Biomedical Research Core 3 - Clinical Research Core

生物医学研究核心 3 - 临床研究核心

• 批准号: 10686076
• 负责人: Alan S Yu
• 金额: $ 26.65万
• 依托单位: UNIVERSITY OF KANSAS MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-07-15 至 2025-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10686076
• 关键词: Address; Advocacy; Age; Autosomal Dominant Polycystic Kidney; Belief; Biological Markers; Biomedical Research; Blood specimen; Caregivers; Clinical Research; Clinical Trials; Clinical Trials Network; Collection; Common Data Element; Communities; Computer software; Consensus; Data Collection; Data Sources; Databases; Development; Disease; Disease Progression; Disease model; Enrollment; Environment; Family; Family member; Functional disorder; Goals; Health Personnel; Healthcare; Individual; Industry; Infrastructure; International; Kansas; Longitudinal cohort; Maps; Natural History; Outcome; Outcome Measure; Patient-Focused Outcomes; Patients; Pharmacotherapy; Phenotype; Prognostic Marker; Protocols documentation; Race; Registries; Research; Research Personnel; Research Priority; Resources; Risk; Site; Source; Standardization; Terminology; Translating; Translations; Update; Urine; Work; biomarker discovery; clinical development; cohort; compare effectiveness; data format; data harmonization; data integration; data modeling; data standards; design; diverse data; empowerment; exosome; experience; information gathering; instrument; multiple data sources; novel; novel therapeutics; patient engagement; patient oriented; preference; programs; recruit; shared decision making; socioeconomics; tolvaptan; tool; translational pipeline; trial design; trial readiness; urinary

Clinical Research Core - Project Summary/Abstract The overarching challenge in PKD is how to translate the many advances in basic scientific understanding of the pathophysiology of PKD into the development of effective new drugs. Specific challenges include a shrinking pool of patients available for clinical trials, lack of biomarkers in early disease, lack of widely- available, standardized data models to harmonize diverse data sources, and limited engagement of patients and their families in prioritizing research questions. The goal of the Clinical Research Core is to facilitate research across the translational spectrum from T1 to T4 by PKD investigators nationwide. This will be achieved through the development of multiple tools and resources that will be made freely available to the research community. The specific aims are: 1) to create a consortium of academic centers across the Central and Western regions to form an ADPKD clinical trial network; 2) to expand and continue to follow a longitudinal cohort of young patients with early ADPKD and control subjects and collect biospecimens for biomarker discovery; 3) create a standardized data collection instrument for PKD studies in RedCAP and software for automated mapping into a standard data format required for registration studies; and 4) use our patient engagement resource to identify patients' priorities for research questions and use this to inform research strategy and directions in the PKD community.

临床研究核心 - 项目摘要/摘要 PKD 的首要挑战是如何将基础科学理解中的许多进步转化为 将 PKD 的病理生理学转化为有效新药的开发。具体挑战包括 可用于临床试验的患者数量不断减少，缺乏早期疾病的生物标志物，缺乏广泛的 可用的标准化数据模型来协调不同的数据源，并且患者的参与有限 和他们的家人优先考虑研究问题。临床研究核心的目标是促进 全国 PKD 研究人员进行从 T1 到 T4 的转化谱研究。这将是 通过开发多种工具和资源来实现，这些工具和资源将免费提供给 研究社区。具体目标是：1）创建一个跨中央的学术中心联盟 与西部地区组建ADPKD临床试验网络； 2）扩大并继续沿纵向 早期 ADPKD 年轻患者和对照受试者队列并收集生物样本作为生物标志物 发现; 3) 在 RedCAP 和软件中创建用于 PKD 研究的标准化数据收集工具 自动映射为注册研究所需的标准数据格式； 4）使用我们的病人 参与资源，以确定患者对研究问题的优先顺序，并利用其为研究提供信息 PKD 社区的战略和方向。