Neuroprotection in Parkinson Disease: Clinical Center

帕金森病的神经保护：临床中心

基本信息

批准号：
7555144
负责人：
Rodger Elble
金额：
$ 12.08万
依托单位：
SOUTHERN ILLINOIS UNIVERSITY SCH OF MED
依托单位国家：
美国
项目类别：
财政年份：
2002
资助国家：
美国
起止时间：
2002-09-30 至 2012-11-30
项目状态：
已结题

项目摘要

DESCRIPTION (provided by applicant): We propose that Southern Illinois University Parkinson Disease Center participate in the design and performance of a large, multicenter clinical trial of neuroprotective agents in Parkinson disease. There are more than 500,000 people in the United States who are affected by Parkinson disease with 50,000 new cases each year. The annual cost is estimated to be 10 billion dollars per year [1]. Parkinson disease is an age-related disease and with the aging of the baby boom population, the number of people with Parkinson disease will increase substantially after the year 2010. Parkinson disease is characterized by a long preclinical phase, the onset of symptoms at about 60 years of age and progression to severe disability over about 10 to 25 years. The objective of the present project is to identify compounds that are safe and effective in retarding clinical progression in Parkinson disease. The specific aims of the project are to: (1) identify compounds that have potential as neuroprotective agents in Parkinson disease; (2) design clinical trials to test the effects of experimental compounds on the rate of progression of Parkinson disease; (3) conduct pilot studies of selected compounds to determine tolerance and safety of their use in man; and, (4) conduct a large, multicenter clinical trial to determine if selected compounds retard the rate of progression in Parkinson disease. The final design of the pilot studies and large, multicenter clinical trial will be developed collaboratively by the Steering and Oversight Committees, the Coordinating Center and Statistical Center, and the NINDS Scientific Program Director for the project. The following suggestions are proposed regarding the study design: (1) to include the study of estrogen as a potential neuroprotective agent in Parkinson disease; (2) to stratify the study population on the basis of rural vs. urban residence; (3) to use cognitive impairment as one of the measures of the progression of Parkinson disease; (5) to study the pharmacogenomics of the selected compounds studied in the large clinical trial; and, (6) to include a brain autopsy study to confirm the diagnosis and a neuropathology study of the effects of the selected compounds tested on the pathological changes in Parkinson disease. It is proposed that the Autopsy/Neuropathology Program at Southern Illinois University serve as the central resource for a brain autopsy and neuropathology study if it is included in the final study design.

描述（由申请人提供）：我们建议伊利诺伊州南部大学帕金森氏病中心参加帕金森氏病神经保护剂的大型多中心临床试验的设计和表现。美国有50万人患有帕金森氏病影响，每年有50,000例新病例。年度成本估计为每年100亿美元[1]。帕金森氏病是一种与年龄有关的疾病，随着婴儿潮一代的老龄化，帕金森病的人数将在2010年之后大幅增加。帕金森病的特征是长时间的临床前阶段，大约60岁的症状和大约10至25年的严重残疾进展到严重的残疾。本项目的目的是识别在延迟帕金森氏病临床进展方面安全有效的化合物。该项目的具体目的是：（1）识别具有帕金森病神经保护剂的潜力的化合物；（2）设计临床试验，以测试实验化合物对帕金森氏病进展率的影响；（3）对选定化合物进行试点研究，以确定其在人类中使用的耐受性和安全性；（4）进行大型多中心临床试验，以确定选定的化合物是否阻碍了帕金森氏病进展的速度。试点研究和大型多中心临床试验的最终设计将由转向和监督委员会，协调中心和统计中心以及该项目的NINDS科学计划主管共同开发。提出了有关研究设计的以下建议：（1）将雌激素作为帕金森氏病潜在的神经保护剂的研究包括在内；（2）根据农村与城市居住地对研究人群进行分层；（3）将认知障碍用作帕金森氏病进展的措施之一；（5）研究大型临床试验中研究的化合物的药物基因组学；（6）包括一项脑尸检研究，以确认诊断和神经病理学研究，以研究所测试化合物对帕金森氏病的病理变化的作用。有人建议，如果将其包括在最终的研究设计中，则伊利诺伊大学南部大学的尸检计划是脑尸检和神经病理学研究的中心资源。