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。子弹和调查员（PI）可能已经从其他NIH来源获得了主要资金，因此可以在其他清晰的条目中代表。列出的机构适用于该中心，这不一定是调查员的机构。评估因先天性乳酸酸血症的儿童和成人的治疗，原因是二氯乙酸钠（DCA）的线粒体代谢误差（DCA）是一种研究性药物，已知在此类个体中降低血液乳酸浓度。确定治疗是否会在基础条件下降低血液和尿液中乳酸的浓度，减少酸性代偿性急性发作的频率，改善神经系统状况或缓慢的神经系统恶化并改善儿童的线性生长。