NCT03201627Unknown早期 1 期
a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine1 个研究点 分布在 1 个国家目标入组 18 人开始时间: 2017年7月5日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 早期 1 期
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- NPC clinical severity score
研究概览
简要总结
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 7 Years 至 40 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •aged from 7 years to 40 years
- •onset of neurological symptoms prior to 15 years of age
- •confirmed diagnosis of NPC1 determined by either two NPC1 mutations or one NPC1 mutation with elevated plasma 7-ketocholesterol
- •ability to walk either independently or with assistance
- •Subject or parent/guardian able to communicate with investigator, understand and abide the rules of this clinical trial.
- •parent/guardian able to accompany the subjects to participate in the clinical trial
- •Subject or parent/guardian must provide written informed consent
排除标准
- •One NPC clinical severity scale score reached 5
- •Female in pregnancy or breastfeeding
- •The predicted life span less than 1 year
- •Severe liver insufficiency (defined as hepatic laboratory parameters, AST and/or ALT greater than three-times the upper limit of normal
- •Renal insufficiency, with the serum creatine greater than 1.5 times the upper limit of normal
- •Free with neurological symptoms
- •Take antidiuretic hormone
研究组 & 干预措施
treatment
Experimental
干预措施: Lithium Carbonate (Drug)
结局指标
主要结局
NPC clinical severity score
时间窗: 52 weeks
次要结局
- 7-ketocholesterol(52 weeks)
研究者
Huiwen Zhang, MD
Associate Prof.
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
研究点 (1)
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