NAPS2 Clinical Core

摘要：NAPS2临床核心 北美RBD先兆综合征联盟，阶段2(NAPS2)方案 代表了一个扩大和整合的国际研究联盟，专注于RBD，涉及八个 核心和一个项目。NAPS2旨在大幅和全面地提高纵向 临床和生物标记物(血液、脑脊液、PSG、MRI、DATcan)数据将可用于RBD的研究 内部和外部调查人员，目标是最终促进疾病修改的发展 针对RBD患者的治疗，以延缓或防止显性DLB、PD或MSA的发展。海流 NAPS方案(R34 AG056639，以下简称NAPS1)是一项为期2年的临床试验计划拨款，已成功 注册了200名参与者，拥有PSG认证的RBD，分布在10个地点。一套全面标准化的临床 并对每个参与者进行神经认知测量，包括全国阿尔茨海默氏症 协调中心(NACC)统一数据集版本3、路易体痴呆模块和其他特定模块 与PD和MSA更相关的措施。生物液样本，包括DNA和血浆样本，以及脑脊液样本 子集，已在国家阿尔茨海默氏症中央资料库收集、转移和处理 疾病及相关痴呆(NCRAD)。这些数据有助于关键的遗传和生物流体标记 与RBD有关联，这需要进一步调查。来自NAPS1的PSG数据已经 集中分析，REM睡眠不需要紧张症的量化测量；PSG将被 在NAPS2中进行，关键数据将被上传到国家睡眠研究资源(NSRR)。那里 没有足够的资金在NAPS1的所有地点进行神经成像研究，但MRI和 在几个中心收集的DaTcan成像数据导致了有希望的初步发现并建立了 NAPS2中包含成像的基础；这些扫描将上载到 神经成像(LONI)。NAPS2临床核心计划将建立在NAPS1计划明显成功的基础上，通过 招募一大批RBD参与者(N&>300)，并每年对他们进行全面和 规范的方式。年龄和性别匹配的健康对照的较小队列也将被评估 具体岩心和项目的对比分析。前驱综合征评定量表 还将开发用于RBD相关研究和临床试验的药物。因为预计会有更广泛的 人群筛查和评估，一些新的虚拟和数字措施，选择RBD患者和 控制系统将试行。还将为所有RBD参与者寻求高级尸检指令。《临床》 因此，CORE将获得高质量的临床数据、生物流体样本、PSG数据以及核磁共振和DaTcan 综合成像数据，以供所有核心和项目使用，并与科学界共享，如 并探索其他分析，以期在未来进行修改疾病的临床试验。

ABSTRACT: NAPS2 CLINICAL CORE The North American Prodromal Synculeinopathy Consortium for RBD, Stage 2 (NAPS2) protocol represents an expanded and integrated international research consortium focused on RBD involving eight cores and one project. NAPS2 is designed to substantially and comprehensively increase the longitudinal clinical and biomarker (blood, CSF, PSG, MRI, DaTscan) data that will be available for research of RBD by internal and external investigators, with the goal of ultimately fostering the development of disease-modifying therapies targeting those with RBD to delay or prevent the development of overt DLB, PD or MSA. The current NAPS protocol (R34 AG056639, hereafter NAPS1) is a 2-year clinical trial planning grant that has successfully enrolled >200 participants with PSG-proven RBD across 10 sites. A comprehensive standardized set of clinical and neurocognitive measures is obtained for each participant, and includes the National Alzheimer’s Coordinating Center (NACC) Uniform Data Set Version 3, the Lewy Body Dementia Module and other specific measures more relevant to PD and MSA. Biofluid samples that include DNA and plasma on all, and CSF on a subset, have been collected, transferred and processed at National Centralized Repository for Alzheimer's Disease and Related Dementias (NCRAD). These data contribute to key genetic and biofluid markers that have been associated with RBD and that warrant further investigation. The PSG data from NAPS1 have been analyzed centrally, with REM sleep without atonia quantitative measures now developed; PSGs will be performed in NAPS2 and key data will be uploaded to the National Sleep Research Resource (NSRR). There was insufficient funding to perform neuroimaging studies across all of the sites in NAPS1, but MRI and DaTscan imaging data collected at a few of the centers resulted in promising preliminary findings and establish the basis for the inclusion of imaging in NAPS2; these scans will be uploaded to the Laboratory of Neuroimaging (LONI). The NAPS2 Clinical Core will build on the obvious success of the NAPS1 program by recruiting a large cohort of RBD participants (N>300) and evaluating them annually in a comprehensive and standardized manner. A smaller cohort of age- and sex-matched healthy controls will also be evaluated for comparative analyses in specific Cores and for the Project. The Prodromal Synucleinopathy Rating Scale for use in RBD-related research and clinical trials will also be developed. In anticipation for more widespread population screening and assessment, some novel virtual and digital measures in select RBD patients and controls will be piloted. Advanced directive for autopsy will also be sought for all RBD participants. The Clinical Core will therefore acquire high quality clinical data, biofluid samples, PSG data, and MRI and DaTscan imaging data to be integrated for use in all Cores and the Project and shared with the scientific community, as well as explore additional analyses in anticipation of future disease-modifying clinical trials.