Jolla 5363943, California 5332921 92037
La Jolla 5363943, California 5332921 92037
Indiana University - Riley Children's Health
Indianapolis 4259418, Indiana 4921868 46202
Indianapolis 4259418, Indiana 4921868 46202
Boston Children's Hospital
Boston 4930956, Massachusetts 6254926 02115
Boston 4930956, Massachusetts 6254926 02115
Washington University School of Medicine in St. Louis
St Louis 4407066, Missouri 4398678 63110
St Louis 4407066, Missouri 4398678 63110
Cincinnati Children's Hospital
Cincinnati 4508722, Ohio 5165418 45229
Cincinnati 4508722, Ohio 5165418 45229
More Details
- 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. After completing at least 26 weeks in Study LX2006-02, participants who meet the eligibility criteria may have the opportunity to participate in an LX2006 interventional study and receive gene therapy.