NAPS2 Administrative Core

摘要：NAPS2管理核心 并核病包括帕金森病(PD)、路易体痴呆(DLB)和多发性 系统萎缩(MSA)总共困扰着大约200万美国人，并导致进行性神经变性 以及严重残疾和死亡的功能丧失。快速眼动(REM)睡眠行为障碍 (RBD)通常反映一种前驱综合征，并在治疗时出现机会之窗。 可能有效地预防或延缓进展为临床上的联核症。北美的前驱生物 RBD突触核病(NAPS)联盟于2018年成立，为此类神经保护做准备 治疗试验。作为NAPS1-目前R34支持的NAP阶段-我们在以下方面非常成功 让北美各地的关键RBD研究人员和队列参与进来。NAPS1非常富有成效： 到目前为止，已有200名参与者在10个地点注册，并完成了标准化、 全面评估。我们建立了关键的行政程序和流程，同时 快速注册，NAPS1作为一个透明、高效的联盟顺利运作。要解决重要问题 关于前驱联核病的生物标记物和表型轨迹的问题，我们建议第二阶段 NAPS，或NAPS2，以实现纵向跟踪和扩大生物标记物收集。NAPS2将构建和 在NAPS1的基础上大幅扩展，建立支持中央研究项目的8个核心。这个 管理核心将正式确定并简化NAPS2及其所有 组件。目前的网站将纳入行政核心的保护伞，因为它们将继续进入 NAPS2，管理核心将监督所有NAPS2之间的活动和交互 组件。管理核心的目标是1)协调所有NAPS2核心、站点、项目、 人员、资源；2)促进国家行动方案内外沟通；3)保障数据质量 和整个国家行动方案联盟的一致性；4)促进对RBD的研究；和5)保证遵守 所需的监管机构。NAPS2管理核心将监督以下各项之间的相互关系 NAPS2组件，目标是建立一个高效、多产的联盟。通过拟议的目标， 管理核心将使所有NAPS2组件能够以最佳方式运行，从而实现 延缓或阻止联体核病进展的神经保护性临床试验。

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.