临床试验/NCT00713245

NCT00713245Unknown不适用

A Long Term Follow up Study in Late-onset Pompe Disease

National Taiwan University Hospital1 个研究点分布在 1 个国家目标入组 70 人开始时间: 2008年5月最近更新: 2013年12月4日

适应症

试验速览

阶段: 不适用
发起方: National Taiwan University Hospital
入组人数: 70
试验地点: 1
主要终点: all muscle function

研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This study is to observe the progression of disease in late-onset Pompe disease

研究设计

研究类型: Observational
观察模型: Case Only
时间视角: Prospective

入排标准

性别: All
接受健康志愿者: 否

入选标准

•individuals with acid alpha glucosidase deficiency without obvious symptoms
•parents signed inform consent

排除标准

•individuals with acid alpha glucosidase deficiency presenting symptoms

结局指标

主要结局

all muscle function

时间窗: 10 years

次要结局

未报告次要终点

研究者

发起方

National Taiwan University Hospital

申办方类型

Other

责任方

Sponsor

研究点 (1)

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