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

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。 亨廷顿氏病（HD）是一种遗传性疾病，其特征是运动和智力的变化，通常开始于成年期。 这项观察性研究将定期检查那些没有明确的HD体征，但被认为有发展这种疾病的“风险”的人，因为他们有（或曾经有）父母或兄弟姐妹患有HD。 很少有研究在有遗传性疾病风险的个体（如HD患者的儿子或女儿）中进行。 在我们能够在携带HD基因的健康个体中测试实验性药物之前，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： - 在一群有患HD风险的人中，有多少人会在三年内出现这种疾病的迹象？ - 疾病的早期症状是什么，什么时候开始？ - 研究人员在检测HD发病时使用的测量方法有多准确？ - 什么因素会影响携带HD基因的人发病的年龄？ 灯塔是一个统一的研究工作，涉及大约38个中心在美国。 预计将招募1000名参与者，其中50人将在北卡罗来纳州的维克森林大学医学院就读。

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.