Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 45
研究概览
简要总结
OBJECTIVES:
I. Compare the safety of sodium dichloroacetate (DCA) vs placebo in children with congenital lactic acidosis.
II. Determine the quality of life of these patients.
III. Determine the pharmacokinetics and metabolic fate of DCA over the course of drug administration in these patients.
详细描述
PROTOCOL OUTLINE: This is a randomized, double blind, crossover study. Patients are stratified according to age (3 months to 2 years vs over 2 to 18 years).
All patients receive at least 12 months of sodium dichloroacetate (DCA) during a 2 year period of double blind, crossover evaluation of DCA and placebo by mouth.
Quality of life is assessed before treatment and periodically during treatment.
Completion date provided represents the completion date of the grant per OOPD records
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double
入排标准
- 年龄范围
- 3 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供