PHASE II OPEN TRIAL OF DICHLOROACETATE IN CONGENITAL LACTIC ACIDEMIA

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。评估二氯乙酸钠（DCA）治疗先天性乳酸血症儿童和成人由于先天性线粒体代谢错误，这是一种已知的降低这些个体血乳酸浓度的研究药物。确定治疗是否会降低基础条件下血液和尿液中的乳酸浓度，减少急性酸中毒失代偿发作的频率，改善神经系统状态或减缓神经系统恶化，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.