NCT04012671招募中不适用
A Registered Cohort Study on Duchenne Muscular Dystrophy
Ning Wang, MD., PhD.1 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
- Age at death
研究概览
简要总结
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy 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
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Beyond 2 years old
- •Diagnosis with Duchenne Muscular Dystrophy, and female carriers, genotypically confirmed
- •Diagnosis should be supported by muscle biopsy, if no genetic confirmation.
排除标准
- •Presence of other clinically significant illness
结局指标
主要结局
Age at death
时间窗: 20 years
the time when patient die
次要结局
未报告次要终点
研究者
Ning Wang, MD., PhD.
Professor
First Affiliated Hospital of Fujian Medical University
研究点 (1)
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