Clinical Trials in the USA / NCT06865482
Recruiting
Observational
Clinical Course Of Disease In Participants With FA-CM
NCT06865482 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Friedreich AtaxiaCardiomyopathy
Study summary
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Eligibility
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 ≥30%
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
Primary outcome measure(s)
- Characterize cardiac disease presentation and progression among participants — 52 weeks
Change from baseline in left ventricular mass index (LVMi)
Trial sites (19)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California San Diego | La Jolla | California | Recruiting |
| University of South Florida | Tampa | Florida | Recruiting |
| Indiana University - Riley Children's Health | Indianapolis | Indiana | Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | Recruiting |
| Mayo Clinic | Rochester | Minnesota | Recruiting |
| Washington University School of Medicine in St. Louis | St Louis | Missouri | Recruiting |
| Cincinnati Children's Hospital | Cincinnati | Ohio | Recruiting |
| Oregon Health & Science University | Portland | Oregon | Withdrawn |
| Hospital Universitário Walter Cantídio - Universidade Federal do Ceará | Fortaleza | Ceará | Recruiting |
| Instituto D'or de Pesquisa e Ensino - Hospital São Rafael | Salvador | Estado de Bahia | Recruiting |
| Hospital Das Clínicas Da Universidade Federal De Minas Gerais (UFMG) | Belo Horizonte | Minas Gerais | Recruiting |
| Campinas State University (UNICAMP) | Campinas | São Paulo | Recruiting |
| UPC Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto | Ribeirão Preto | São Paulo | Recruiting |
| Centre Hospitalier de Universite de Montreal (CHUM) | Montreal | Quebec | Recruiting |
| Motol University Hospital and the Second Faculty of Medicine | Prague | Czechia | Recruiting |
| Institut de Cardiologie Hopital Pitie Salpetriere | Paris | France | Recruiting |
| University Hospital Tübingen | Tübingen | Baden-Wurttemberg | Recruiting |
| Federico II University | Naples | Italy | Recruiting |
| Hospital Univeritario Puerta de Hierro de Majadahonda | Majadahonda | Madrid | Recruiting |
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Other trials for the same condition
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06865482 on ClinicalTrials.gov ↗ ← All trials in the USA