A French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat - the MIGA-FAB Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 48
- 试验地点
- 13
- 主要终点
- Safety - vital signs
研究概览
简要总结
This is a noninterventional cohort study to evaluate the effects of migalastat, on long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease.
详细描述
Occurrence of key indicators of safety and effectiveness will be evaluated, such as cardiac, cerebrovascular and renal events, and overall survival. The study is designed to provide effectiveness and safety data by Q2 2023 which will cover a period up to 5 years after the migalastat launch date.
This will involve a retrospective data collection up to migalastat initiation (for patients already receiving migalastat) and a prospective follow-up from 1 to 3.5 years (depending on the time of enrollment) in migalastat-treated patients with Fabry disease who have a GLA mutation amenable to migalastat.
All visits will be scheduled and conducted according to the clinical site's standard of care. Standard of care is defined as a diagnostic and customary clinical treatment/practice process that a clinician chooses according to their clinical judgement for a Fabry disease patient. There are no study- required visits, tests or clinical assessments.
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
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with Fabry disease aged 16 years or older
- •eGFRCKD-EPI > 30 mL/min/1.73 m2
- •treated with migalastat, or who are starting migalastat upon enrollment
- •Patients with Fabry disease and/or parents/guardians (when applicable) who are able to understand and have provided a signed non-opposition form.
- •Equipped with a web connection via a computer or tablet
排除标准
- •Patients who are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment.
- •Patients already included in the present study
结局指标
主要结局
Safety - vital signs
时间窗: up to 60 months
Evaluation of resting blood pressure
Safety - SAEs
时间窗: up to 60 months
Evaluation of the occurrence ie the number of SAEs
Effectiveness - Fabry Associated Clinical Events (FACEs)
时间窗: up to 60 months
Evaluation of the occurrence of the FACEs ie total number of cardiac, cerebrovascular, and renal events
SF-12 12-Item Short Form Health Survey
时间窗: up to 60 months
Evaluation of QOL by the 12-Item Short Form Health Survey (SF-12) ; the higher the score the worse the quality of life is
BPI
时间窗: up to 60 months
Brief pain inventory questionnaire ; the higher the score the more intense the pain is
Cardiac echo imagery
时间窗: up to 60 months
Echocardiogram (Echo) Left Ventricular Mass Index (LVMI)
Treatment compliance
时间窗: up to 60 months
Patient adherence evaluation (% of taken intakes per month) as reported monthly through self-reports of forgotten intakes by the patient
Effectiveness - survival
时间窗: From date of inclusion until the date of death from any cause, assessed up to 60 months
Survival among all patients enrolled, as assessed by recorded patient death from any cause
FABPRO-GI
时间窗: inclusion to last visit
Fabry Disease Patient-Reported Outcome-Gastro intestinal Signs and Symptoms Questionnaire ; the higher the score the more importante the GI symptoms are
次要结局
未报告次要终点