Clinical Course Of Disease In Participants With FA-CM

Recruiting now

Conditions studied: Friedreich Ataxia, Cardiomyopathy

In brief

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

Key facts

Study ID

NCT06865482

Run by

Lexeo Therapeutics

People needed

65

Starts

2025-09-30

Expected to finish

2027-09-01

Last updated by the study team

2026-06-08

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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%

You may not qualify if…

• 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

Where it is running

• University of California San Diego — La Jolla, California, United States (enrolling)
• University of South Florida — Tampa, Florida, United States (enrolling)
• Indiana University - Riley Children's Health — Indianapolis, Indiana, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• Mayo Clinic — Rochester, Minnesota, United States (enrolling)
• Washington University School of Medicine in St. Louis — St Louis, Missouri, United States (enrolling)
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States (enrolling)
• Oregon Health & Science University — Portland, Oregon, United States (enrolling)
• Hospital Universitário Walter Cantídio - Universidade Federal do Ceará — Fortaleza, Ceará, Brazil (enrolling)
• Instituto D'or de Pesquisa e Ensino - Hospital São Rafael — Salvador, Estado de Bahia, Brazil (enrolling)
• Hospital Das Clínicas Da Universidade Federal De Minas Gerais (UFMG) — Belo Horizonte, Minas Gerais, Brazil (enrolling)
• Campinas State University (UNICAMP) — Campinas, São Paulo, Brazil (enrolling)
• UPC Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto — Ribeirão Preto, São Paulo, Brazil (enrolling)
• Centre Hospitalier de Universite de Montreal (CHUM) — Montreal, Quebec, Canada (enrolling)
• Motol University Hospital and the Second Faculty of Medicine — Prague, Czechia (enrolling)
• Institut de Cardiologie Hopital Pitie Salpetriere — Paris, France (enrolling)
• University Hospital Tübingen — Tübingen, Baden-Wurttemberg, Germany (enrolling)
• Federico II University — Naples, Italy (enrolling)
• Hospital Univeritario Puerta de Hierro de Majadahonda — Majadahonda, Madrid, Spain (enrolling)

Full record on ClinicalTrials.gov

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