PKU-007: A PHASE 2, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY AND EFF

PKU-007：评估安全性和有效性的 2 期、多中心、开放标签研究

• 批准号: 7606093
• 负责人: Chester B. Whitley
• 金额: $ 0.22万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-12-01 至 2007-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7606093
• 关键词: Blood; Computer Retrieval of Information on Scientific Projects Database; Diet; Funding; Grant; Hyperphenylalaninaemias; Institution; Label; Pharmaceutical Preparations; Phase; Phenylalanine; Rare Diseases; Research; Research Personnel; Resources; Safety; Source; Testing; United States National Institutes of Health; disorder control; effusion; tetrahydrobiopterin

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. This study will test an experimental medication in subjects who have hyperphenylalaninemia due to a primary BH4 deficiency. Hyperphenylalaninemia (HPA) is a very rare disease, estimated to have 450 sufferers worldwide. There is no cure. The mechanism to control this disease is done through strict diet control in order to maintain a low blood phenylalanine level.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 本研究将在因原发性BH 4缺乏而患有高苯丙氨酸血症的受试者中测试实验性药物。高苯丙氨酸血症（HPA）是一种非常罕见的疾病，估计全世界有450名患者。无药可救。控制这种疾病的机制是通过严格的饮食控制来实现的，以保持低的血液苯丙氨酸水平。