Actively Recruiting
Characteristics and Clinical Course of Disease in Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
Led by Lexeo Therapeutics · Updated on 2026-06-08
65
Participants Needed
19
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the characteristics and progression of cardiomyopathy linked to Friedreich Ataxia (FA-CM), a genetic condition affecting the heart. This observational, global study aims to understand how cardiac disease presents and changes over time in participants diagnosed with FA-CM. The study includes both children and adults with genetically confirmed FA-CM and left ventricular hypertrophy and reduced ejection fraction. Participants are divided into two groups based on age: those 16 years and older, and those between 6 and under 16 years old. The study follows participants for at least 26 weeks to track disease progression. After this period, eligible participants may have a chance to join an interventional gene therapy study. The study involves no investigational treatments but focuses on monitoring disease features and changes. During the study, participants undergo assessments including cardiac imaging to evaluate heart structure and function, laboratory tests such as measuring high sensitivity troponin I, and questionnaires assessing illness perception and severity. Researchers will measure changes in left ventricular wall thickness, mass, ejection fraction, medication use, and evaluate major cardiovascular events and mortality over 12 months. The total participation duration and follow-up include detailed evaluations to better understand FA-CM progression.
CONDITIONS
Brief Title
Clinical Course Of Disease In Participants With FA-CM
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 6 years or older at the time of consent
- Diagnosis of Friedreich Ataxia confirmed by clinical signs and genetic testing with onset at age 25 years or younger
- Confirmed left ventricular hypertrophy (LVH)
- Left ventricular ejection fraction of 40% or higher
You will not qualify if you...
- Presence of other types of cardiomyopathy causing heart failure or significant heart abnormalities not due to FA-CM
- Receiving intermittent or continuous intravenous inotrope infusion
- History of ventricular assist device or prior heart transplantation
- Contraindication to cardiac MRI; children under 12 who cannot complete MRI without sedation will have echocardiograms instead
- Prior organ transplantation
- Started cardiac resynchronization therapy within 6 months before screening
- History of gene transfer or cell therapy
- Poorly controlled diabetes with hemoglobin A1c of 8% or higher
- Active blood or solid organ cancer
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 months
Participants are observed over time to characterize the nature and rate of cardiac disease progression associated with Friedreich Ataxia cardiomyopathy (FA-CM).
Multiple visits over 12 months to assess cardiac disease progression and health status
Trial Site Locations
Total: 19 locations
1
University of California San Diego
La Jolla, California, United States, 92037
Actively Recruiting
2
University of South Florida
Tampa, Florida, United States, 33612
Actively Recruiting
3
Indiana University - Riley Children's Health
Indianapolis, Indiana, United States, 46202
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
6
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
7
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229
Actively Recruiting
8
Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
9
Hospital Universitário Walter Cantídio - Universidade Federal do Ceará
Fortaleza, Ceará, Brazil, 60430
Actively Recruiting
10
Instituto D'or de Pesquisa e Ensino - Hospital São Rafael
Salvador, Estado de Bahia, Brazil, 41253-190
Actively Recruiting
11
Hospital Das Clínicas Da Universidade Federal De Minas Gerais (UFMG)
Belo Horizonte, Minas Gerais, Brazil, 30130
Actively Recruiting
12
Campinas State University (UNICAMP)
Campinas, São Paulo, Brazil, 13083-887
Actively Recruiting
13
UPC Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
Ribeirão Preto, São Paulo, Brazil, 14051
Actively Recruiting
14
Centre Hospitalier de Universite de Montreal (CHUM)
Montreal, Quebec, Canada, H2X 0C1
Actively Recruiting
15
Motol University Hospital and the Second Faculty of Medicine
Prague, Czechia, 15006
Actively Recruiting
16
Institut de Cardiologie Hopital Pitie Salpetriere
Paris, France, 75013
Actively Recruiting
17
University Hospital Tübingen
Tübingen, Baden-Wurttemberg, Germany, 72076
Actively Recruiting
18
Federico II University
Naples, Italy, 80131
Actively Recruiting
19
Hospital Univeritario Puerta de Hierro de Majadahonda
Majadahonda, Madrid, Spain, 28222
Actively Recruiting
Research Team
L
Lexeo Clinical Trials
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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