Clinical Core

临床核心

• 批准号: 10629119
• 负责人: David Delmar Limbrick
• 金额: $ 20.78万
• 依托单位: WASHINGTON UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-07-01 至 2028-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10629119
• 关键词: Adolescent; Brain; Cervical; Clinic; Clinical; Clinical Data; Code; Cohort Studies; Collaborations; Communities; Consent; Data; Data Set; Databases; Development; Diffusion Magnetic Resonance Imaging; Elements; Enrollment; Fostering; Foundations; Genetic; Genetic Databases; Goals; Hospitals; Individual; International; Investigation; Lead; Leadership; Longterm Follow-up; Mission; Neurological outcome; Outcome Measure; Participant; Patient Outcomes Assessments; Patient advocacy; Patient-Focused Outcomes; Patients; Pediatric Hospitals; Posterior Fossa; Predictive Value; Procedures; Process; Prospective cohort; Quality Control; Radiology Specialty; Registries; Reproducibility; Research; Research Personnel; Research Priority; Resources; Scientific Advances and Accomplishments; Scientific Inquiry; Scientist; Services; Site; Spinal Cord Diseases; Support Groups; Syringomyelia; Telephone; United States National Institutes of Health; Universities; Washington; aged; clinical database; clinical practice; cognitive development; cohort; comorbidity; design; disease natural history; eligible participant; follow up assessment; follow-up; follower of religion Jewish; health related quality of life; innovation; instrument; malformation; neurobehavioral; randomized, clinical trials; recruit; screening; treatment and outcome; video visit

PROJECT SUMMARY Clinical Core: Abstract The Clinical Core (CC) supports the Park-Reeves Chiari & Syringomyelia Center (PRCSC) by leading participant screening, enrollment, and consenting processes as well as the acquisition of clinical data, neurobehavioral assessments (NIH Toolbox), and patient-reported outcome measures (PROM), including health-related quality of life (hrQOL) instruments. This will include patients with CM recruited for participation through the clinical practices at Washington University/St. Louis Children’s Hospital/Barnes-Jewish Hospital but also through the multicenter Park-Reeves Chiari Genetics Group. The CC will maintain the 5 key datasets to support the aims and scientific initiatives all four proposed P01 Projects: 1) PRCSC multicenter registry; 2) PRCSC’s 42-center PCORI-supported randomized clinical trial Posterior Fossa Decompression With or Without Duraplasty for Chiari Type I Malformation with Syringomyelia (the PFD/D Trial); 3) Chiari Comorbidites database; 4) Park-Reeves Chiari Genetics database; and 5) P01 Prospective Cohort 1 (aged 9-18 years) and Cohort 2 (aged ≥19 years). All data will be coded, anonymized, uploaded, and stored using existing Standing Operating Procedures. Through these and related rigorous processes, the CC will support the PRCSC’s overarching mission to redefine CM using leading-edge, high quality genetic, radiological, and clinical data, and to determine the effect of CM on brain development, neurological outcomes, and PROM. The functions of the CC are to: 1. Recruit and maintain the study cohorts for all aspects of PRCSC. 2. Coordinate all clinical follow-up, neurobehavioral assessments, and PROM across all Projects and Cores. 3. Provide clinical data, developmental assessments, and PROM to all PRCSC Cores and Projects to facilitate their scientific initiatives. 4. Conduct long-term, longitudinal clinical follow-up with neurobehavioral assessment, and PROM and hrQOL scoring to determine disease natural history and treatment durability in CM and SM. 5. Manage the workflow of all PRCSC-wide clinical databases, including the design of forms, data entry, quality control, and the distribution of analyzed clinical data sets to investigators, Projects, and Cores. 6. Engage the CM community to contribute to PRCSC studies and support its research priorities. 7. Support the PRCSC mission by nurturing early-stage investigators so that they develop into independent, effective CM scientists.

项目概要 临床核心：摘要 临床核心 (CC) 由主要参与者支持 Park-Reeves Chiari 和脊髓空洞症中心 (PRCSC) 筛选、登记和同意过程以及临床数据、神经行为学数据的获取 评估（NIH 工具箱）和患者报告的结果测量（PROM），包括健康相关质量 这将包括招募参与临床的 CM 患者。 华盛顿大学/圣路易斯儿童医院/巴恩斯犹太医院的实践，也通过 多中心 Park-Reeves Chiari 遗传学小组。 CC 将维护 5 个关键数据集，以支持所有四个拟议 P01 的目标和科学举措 项目：1) PRCSC 多中心注册；2) PRCSC 的 42 中心 PCORI 支持的随机临床试验 后颅窝减压联合或不联合硬脑膜成形术治疗 Chiari 型 I 型畸形伴脊髓空洞症 （PFD/D 试验）；3) Chiari 合并症数据库；4) Park-Reeves Chiari Genetics 数据库；以及 5) P01 预期队列 1（年龄 9-18 岁）和队列 2（年龄 ≥19 岁）所有数据都将进行编码、匿名化处理。 通过这些和相关的严格操作程序上传和存储。 CC 将支持 PRCSC 的总体使命，即利用领先的高质量重新定义 CM 遗传、放射学和临床数据，并确定 CM 对大脑发育、神经系统的影响 结果和 PROM。 CC 的职能是： 1. 招募和维护 PRCSC 各个方面的研究队列。 2. 协调所有项目和核心的所有临床随访、神经行为评估和 PROM。 3. 向所有 PRCSC 核心和项目提供临床数据、发育评估和 PROM，以 促进他们的科学举措。 4. 进行长期、纵向的临床随访，包括神经行为评估、PROM 和 hrQOL 评分以确定 CM 和 SM 的疾病自然史和治疗持久性。 5. 管理所有 PRCSC 范围内的临床数据库的工作流程，包括表格设计、数据录入、 质量控制，以及将分析后的临床数据集分发给研究者、项目和核心。 6. 让 CM 界参与 PRCSC 研究并支持其研究重点。 7. 支持 PRCSC 的使命，培养早期调查人员，使其发展为独立的、 有效的 CM 科学家。