Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
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.
Study Type
OBSERVATIONAL
Enrollment
65
University of California San Diego
La Jolla, California, United States
Characterize cardiac disease presentation and progression among participants
Change from baseline in left ventricular mass index (LVMi)
Time frame: 52 weeks
Describe progression of maximal wall thickness (MWT) among this population
Change from baseline
Time frame: 52 weeks
Describe progression of high sensitivity troponin I among this population
Change from baseline
Time frame: 52 weeks
Describe participant perception and clinician assessment of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)
Change from baseline
Time frame: 52 weeks
Describe clinician assessment of illness in modified Friedreich Ataxia Rating Scale (mFARS)
Change from baseline
Time frame: 52 weeks
Describe participant perception of illness in Patient Global Impression of Severity (PGI-S)
Change from baseline
Time frame: 52 weeks
Describe participant perception of illness in Patient Global Impression of Change (PGI-C)
Change from baseline
Time frame: 52 weeks
Describe patterns of concomitant medication use among this population
Change from baseline in concomitant medication use, assessed using medication logs, electronic health records and patient self-report.
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University of South Florida
Tampa, Florida, United States
RECRUITINGIndiana University - Riley Children's Health
Indianapolis, Indiana, United States
RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
RECRUITINGMayo Clinic
Rochester, Minnesota, United States
RECRUITINGWashington University School of Medicine in St. Louis
St Louis, Missouri, United States
RECRUITINGCincinnati Children's Hospital
Cincinnati, Ohio, United States
RECRUITINGOregon Health & Science University
Portland, Oregon, United States
RECRUITINGHospital Universitário Walter Cantídio - Universidade Federal do Ceará
Fortaleza, Ceará, Brazil
RECRUITINGInstituto D'or de Pesquisa e Ensino - Hospital São Rafael
Salvador, Estado de Bahia, Brazil
RECRUITING...and 9 more locations
Time frame: 52 weeks
Describe changes to medication use among this population
Change from baseline in medication use, evaluated using prescription records, patient-reported medication, or clinician-reported changes.
Time frame: 52 weeks
Evaluate cardiovascular (CV) events
Number of CV events defined as death due to any cause plus adjudicated events of hospitalization for heart failure, non-fatal stroke, non-fatal myocardial infarction, non-fatal life-threatening arrhythmia, heart transplant, implantation of left ventricular assisted device (LVAD)
Time frame: 52 weeks
Cumulative measure of CV and non-CV related health care utilization (HRU)
Health care utilization will be assessed through a combination of: * Data captured via Electronic Data Capture (EDC) system (e.g., hospitalizations, emergency room visits, procedures), * Patient-reported information collected by the physician during scheduled study visits.
Time frame: 52 Weeks