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

点击翻译按钮获取中文摘要

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.