Access for All in ALS (ALL ALS) East Clinical Coordinating Center

修改项目摘要/摘要部分 为了解决肌萎缩侧索硬化症（ALS）研究中存在的挑战，我们建议建立一个新的国家肌萎缩侧索硬化症（ALS）临床研究联盟，即ALS（ALL ALS）联盟。这个新的ALL ALS联盟将实施加速获得ALS关键疗法法案（ACT for ALS）公私合作伙伴关系（PPP）的建议，以加快ALS药物的开发。ALL ALS计划将有两个协调中心（ALL ALS东部协调中心和ALL ALS西部协调中心），共同管理和监督美国和波多黎各的34个临床中心。具体而言，ALL ALS东部协调中心将位于马萨诸塞州总医院的神经临床研究所（NCRI），并将管理15个临床研究中心。MGH NCRI带来了作为协调中心的丰富经验，并与行业，学术研究人员和非营利研究团体合作，在高质量，多地点ALS观察性研究，生物库项目和临床试验方面处于领先地位。新的ALL ALS联盟将提供大规模，集中和易于访问的基础设施，用于收集和存储广泛的数据，包括纵向收集的临床测量和生物流体1）ALS患者，2）患有肌萎缩侧索硬化症（ALS）的遗传风险的人，和3）健康对照。为了促进人群的广泛代表性，将从远程和亲自访问中收集数据，许多临床研究中心将位于少数民族代表性不足的地理区域。此外，为便利今后的研究和合作，将在一个单一的中央数据门户收集和统一数据。

Modified Project Summary/Abstract Section To address existing challenges in Amyotrophic Lateral Sclerosis (ALS) research, we propose creation of a new national Amyotrophic Lateral Sclerosis (ALS)clinical research consortium, the Access for All in ALS (ALL ALS) Consortium. This new ALL ALS Consortium will operationalize recommendations of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) Public Private Partnership (PPP) to hasten drug development for ALS. The ALL ALS Program will have two coordinating centers (the ALL ALS East Coordinating Center and the ALL ALS West Coordinating Center) which together will administer and oversee 34 clinical sites across the United States and Puerto Rico. Specifically, the ALL ALS East Coordinating Center will be located at the Neurological Clinical Research Institute (NCRI) of Massachusetts General Hospital, MA, and will administer 15 clinical sites. MGH NCRI brings extensive experience functioning as coordination center and leading scores of high-quality, multi-site ALS observational studies, biobanking projects and clinical trials, in partnership with industry, academic investigators, and not-for-profit research groups. The new ALL ALS Consortium will provide large-scale, centralized, and readily accessible infrastructure for collection and storage of a wide range of data, including longitudinally collected clinical measures and biofluids from 1) people living with ALS, 2) people at genetic risk for developing Amyotrophic Lateral Sclerosis (ALS), and 3) healthy controls. To foster wide representation across populations, data will be collected from both remote and in person visits, and many clinical sites will be located in geographical areas with underrepresented minorities. Furthermore, to facilitate future research and collaboration, data will be collected and harmonized in a single central data portal.