Clinical Course Of Disease In Participants With FA-CM

Официальное название

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

Обзор

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

Подробное описание

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.

Первичные конечные точки

• Characterize cardiac disease presentation and progression among participants [Срок оценки: 26 weeks]

Вторичные конечные точки (12)

• Describe progression of left ventricular wall thickness (LVWT) among this population [Срок оценки: 52 weeks]
• Describe progression of relative wall thickness (RWT) among this population [Срок оценки: 52 weeks]
• Describe progression of relative wall mass (RWM) among this population [Срок оценки: 52 weeks]
• Describe progression of left ventricular ejection fraction (LVEF) among this population [Срок оценки: 52 weeks]
• Describe progression of left ventricular mass index (LVMi) among this population [Срок оценки: 52 weeks]
• Describe progression of high sensitivity troponin I among this population [Срок оценки: 52 weeks]
• Describe participant perception and clinician assessment of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12) [Срок оценки: 52 weeks]
• Describe clinician assessment of illness in modified Friedreich Ataxia Rating Scale (mFARS) [Срок оценки: 52 weeks]
• Describe participant perception of illness in Patient Global Impression of Severity (PGI-S) [Срок оценки: 52 weeks]
• Describe participant perception of illness in Patient Global Impression of Change (PGI-C) [Срок оценки: 52 weeks]
• Describe patterns of concomitant medication use among this population [Срок оценки: 12 months]
• Describe changes to medication use among this population [Срок оценки: 12 months]

Критерии участия

Критерии включения

• 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%

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 8 центров

• 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

Бразилия · 5 центров

• 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

Канада · 1 центр

• Centre Hospitalier de Universite de Montreal (CHUM) — Montreal

Чехия · 1 центр

• Motol University Hospital and the Second Faculty of Medicine — Prague

Франция · 1 центр

• Institut de Cardiologie Hopital Pitie Salpetriere — Paris

Германия · 1 центр

• University Hospital Tübingen — Tübingen

Италия · 1 центр

• Federico II University — Naples

Испания · 1 центр

• Hospital Univeritario Puerta de Hierro de Majadahonda — Majadahonda

Идентификаторы

NCT: NCT06865482 · LX2006-02

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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