Neuroprotection Exploratory Trials in PD (NET-PD)

描述(申请人提供)：帕金森病(PD)是一种缓慢但无情的进行性神经退行性疾病，导致临床症状随时间恶化。抗帕金森病药物有效地改善了大多数患者的大部分主要帕金森症状和体征。然而，传统的药物治疗不能改变运动功能的进行性恶化，随着时间的推移，功能残疾会恶化。因此，迫切需要能够减缓临床进展、促进受损神经元修复、恢复或增强多巴胺(DA)系统残留部分功能的疾病修饰药物。由于疾病的进展通常是缓慢的，使用针对多种致病机制的化合物可以在相对较长的时间内实施治疗。Net-PD多中心试验旨在测试潜在的神经保护化合物在早期PD患者中的疗效。在无效性研究中被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的神经退行性变和临床试验小组正在申请新的中心，以合作进行临床试验，以测试潜在的神经保护剂。我们计划成为Net-PD中心。肯塔基大学的运动障碍中心为大列克星敦地区提供服务，该地区主要是城市社区，以及肯塔基州中部、东部和东南部的63个县的农村地区。此外，我们还治疗居住在西弗吉尼亚州和俄亥俄州南部的几名PD患者(-25岁)。我们跟踪了600名PD患者，平均每个月诊断出5名新的PD患者。因此，我们希望每个月能够招募1-2名早期PD患者参加试点试验。

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.