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: 7951706
• 负责人: Chester B. Whitley
• 金额: $ 0.18万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-12-01 至 2009-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7951706
• 关键词: Blood; Clinical Research; Computer Retrieval of Information on Scientific Projects Database; Diet; Funding; Grant; Hyperphenylalaninaemias; Institution; Pharmaceutical Preparations; Phase; Phenylalanine; Rare Diseases; Research; Research Personnel; Resources; Safety; Source; Testing; United States National Institutes of Health; disorder control; open label; phenoptin; 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, Phenoptin, 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利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这项研究将在由于BH4缺乏而患有高苯丙氨酸血症的受试者中测试一种实验性药物苯那普汀。高苯丙氨酸血症(HPA)是一种非常罕见的疾病，据估计全世界有450名患者。没有治愈的方法。控制这种疾病的机制是通过严格的饮食控制来维持血液中较低的苯丙氨酸水平。