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，在美国约有 32,000 例病例，估计终生风险为 1/400，美国每年平均诊断出 5,000 例新病例。鉴于在将实验室结果转化为临床有效疗法方面存在大量失败，从不同的 ALS 队列中生成高质量、有临床注释的纵向患者生物样本并进行下游分析。预计将加速开发有效的新药以延长 ALS 患者的生命可以利用公私合作伙伴关系 (PPP) 快速推进监管科学和科学研究，以支持和加速开发有效的新药以延长 ALS 患者的生命患有 ALS 的人。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，我们建议创建一个新的国家 ALS 临床研究联盟，即“为所有人提供 ALS (ALL ALS) 联盟”。 ALL ALS 将设有 2 个协调中心（ALL ALS 东部协调中心和 ALL ALS 西部协调中心），它们将共同管理和监督美国和波多黎各的 34 个临床中心。 ALL ALS 西部协调中心将设在 ALL ALS 西部协调中心。圣约瑟夫医院和医疗中心的巴罗神经学研究所以我们的目标 ALS 自然史研究和联盟的持续成功为基础，ALL ALS 西部协调中心将继续管理临床。目前正在为 Target ALS 自然历史研究做出贡献的站点，以及另外 10 个新的临床站点。新的 ALL ALS 联盟将提供大规模、集中且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数据和生物体液，这些数据来自 1) ALS 患者，2) 有患 ALS 风险的个人。开发 ALS，以及 3) 健康对照，以促进跨人群的广泛代表性，将从远程和亲自访问中收集数据，并且许多临床地点将位于少数族裔代表性不足的地理区域。协作、数据将在一个中央数据门户中收集和协调。