Neuroprotection Studies in PD: A Coordinating Center

神经科学界的一个主要目标是开发神经保护治疗方法，减缓或阻止 慢性神经退行性疾病的进展。在认识生物多样性方面的最新进展 帕金森病（PD）的发病机制，包括相关动物模型的发展，提供了 有机会进行合理的临床试验，以评估神经保护治疗。本申请提出了一种 以学术为基础的临床试验协调中心，以最终确定研究方案，培训临床中心， 并管理所有临床试验数据，维护质量和监管标准，并组织包装和 向临床中心提供实验性治疗。该协调中心将保持 促进统计中心、临床中心、NINDS之间密切合作的行政结构 科学计划人员和NIH监督委员会。其他建议包括一个合理的 选择潜在的神经保护剂的过程，将最大限度地提高成功选择的可能性， 初步安全性耐受性和剂量范围研究设计，以及大型简单临床试验设计， 评估干预措施对PD进展的影响。新出现的姿势不稳， PD的临床体征和发病率和死亡率的可靠预测因子，被提议作为主要的临床指标。 衡量疾病进展。除了作为PD的核心临床特征和可靠的 可测量的姿势不稳定固有地抵抗当前PD的短期症状改善 药物治疗提出了三种潜在的神经保护化合物，尽管最终选择的是 将根据与其他调查人员的讨论确定化合物。适当的研究基础设施 结合相关的科学专业知识，进行拟议的神经保护研究， 警局的探员

A major goal of the neuroscience community is to develop neuroprotective treatments that will slow or forestall the progression of chronic neurodegenerative diseases. Recent advances in the understanding of the pathogenesis of Parkinson's disease (PD),including the development of relevant animal models, provide the opportunity for rationale clinical trials to assess neuroprotective treatments. This application proposes an academically based clinical trials coordination center to finalize study protocols, train clinical centers, receive and manage all clinical trial data, maintain quality and regulatory standards, and organize packaging and delivery of experimental treatments to clinical centers. This Coordination Center will maintain an administrative structure that fosters close collaboration among, the Statistical center, clinical centers, NINDS Scientific Program personnel and an NIH Oversight Committee. Additional proposals include a rational process to select potential neuroprotective agents that will maximize the likelihood of successful choices, designs for pilot safety tolerability and dosage ranging studies, and the design for a large simple clinical trial to assess the impact of interventions on PD progression. The new onset of postural instability, one of the cardinal clinical signs of PD and a robust predictor of morbidity and mortality, is proposed as the primary clinical measure of disease progression. In addition to being a core clinical feature of PD and being reliably measurable, postural instability is inherently resistant to short-term symptomatic amelioration by current PD medications. Three potential neuroprotective compounds are proposed, although the final selection of compounds will be based on deliberations with other investigators. An appropriate research infrastructure combined with relevant scientific expertise is in place to conduct the proposed studies of neuroprotective agents in PD.