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: 10475048
• 负责人: Alan S Yu
• 金额: $ 27.13万
• 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/10475048
• 关键词: Biomedical Research; Clinical Research; Kansas; Research; Translations

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面临的首要挑战是如何将基础科学理解中的许多进展转化为 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池缩小，早期疾病缺乏生物标志物，缺乏广泛的 可用标准化的数据模型来协调不同的数据源，而患者的参与度有限 以及他们的家人对研究问题进行优先排序。临床研究核心的目标是促进 由全国范围内的PKD调查人员对从T1到T4的翻译范围进行研究。这将是 通过开发多种工具和资源实现，这些工具和资源将免费提供给 研究社区。具体目标是：1)在整个中环建立一个学术中心联合体 与西部地区形成ADPKD临床试验网络；2)扩大并继续遵循纵向 青年早期ADPKD患者和对照组的队列和收集生物标记物 发现；3)为RedCap的PKD研究创建一个标准化的数据收集工具，并为 自动映射到注册研究所需的标准数据格式；以及4)使用我们的患者 参与资源，以确定患者对研究问题的优先顺序，并利用此资源为研究提供信息 PKD社区的战略和方向。