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
- •PROTOCOL ENTRY CRITERIA:
- •-Disease Characteristics--
- •Diagnosis of congenital lactic acidosis (CLA) meeting the following criteria: Three basal venous lactates at least 2.5 mM, arterial lactates at least 2.0 mM, or CSF lactates at least 2.5 mM OR any combination of these, obtained over at least 1 month and within 6 months OR Increase in blood lactate at least 1.0 mM over basal following a carbohydrate meal challenge
- •Enzymatic or molecular genetic proof of a defect of pyruvate dehydrogenase complex, one or more respiratory chain enzymes, or a Krebs cycle enzyme OR Over production of C14-lactate from C14-glucose by cultured skin fibroblasts
- •Ability to withstand an 8 hour (if 2 years and under) or 12 hour (if over 2 years) fast without developing hypoglycemia (blood glucose less than 50 mg/dL)
- •No secondary lactic acidosis due to impaired oxygenation or circulation
- •No hyperlactatemia associated with proven biotinidase deficiency (biotin responsive CLA) or with enzyme deficiencies of gluconeogenesis
- •No primary, defined organic acidurias other than lactic acidosis, for which effective therapy is available (e.g., propionic aciduria)
- •No primary disorders of amino acid metabolism or fatty acid oxidation
- •No malabsorption syndromes associated with D-lactic acidosis
- •-Prior/Concurrent Therapy--
- •No chronic dialysis
- •-Patient Characteristics--
- •Hepatic: No primary hepatic disease unrelated to CLA
- •Renal: Creatinine less than 1.2 mg/dL OR Creatinine clearance at least 60 mL/min
- •Other: No concurrent infection or fever
排除标准
- 未提供