Recruiting NCT06865482
Clinical Course Of Disease In Participants With FA-CM
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Friedreich Ataxia, Cardiomyopathy. Basic parameters: from 6 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Brazil, Canada, Czechia, France +3
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
Overview
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Detailed description
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.
Primary outcome measures
- Characterize cardiac disease presentation and progression among participants [Time frame: 26 weeks]
Secondary outcome measures (12)
- Describe progression of left ventricular wall thickness (LVWT) among this population [Time frame: 52 weeks]
- Describe progression of relative wall thickness (RWT) among this population [Time frame: 52 weeks]
- Describe progression of relative wall mass (RWM) among this population [Time frame: 52 weeks]
- Describe progression of left ventricular ejection fraction (LVEF) among this population [Time frame: 52 weeks]
- Describe progression of left ventricular mass index (LVMi) among this population [Time frame: 52 weeks]
- Describe progression of high sensitivity troponin I among this population [Time frame: 52 weeks]
- Describe participant perception and clinician assessment of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12) [Time frame: 52 weeks]
- Describe clinician assessment of illness in modified Friedreich Ataxia Rating Scale (mFARS) [Time frame: 52 weeks]
- Describe participant perception of illness in Patient Global Impression of Severity (PGI-S) [Time frame: 52 weeks]
- Describe participant perception of illness in Patient Global Impression of Change (PGI-C) [Time frame: 52 weeks]
- Describe patterns of concomitant medication use among this population [Time frame: 12 months]
- Describe changes to medication use among this population [Time frame: 12 months]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 8 centers
- University of California San Diego — La Jolla
- University of South Florida — Tampa
- Indiana University - Riley Children's Health — Indianapolis
- Boston Children's Hospital — Boston
- Mayo Clinic — Rochester
- Washington University School of Medicine in St. Louis — St Louis
- Cincinnati Children's Hospital — Cincinnati
- Oregon Health & Science University — Portland
Brazil · 5 centers
- Hospital Universitário Walter Cantídio - Universidade Federal do Ceará — Fortaleza
- Instituto D'or de Pesquisa e Ensino - Hospital São Rafael — Salvador
- Hospital Das Clínicas Da Universidade Federal De Minas Gerais (UFMG) — Belo Horizonte
- Campinas State University (UNICAMP) — Campinas
- UPC Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto — Ribeirão Preto
Canada · 1 center
- Centre Hospitalier de Universite de Montreal (CHUM) — Montreal
Czechia · 1 center
- Motol University Hospital and the Second Faculty of Medicine — Prague
France · 1 center
- Institut de Cardiologie Hopital Pitie Salpetriere — Paris
Germany · 1 center
- University Hospital Tübingen — Tübingen
Italy · 1 center
- Federico II University — Naples
Spain · 1 center
- Hospital Univeritario Puerta de Hierro de Majadahonda — Majadahonda
Identifiers
NCT: NCT06865482 · LX2006-02