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临床试验/NCT04012658
NCT04012658招募中不适用

A Registered Cohort Study on Wilson's Disease

Wan-Jin Chen1 个研究点 分布在 1 个国家目标入组 2,000 人开始时间: 2019年7月1日最近更新:
适应症

试验速览

阶段
不适用
状态
招募中
发起方
入组人数
2,000
试验地点
1
主要终点
The change of Unified Wilson's Disease Rating Scale (UWDRS)

研究概览

简要总结

The aim of this study is to determine the clinical spectrum and natural progression of Wilson's Disease in a prospective multicenter natural history study, to assess the clinical, genetic, epigenetic features and biomarkers of patients with Wilson's Disease to optimize clinical management.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Prospective

入排标准

性别
All
接受健康志愿者

入选标准

  • Patients with the genetic diagnosis of Wilson's Disease
  • Asymptomatic Wilson's Disease carriers
  • Relatives of Wilson's Disease patients or carriers
  • Unrelated healthy controls
  • Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process

排除标准

  • * Participants are unable to comply with study procedures and visit schedule

结局指标

主要结局

The change of Unified Wilson's Disease Rating Scale (UWDRS)

时间窗: Up to 30years

Disease severity will be assessed by application of the Unified Wilson's Disease Rating Scale (UWDRS), a clinical rating scale consists of three subscales. Higher UWDRS total scores indicate more severe disease.

次要结局

未报告次要终点

研究者

发起方
Wan-Jin Chen
申办方类型
Other
责任方
Sponsor Investigator
主要研究者

Wan-Jin Chen

The Vice-Director for the Department of Neurology

First Affiliated Hospital of Fujian Medical University

研究点 (1)

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A Registered Cohort Study on Wilson's Disease | 临床试验