Clinical Center for Neuroprotection in Parkinson's Disease

帕金森病神经保护临床中心

• 批准号: 7544278
• 负责人: RICHARD B DEWEY
• 金额: $ 13.02万
• 依托单位: UT SOUTHWESTERN MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-15 至 2012-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7544278
• 关键词: Blinded; Brain; Clinical; Clinical Trials; Clinical Trials Cooperative Group; Condition; Country; Deterioration; Development; Disease; Disease Progression; Dopamine; Follow-Up Studies; Medical center; Movement Disorders; Nerve Degeneration; Neurologist; Neurons; Neuroprotective Agents; Numbers; Parkinson Disease; Pathogenesis; Patients; Personal Satisfaction; Pilot Projects; Reporting; Research; Research Design; Series; Site; Substantia nigra structure; Testing; Texas; clinical research site; design; disabling disease; experience; improved; insight; neurochemistry; neuroprotection

DESCRIPTION (provided by applicant): Parkinson's disease is an inexorably progressive disorder of unknown cause in which neurons of the substantial nigra progressively degenerate resulting in ever-greater degrees of brain dopamine deficiency. While a number of treatments have been developed that improve the neurochemical deficit, no treatment has been demonstrated to ameliorate the neuronal deterioration. Such a neuroprotective effect is highly desirable because if this could be achieved, a significant delay in clinical deterioration of patients could be realized. The proposal outlined here aims to establish a Clinical Center for neuroprotective research at UT Southwestern Medical Center which will cooperate with up to 42 other such centers around the country in the development and execution of a large-scale clinical trial to test one or more potentially neuroprotective agents for Parkinson's disease. The Clinical Center for Movement Disorders at UT Southwestern Medical Center in Dallas, TX is well suited to becoming such a Clinical Center. It is the primary academic referral center for North Texas and its two staff neurologists are experienced in the design, conduct, and reporting of clinical trials in Parkinson's disease. Once the Clinical Centers are designated, a series of pilot studies will be conducted first to identify the most promising agent or agents. Subsequently, a large trial involving several thousand patients will be developed and conducted to establish whether the chosen agent is actually capable of slowing down the progression of this disease. By studying such a large number of patients under rigorously blinded and controlled conditions, it should be possible to identify even a small beneficial effect on disease progression which would nevertheless be very important, not only for the implications on retarding disease progression, but also because observing such an effect might yield insights into the underlying pathogenesis of this common and disabling disease.

描述（由申请人提供）：帕金森氏病是一种不可避免的疾病，存在着未知原因的疾病，其中大量的nigra的神经元逐渐退化，导致脑多巴胺缺乏症的不断变化。尽管已经开发了许多改善神经化学缺陷的治疗方法，但尚未证明可以改善神经元恶化的治疗方法。这种神经保护作用是非常可取的，因为如果可以实现这一点，则可以实现患者临床恶化的显着延迟。此处概述的提案旨在在UT西南医学中心建立一个神经保护研究中心，该中心将与全国多达42个其他此类中心合作，以开发和执行大规模的临床试验，以测试一个或多个潜在的神经保护药物治疗帕金森氏病。 德克萨斯州达拉斯的UT西南医学中心运动障碍临床中心非常适合成为这样的临床中心。它是北德克萨斯州的主要学术转诊中心，其两名员工神经科医生在帕金森氏病临床试验的设计，行为和报告中经验丰富。指定临床中心后，将首先进行一系列试验研究，以识别最有前途的药物或药物。随后，将开发并进行一项涉及数千名患者的大型试验，以确定所选药物是否实际上能够减慢该疾病的进展。通过在严格盲目和受控状况下研究如此大量的患者，甚至可以识别出对疾病进展的微小有益作用，这仍然非常重要，这不仅是对延迟疾病进展的影响，而且还可以使这种影响能够洞悉这种常见和破坏这种常见疾病的潜在病原体。