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

修改后的项目摘要/摘要部分 为了应对肌萎缩侧索硬化症(ALS)研究中存在的挑战，我们建议成立一个新的全国性肌萎缩侧索硬化症(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)公私伙伴关系(PPP)的建议，以加速ALS的药物开发。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)，这两个中心将共同管理和监督美国和波多黎各的34个临床地点。具体地说，ALL ALS东部协调中心将设在马萨诸塞州马萨诸塞州总医院的神经临床研究所(NCRI)，并将管理15个临床地点。MGH NCRI作为协调中心提供了丰富的经验，并与业界、学术研究人员和非营利性研究团体合作，领导了数十项高质量的多地点ALS观察研究、生物库项目和临床试验。新的ALL ALS联盟将为收集和存储广泛的数据提供大规模、集中化和易于访问的基础设施，包括从1)ALS患者、2)肌萎缩侧索硬化症(ALS)遗传风险患者和3)健康对照收集的纵向临床测量和生物液。为了促进广泛的人口代表性，将从远程和面对面的访问中收集数据，许多诊所将位于少数民族代表不足的地理区域。此外，为便利今后的研究和协作，将在一个单一的中央数据门户中收集和统一数据。

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.