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Clinical Course Of Disease In Participants With FA-CM

Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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

Who May Be Eligible (Plain English)

Who May Qualify: - Male or female, ages ≥6 years at the time of signing the willing to sign a consent form (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% Who Should NOT Join This Trial: - 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Locations (10)

University of California San Diego
La Jolla, California, United States
University of South Florida
Tampa, Florida, United States
Indiana University - Riley Children's Health
Indianapolis, Indiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Centre Hospitalier de Universite de Montreal (CHUM)
Montreal, Quebec, Canada
Motol University Hospital and the Second Faculty of Medicine
Prague, Czechia
Institut de Cardiologie Hopital Pitie Salpetriere
Paris, France
Hospital Univeritario Puerta de Hierro de Majadahonda
Majadahonda, Madrid, Spain