Neuroprotection Exploratory Trials in PD (NET-PD)

描述（由申请人提供）：帕金森病（PD）是一种缓慢但持续进行的神经退行性疾病，导致临床症状随时间推移而恶化。抗PD药物有效地改善了大多数患者的大多数主要帕金森病体征和症状。然而，传统的药物治疗并不能改变运动功能和功能性残疾随时间的进行性恶化。因此，非常需要能够减缓临床进展、增强受损神经元的修复、恢复或增强多巴胺（DA）系统的残留部分的功能的疾病修饰药物。由于疾病进展通常缓慢，因此可以在相对长的时间段内实施靶向多种致病机制的化合物的治疗。NET-PD多中心试验旨在测试潜在神经保护化合物在早期PD患者中的疗效。在无效性研究中确定有效的化合物将在大型双盲安慰剂对照研究中进行测试。NINDS的神经变性和临床试验组正在申请新的中心合作进行临床试验，以测试潜在的神经保护剂。我们正在努力成为一个网络中心。肯塔基州大学的运动障碍中心服务于大列克星敦地区，这是一个主要的城市社区，也是肯塔基州中部、东部和东南部的63个县的大部分农村地区。此外，我们治疗了几名居住在西弗吉尼亚州和南俄亥俄州的PD患者（-25）。我们跟踪约600名PD患者，平均每月诊断5名新的PD患者。因此，我们预计初步试验每月能够入组1-2例早期PD患者。

DESCRIPTION (provided by applicant): Parkinson's disease (PD) is a slowly but relentlessly progressive neurodegenerative disorder resulting in a time-dependent worsening of clinical symptoms. Anti-PD drugs effectively ameliorate most of the major parkinsonian signs and symptoms in a majority of patients. However, traditional drug therapies do not modify the progressive deterioration of motor function and functional disability worsens over time. Thus, disease modifying drugs that can slow clinical progression, enhance repair of damaged neurons, restore, or enhance function of residual parts of the dopamine (DA) system are much needed. Since disease progression is generally slow, treatment with compounds that target multiple pathogenic mechanisms can be implemented over a relatively long period of time. The NET-PD multicenter trial is aimed at testing the efficacy of potential neuroprotective compounds in early stage PD patients. Compounds that are ascertained to be effective in the Futility studies will be tested in large double-blinded placebo control studies. The Neurodegeneration and Clinical Trial Groups of the NINDS are requesting applications for new centers to collaborate in clinical trials to test potential neuroprotective agents. We are proposing to become a NET-PD center. The Movement Disorders Center at University of Kentucky serves the greater Lexington area, a primarily urban community and the largely rural, 63-county area of central, eastern, and southeastern Kentucky. In addition, we treat several PD patients (-25) who reside in West Virginia and Southern Ohio. We follow -600 PD patients and we diagnose on average 5 new PD patients per month. Therefore, we expect to be able to enroll 1-2 early stage PD patients per month into the Pilot Trials.