NCT01760564已完成3 期
Application of Miglustat in Patients With Niemann-Pick Type C
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Swallowing
研究概览
简要总结
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.
详细描述
Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirm diagnosis of Niemann-Pick C disease
- •Symptomatic including motor or mental symptoms
排除标准
- •Unknown severe diarrhea for more than 7 day
- •Allergy to miglustat
研究组 & 干预措施
Miglustat
Experimental
miglustat 200mg tid
干预措施: Miglustat (Drug)
结局指标
主要结局
Swallowing
时间窗: 12th month
videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue
次要结局
- Mental(12th month)
研究者
研究点 (1)
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