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

ALS 所有人 (ALL ALS) 西部临床协调中心

• 批准号: 10878596
• 负责人: ROBERT P BOWSER
• 金额: $ 1673.35万
• 依托单位: ST. JOSEPH'S HOSPITAL AND MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-25 至 2024-09-24
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10878596
• 关键词: ALS patients; Acceleration; Advisory Committees; Amendment; Amyotrophic Lateral Sclerosis; Annual Reports; Autopsy; Biological Markers; Blood; Brain; Budgets; Clinic; Clinic Visits; Clinical; Clinical Data; Clinical Research; Clinical Trials; Collaborations; Collection; Common Data Element; Communication; Communities; Community Care Networks; Contracts; Country; Critical Pathways; DNA Sequence Alteration; Data; Databases; Deposition; Development; Diagnosis; Digital biomarker; Disease; Documentation; Educational workshop; Ensure; Equipment and supply inventories; Ethics; Ethnic Population; Failure; Family; Fostering; Foundations; Funding; Funding Agency; Future; Generations; Genome; Geographic Locations; Geography; Goals; Grant; Healthcare; Home; Hospitals; Human Resources; Image; Individual; Industry; Infrastructure; Institutional Review Boards; International; Laboratories; Leadership; Life; Link; Location; Magnetic Resonance Imaging; Manuals; Measures; Medical center; Metadata; Monitor; National Institute of Neurological Disorders and Stroke; Natural History; Neurodegenerative Disorders; Neurologic; New York; Nonprofit Organizations; Online Systems; Onset of illness; Participant; Patient Participation; Patients; Peer Review; Persons; Phase; Population; Population Heterogeneity; Procedures; Protocols documentation; Puerto Rico; Quality Control; Records; Reporting; Request for Applications; Research; Research Personnel; Research Project Grants; Respiratory physiology; Review Committee; Risk; Route; Rural; Rural Community; Rural Population; Saliva; Sampling; Schedule; Science; Scientist; Shipping; Site; Specimen; Speech; Spinal Cord; Spirometry; Structure; Subjects Selections; Symptoms; System; Tissue Banks; Tissue Procurements; Tissue Sample; Tissues; Training; Training Activity; Translating; Underrepresented Minority; United States; United States National Institutes of Health; Vendor; Vial device; Visit; amyotrophic lateral sclerosis therapy; biobank; clinical outcome measures; clinical research site; cohort; data curation; data exchange; data integrity; data management; data modeling; data portal; data sharing; data submission; design; drug development; effective therapy; end stage disease; ethnic diversity; experience; genome sequencing; genomic data; human error; lifetime risk; meetings; member; mutation carrier; neuroimaging; novel; novel therapeutics; operation; outreach; outreach program; patient advocacy group; programs; public-private partnership; recruit; research and development; research study; response; rural patients; success; transcriptome sequencing; transcriptomics; whole genome

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, ultimately fatal, neurodegenerative disease with approximately 32,000 cases in the United States (US). With an estimated lifetime risk of 1/400, an average of 5,000 new US cases are diagnosed every year. Given the numerous failures in translating laboratory results into clinically effective therapies, the generation of high quality, clinically annotated longitudinal patient-derived biosamples from diverse ALS cohorts and resulting downstream analyses are expected to accelerate the development of effective, new drugs to extend life for people living with ALS A public private partnership (PPP) can be leveraged to rapidly advance regulatory science and scientific research to support and accelerate development of effective new drugs to extend life for people living with ALS. To operationalize the research objectives of the newly formed ACT for ALS Public Private Partnership (PPP) to accelerate drug development, we propose creation of a new national ALS clinical research consortium, the Access for All in ALS (ALL ALS) Consortium. ALL ALS will have 2 coordinating centers (the ALL ALS East Coordinating Center and the ALL ALS West Coordinating Center) which will together administer and oversee 34 clinical sites across the United States and Puerto Rico. The ALL ALS West Coordinating Center will be based at the Barrow Neurological Institute at St Joseph’s Hospital and Medical Center. Building upon the ongoing success of our Target ALS natural history study and consortium, the ALL ALS West Coordinating Center will continue to administer clinical sites currently contributing to the Target ALS natural history study, along with 10 additional new clinical sites. The new ALL ALS Consortium will provide large-scale, centralized, and readily accessible infrastructure for collection and storage of a wide range of data: these include longitudinally collected clinical measures and biofluids from 1) people living with ALS, 2) individuals at risk for developing 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 under-represented minorities. Furthermore, to facilitate future research and collaboration, data will be collected and harmonized in a single central data portal.

肌萎缩侧索硬化症（ALS）是一种进展迅速、最终致命的神经退行性疾病，在美国（US）约有32，000例。估计终生风险为1/400，美国每年平均诊断出5,000例新病例。考虑到将实验室结果转化为临床有效疗法的众多失败，预期从不同ALS群组产生高质量的、临床注释的纵向患者来源的生物样品以及由此产生的下游分析将加速开发有效的，延长ALS患者生命的新药公私合作伙伴关系（PPP）可用于快速推进监管科学和科学研究，以支持和加速有效新药的开发，从而延长ALS患者的生命。 为了实现新成立的ACT for ALS公私合作伙伴关系（PPP）的研究目标，以加速药物开发，我们建议建立一个新的国家ALS临床研究联盟，即ALS（ALL ALS）联盟。 所有ALS将有2个协调中心（所有ALS东部协调中心和所有ALS西部协调中心），共同管理和监督美国和波多黎各的34个临床研究中心。 所有ALS西部协调中心将设在圣约瑟夫医院和医疗中心的巴罗神经学研究所。在我们的目标ALS自然史研究和联盟持续取得成功的基础上，ALL ALS西部协调中心将继续管理目前为目标ALS自然史研究做出贡献的临床站点，沿着10个新的临床站点。 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：这些数据包括纵向收集的临床测量和生物流体，来自1）ALS患者，2）有发展ALS风险的个体，以及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。