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.

项目摘要 临床核心：摘要 临床核心（CC）支持领先参与者的公园冰河Chiari＆syryomyelia中心（PRCSC） 筛查，注册和同意过程以及临床数据的获取，神经行为 评估（NIH工具箱）和患者报告的结果指标（PROM），包括与健康相关的质量 生命（HRQOL）乐器。这将包括通过临床招募CM的患者 在华盛顿大学/街。路易斯儿童医院/巴恩斯 - 犹太医院，但也通过 多中心公园冰河奇亚里遗传学组。 CC将维护5个关键数据集以支持目标和科学计划，这四个拟议的P01 项目：1）PRCSC多中心注册表； 2）PRCSC的42个中心PCORI支持的随机临床试验 Chiari I型I型畸形的后窝解压缩，有或没有尿道术 （PFD/D试验）； 3）Chiari合并数据库； 4）Park-Reeves Chiari Genetics数据库；和5）P01 前瞻性队列1（9-18岁）和队列2（年龄≥19岁）。所有数据将被编码，匿名化， 上传并使用现有的站立操作程序存储。通过这些和相关的严格 过程，CC将支持PRCSC使用前沿，高质量重新定义CM的总体任务 遗传，放射学和临床数据，并确定CM对脑发育的影响 结果和舞会。 CC的功能是： 1。在PRCSC的各个方面招募和维护研究队列。 2。协调所有项目和核心的所有临床随访，神经行为评估和舞会。 3。向所有PRCSC核心和项目提供临床数据，发展评估和舞会 促进他们的科学计划。 4。通过神经行为评估进行长期，纵向临床随访，舞会和 HRQOL评分以确定CM和SM中的疾病自然病史和治疗耐用性。 5。管理所有PRCSC范围范围临床数据库的工作流程，包括形式的设计，数据输入， 质量控制，以及分析的临床数据集分布到研究人员，项目和核心。 6.让CM社区为PRCSC研究做出贡献，并支持其研究重点。 7.通过培养早期调查员来支持PRCSC任务，以便他们发展成为独立的调查员 有效的CM科学家。