NCT01563705Unknown不适用
Prospective Follow-up of Patients With Glycogen Storage Disease Type III
Institut de Myologie, France1 个研究点 分布在 1 个国家目标入组 30 人开始时间: 2011年2月1日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Knee flexion and extension strength
研究概览
简要总结
The aim of this study is to improve knowledge of natural history and methods of monitoring the evolution of Glycogen storage disease type III regarding the muscle and to study the prospective approach of large series of patients, and using the same protocol for the follow up of the children and adults.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patients with a debranching enzyme deficiency confirmed by enzymatic analysis (debranching enzyme deficiency).
- •Written consent
- •Age: at least 10 years
- •Affiliated with a social security system
排除标准
- •Pregnant and lactating women
结局指标
主要结局
Knee flexion and extension strength
时间窗: 4 years after baseline
Time to perform each motor function test
时间窗: 4 years after baseline
Gait speed measured during the 6 minutes walk test
时间窗: 4 years after baseline
次要结局
- Accelerometric variables measured for the 6 minutes walk test(4 years after baseline)
- NMR variables measured during each specific NMR sequence(4 years after baseline)
研究者
研究点 (1)
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