CLINICAL TRIAL: PILOT HUNTINGTON AT RISK OBSERVATIONAL STUDY (PHAROS)

临床试验：飞行员亨廷顿风险观察研究 (PHAROS)

• 批准号: 7951362
• 负责人: JAMES B CARESS
• 金额: $ 1.61万
• 依托单位: WAKE FOREST UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-03-01 至 2010-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7951362
• 关键词: Address; Age; Clinical Research; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Daughter; Disease; Enrollment; Funding; Genes; Grant; Hereditary Disease; Huntington Disease; Individual; Inherited; Institution; Measurement; Movement; Observational Study; Parents; Participant; Persons; Pharmaceutical Preparations; Research; Research Personnel; Resources; Risk; Siblings; Son; Source; Testing; United States; United States National Institutes of Health; Universities; forest; medical schools

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. Huntington's Disease (HD) is an inherited (genetic) disease characterized by changes in movement and intellect that usually begin in adulthood. This observational study will periodically examine individuals who have no definite signs of HD, but who are considered "at risk" for developing the illness because they have (or had) a parent or sibling with HD. Little research has been done in individuals who are at risk for developing an inherited disease (like the son or daughter of a person with HD). Before we can test experimental drugs in healthy individuals who carry the HD gene, we have to address some important issues involving all persons at risk for HD, whether or not they have inherited the abnormal gene. Some questions we hope to answer in PHAROS include: - In a group of people who are at risk for HD, how many will develop signs of the illness over a three-year period? - What are the earliest signs of the illness, and when do they start? - How accurate are the measurements that researchers use in detecting the onset of HD? - What factors influence the age at which a person carrying the HD gene develops the illness? Pharos is a unified research effort involving approximately 38 centers in the United States. A total of 1000 participants are expected to be enrolled, 50 of whom will be enrolled at the Wake Forest University School of Medicine, NC.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目和 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 中心，不一定是研究者的机构。 亨廷顿舞蹈病 (HD) 是一种遗传性疾病，其特征是运动和智力的变化，通常始于成年期。 这项观察性研究将定期检查那些没有明确亨廷顿舞蹈症症状但因父母或兄弟姐妹患有亨廷顿舞蹈症而被认为“处于患病风险”的个体。 对于有患遗传性疾病风险的个体（如亨廷顿舞蹈症患者的儿子或女儿）的研究很少。 在我们能够在携带 HD 基因的健康个体中测试实验药物之前，我们必须解决一些涉及所有有 HD 风险的人的重要问题，无论他们是否遗传了异常基因。 我们希望在 PHAROS 中回答的一些问题包括： - 在一群有 HD 风险的人群中，有多少人会在三年内出现这种疾病的迹象？ - 疾病最早的迹象是什么？它们何时开始？ - 研究人员用于检测 HD 发作的测量结果有多准确？ - 哪些因素会影响携带 HD 基因的人患病的年龄？ Pharos 是一项涉及美国约 38 个中心的统一研究成果。 预计共有 1000 名参与者参加，其中 50 名将在北卡罗来纳州维克森林大学医学院注册。