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-CM. After completing at least 26 weeks in Study LX2006-02, participants who meet the eligibility criteria may have the opportunity to participate in an LX2006 interventional study and receive gene therapy.
Study Type
OBSERVATIONAL
Enrollment
65
University of California San Diego
La Jolla, California, United States
RECRUITINGUniversity of South Florida
Tampa, Florida, United States
RECRUITINGCharacterize cardiac disease presentation and progression among participants
Change from baseline in left ventricular mass index (LVMi)
Time frame: 26 weeks
Describe progression of left ventricular wall thickness (LVWT) among this population
Change from baseline
Time frame: 52 weeks
Describe progression of relative wall thickness (RWT) among this population
Change from baseline
Time frame: 52 weeks
Describe progression of relative wall mass (RWM) among this population
Change from baseline
Time frame: 52 weeks
Describe progression of left ventricular ejection fraction (LVEF) among this population
Change from baseline
Time frame: 52 weeks
Describe progression of left ventricular mass index (LVMi) 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
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Indiana University - Riley Children's Health
Indianapolis, Indiana, United States
RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
RECRUITINGWashington University School of Medicine in St. Louis
St Louis, Missouri, United States
RECRUITINGCincinnati Children's Hospital
Cincinnati, Ohio, United States
RECRUITINGCentre Hospitalier de Universite de Montreal (CHUM)
Montreal, Quebec, Canada
RECRUITINGMotol University Hospital and the Second Faculty of Medicine
Prague, Czechia
RECRUITINGInstitut de Cardiologie Hopital Pitie Salpetriere
Paris, France
RECRUITINGHospital Univeritario Puerta de Hierro de Majadahonda
Majadahonda, Madrid, Spain
RECRUITINGDescribe 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.
Time frame: 12 months
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: 12 months
Evaluate all-cause mortality
Time from baseline to the first occurrence of any event of death due to any cause
Time frame: 12 months
Evaluate major adverse cardiovascular events (MACE)
Time from baseline to the first occurrence of any MACE, defined as cardiovascular hospitalization/ambulatory visit, non-fatal stroke, non-fatal life-threatening arrhythmia, heart transplant, implantation of left ventricular assisted device (LVAD)
Time frame: 12 months
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: 12 months