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

基本信息

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
项目类别：
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

项目摘要

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.

肌萎缩性侧索硬化症（ALS）是一种快速进行的，最终是致命的，神经退行性疾病的，在美国（美国）约有32,000例。估计终身风险为1/400，每年平均诊断出5,000个新病例。鉴于将实验室结果转化为临床有效疗法的许多失败，高质量的临床注释的产生来自潜水员ALS同伙的纵向患者衍生的生物样本以及导致的下游分析预计将加速有效的，有效的新药，以延长与Als的私人合作伙伴（PPP）的发展，以延长生命，以延长延长生命的生命（PPP）。药物以延长与ALS的人们的生命。为了实现新成立的ALS公共私人合作伙伴法案（PPP）的研究目标，以加速药物开发，我们建议创建一个新的国家ALS临床研究联盟，这是ALS（ALS）联盟中所有人的访问。所有ALS将设有2个协调中心（所有ALS East协调中心和所有ALS West协调中心），该中心将共同管理并监督美国和波多黎各的34个临床场所。全ALS West协调中心将位于圣约瑟夫医院和医疗中心的Barrow神经学研究所。在我们的目标ALS自然历史研究和财团的持续成功的基础上，所有ALS West协调中心将继续管理目前为目标ALS自然历史研究做出贡献的临床部位，以及其他10个新的临床部位。新的所有ALS财团将提供大规模，集中且易于访问的基础架构，以收集和存储广泛的数据：其中包括纵向收集的临床指标和来自1）患有ALS的人的纵向临床措施和生物流体，2）患有ALS风险的人，以及3）健康控制。为了促进人群之间的广泛代表，将从遥远和亲自访问中收集数据，许多临床地点将位于代表不足的少数民族的地理区域。此外，为了促进未来的研究和协作，将在单个中央数据门户中收集和协调数据。