NCT06865482招募中不适用
Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 65
- 试验地点
- 23
- 主要终点
- Characterize cardiac disease presentation and progression among participants
研究概览
简要总结
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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%
排除标准
- •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
研究组 & 干预措施
Cohort 1: Participants ≥16 years of age with FA-CM
Participants ≥16 years of age with FA-CM
Cohort 2: Participants ≥6 to <16 years of age with FA-CM
Participants ≥6 to <16 years of age with FA-CM
结局指标
主要结局
Characterize cardiac disease presentation and progression among participants
时间窗: 52 weeks
Change from baseline in left ventricular mass index (LVMi)
Characterize cardiac disease presentation and progression among participants
时间窗: 26 weeks
Change from baseline in left ventricular mass index (LVMi)
次要结局
- Describe progression of left ventricular wall thickness (LVWT) among this population(52 weeks)
- Describe progression of relative wall thickness (RWT) among this population(52 weeks)
- Describe progression of relative wall mass (RWM) among this population(52 weeks)
- Describe progression of high sensitivity troponin I among this population(52 weeks)
- Describe participant perception of illness in Patient Global Impression of Severity (PGI-S)(52 weeks)
- Describe participant perception of illness in Patient Global Impression of Change (PGI-C)(52 weeks)
- Describe patterns of concomitant medication use among this population(12 months)
- Describe changes to medication use among this population(12 months)
- Evaluate all-cause mortality(12 months)
- Evaluate major adverse cardiovascular events (MACE)(12 months)
- Describe progression of left ventricular wall thickness (LVWT) among this population(52 weeks)
- Describe progression of relative wall thickness (RWT) among this population(52 weeks)
- Describe progression of relative wall mass (RWM) among this population(52 weeks)
- Describe progression of high sensitivity troponin I among this population(52 weeks)
- Describe participant perception of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)(52 weeks)
- Describe participant perception of illness in modified Friedreich Ataxia Rating Scale (mFARS)(52 weeks)
- Describe participant perception of illness in Patient Global Impression of Severity (PGI-S)(52 weeks)
- Describe participant perception of illness in Patient Global Impression of Change (PGI-C)(52 weeks)
- Describe patterns of concomitant medication use among this population(12 months)
- Describe changes to medication use among this population(12 months)
- Evaluate all-cause mortality(12 months)
- Evaluate major adverse cardiovascular events (MACE)(12 months)
- Cumulative measure of CV and non-CV related health care utilization (HRU)(12 months)
- Describe progression of maximal wall thickness (MWT) among this population(52 weeks)
- Describe patterns of concomitant medication use among this population(52 weeks)
- Describe changes to medication use among this population(52 weeks)
- Evaluate cardiovascular (CV) events(52 weeks)
- Cumulative measure of CV and non-CV related health care utilization (HRU)(52 Weeks)
- Describe progression of left ventricular ejection fraction (LVEF) among this population(52 weeks)
- Describe progression of left ventricular mass index (LVMi) among this population(52 weeks)
- Describe participant perception and clinician assessment of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)(52 weeks)
- Describe clinician assessment of illness in modified Friedreich Ataxia Rating Scale (mFARS)(52 weeks)
研究者
研究点 (23)
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