NCT04916977已完成不适用
Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease: a Retrospective, Longitudinal and Cross-sectional Study
Chiesi Italia7 个研究点 分布在 1 个国家目标入组 200 人开始时间: 2021年6月23日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 200
- 试验地点
- 7
- 主要终点
- Comprehensive burden
研究概览
简要总结
GROUND study is an Italian, multicenter retrospective longitudinal cohort study with a cross-sectional phase with the aim to quantify the severe clinical burden in terms of severe and fatal outcomes and extension of clinical impairment in the Italian Fabry Disease patients' population
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult patients living or deceased of either sex (age ≥18 years) with a documented FD diagnosis in the last 10 years from the beginning of the study.
- •Patients with at least 3 years of follow-up or early occurrence of severe/fatal outcomes. Also, adult patients who were lost by their care provider or physician and cannot be traced, but for which it is possible to retrieve data for at least 3 years before the last available follow-up visit or from diagnosis to the occurrence of severe/fatal outcomes if earlier, can be included.
- •Written informed consent to undergo in the cross-sectional protocol clinical visit and to retrospectively collect genetic/clinical data. In case of deceased or untraceable patients, the written informed consent is not required to collect retrospective clinical data (excluding genetic data).
排除标准
- •Not applicable
结局指标
主要结局
Comprehensive burden
时间窗: Baseline
Comprehensive burden intended as a measure of extension of clinical impairment assessed by median Mainz Severity Score Index (MSSI) at last follow-up or cross-sectional visit.
Occurrence of severe and fatal clinical outcomes
时间窗: Baseline
Occurrence of severe and fatal clinical outcomes derived by event-free survival from birth to last follow-up or cross-sectional visit;
次要结局
- Hamilton depression scale(Baseline)
- Pittsburgh Sleep Quality index(Baseline)
- Brief pain inventory score(Baseline)
- Percentage of patients with disease activity(Baseline)
- SF-36(Baseline)
- Percentage of patients with disease progression(From the first available FASTEX score to baseline)
- HAQ-disability index(Baseline)
- Patients prescribed to different treatments(Baseline)
- Time from first manifestation to diagnosis(Baseline)
研究者
研究点 (7)
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