PHASE II OPEN TRIAL OF DICHLOROACETATE IN CONGENITAL LACTIC ACIDEMIA

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。评价二氯乙酸钠(DCA)治疗儿童和成人先天性乳酸血症的疗效。二氯乙酸钠是一种研究药物，可降低先天性乳酸血症患者的血乳酸浓度。确定治疗是否能降低基础状态下的血乳酸和尿乳酸浓度，减少酸中毒失代偿急性发作的频率，改善神经状况或减缓神经恶化，并改善儿童的线性生长。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Evaluation of the treatment of children and adults with congenital lactic acidemia due to inborn errors of mitochondrial metabolism with sodium dichloroacetate (DCA) an investigational drug known to lower blood lactic acid concentrations in such individuals. Determine if treatment will decrease concentrations of lactic acid in blood and urine under basal conditions, decrease the frequency of acute episodes of acidotic decompensation, improve neurological status or slow neurological deterioration, and improve linear growth in children.