PHASE II OPEN TRIAL OF DICHLOROACETATE IN CONGENITAL LACTIC ACIDEMIA

二氯乙酸治疗先天性乳酸血症的 II 期开放试验

• 批准号: 7374170
• 负责人: BRUCE A BARSHOP
• 金额: $ 0.26万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2006-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7374170
• 关键词: blood; children; conditioning; dichloroacetate; metabolism; sodium; urine

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.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。评价二氯乙酸钠（DCA）治疗先天性线粒体代谢缺陷所致先天性乳酸血症儿童和成人的疗效，DCA是一种已知可降低此类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，降低酸中毒失代偿急性发作的频率，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。