NAPS2 Administrative Core

摘要：NAPS 2管理核心 突触核蛋白病包括帕金森病（PD）、路易体痴呆（DLB）和多发性硬化症。 系统萎缩（MSA）总共折磨着约200万美国人，并导致进行性神经变性 功能丧失导致严重残疾和死亡。快速眼动（REM）睡眠行为障碍 (RBD)通常反映了前驱期突触核蛋白病，并提出了一个机会窗口时，治疗 可以有效地预防或减缓明显的突触核蛋白病的进展。北美前驱期 Synucleinopathy（NAPS）Consortium for RBD成立于2018年，旨在为这种神经保护性疾病做准备。 治疗试验。作为NAPS 1-目前的R34支持的NAPS 1阶段，我们在以下方面非常成功： 吸引北美主要的RBD研究人员和研究群体。国家行动方案1非常富有成效： 到目前为止，已有超过200名参与者在10个地点注册，并完成了标准化， 全面评估。我们建立了与以下方面并行的主要行政程序和流程： NAPS 1作为一个透明、高效的联盟顺利运作。为解决重要 关于前驱突触核蛋白病的生物标志物和表型轨迹的问题，我们提出了第2阶段的 NAPS或NAPS 2，以实现纵向随访和扩大生物标志物收集。NAPS 2将建立和 在国家行动计划1的基础上进行了大幅扩展，建立了8个核心，以支持一个中心研究项目。的 行政核心将正式确定和简化国家行动方案2及其所有 件.目前的网站将在行政核心的保护伞下，因为他们继续进入 国家行动方案2，行政核心将监督所有国家行动方案2之间的活动和互动 件.行政核心的目标是：1）协调所有NAPS 2核心、地点、项目， 人员和资源; 2）促进NAPS内外的沟通; 3）保障数据质量 NAPS联盟的一致性; 4）促进RBD的研究; 5）保证遵守 所需的监管机构。国家行动方案2行政核心将监督以下机构之间的相互关系： 国家行动方案2的组成部分，目标是建立一个高效和富有成效的联合体。通过拟议的目标， 管理核心将使NAPS 2的所有组成部分都能以最佳方式运行，以实现以下目标： 神经保护性临床试验，以减缓或阻止突触核蛋白病的进展。

ABSTRACT: NAPS2 ADMINISTRATIVE CORE Synucleinopathies including Parkinson disease (PD), dementia with Lewy bodies (DLB), and multiple system atrophy (MSA) in aggregate afflict ~2 million Americans, and lead to progressive neurodegeneration and loss of function toward severe disability and death. Rapid eye movement (REM) Sleep Behavior Disorder (RBD) usually reflects a prodromal synucleinopathy, and presents a window of opportunity when treatment may be effective to prevent or slow the progression to overt synucleinopathy. The North American Prodromal Synucleinopathy (NAPS) Consortium for RBD was established in 2018 to prepare for such neuroprotective treatment trials. As NAPS1—the current, R34-supported stage of NAPS—we have been very successful in engaging key RBD researchers and cohorts across North America. NAPS1 has been extremely productive: >200 participants have thus far been enrolled across 10 Sites, and have completed standardized, comprehensive assessments. We established key administrative procedures and processes in parallel with rapid enrollment, and NAPS1 functions smoothly as a transparent, efficient Consortium. To address important questions about biomarkers and phenotypic trajectories in prodromal synucleinopathy, we propose Stage 2 of NAPS, or NAPS2, to enable longitudinal followup and expanded biomarker collection. NAPS2 will build and substantially expand upon NAPS1, to establish 8 Cores that support a central research Project. The Administrative Core will formalize and streamline the administrative tasks across NAPS2 and all its components. The current Sites will come under the umbrella of the Administrative Core as they continue into NAPS2, and the Administrative Core will oversee the activities and interactions between all NAPS2 components. The Aims of the Administrative Core are to 1) coordinate all NAPS2 Cores, Sites, Project, personnel, and resources; 2) facilitate communication within and outside of NAPS; 3) safeguard data quality and consistency across the NAPS Consortium; 4) promote research in RBD; and 5) guarantee compliance with required regulatory bodies. The NAPS2 Administrative Core will oversee the inter-relationships between NAPS2 components with the goal of an efficient and productive Consortium. Through the proposed aims, the Administrative Core will enable all NAPS2 components to perform optimally, toward the goal of neuroprotective clinical trials to slow or stop the progression of synucleinopathies.