NCT00724802Unknown不适用
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
适应症
相关药物
试验速览
- 阶段
- 不适用
- 入组人数
- 1
- 试验地点
- 2
- 主要终点
- brain MRI at the end of the study
研究概览
简要总结
Canavan disease is caused by Aspartoacylase deficiency. There is no treatment for the disease, but there is a food additive that includes acetate . We suggest an early treatment with acetate and a neurologic evaluation, including MRI, after 4 months of treatment. In any case the treatment will be stopped at the age of 22 months, when myelinization is ended.
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
- 年龄范围
- — 至 18 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •clinical diagnosis of Canavan disease
排除标准
- •Age above 18 months
结局指标
主要结局
brain MRI at the end of the study
时间窗: 6 months
次要结局
未报告次要终点
研究者
研究点 (2)
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