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中的总体挑战是如何翻译对基本科学理解的许多进步 PKD的病理生理发展为有效新药的发展。具体挑战包括 缩小可用于临床试验的患者池，早期疾病缺乏生物标志物，缺乏广泛的 可用的，标准化的数据模型，以协调各种数据源和患者的参与度有限 以及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调查人员从T1到T4的翻译光谱进行了研究。这将是 通过开发多种工具和资源来实现，这些工具和资源将自由地提供给 研究社区。具体目的是：1）在中央建立一个学术中心财团 和西方地区形成ADPKD临床试验网络； 2）扩展并继续遵循纵向 年轻患者患有早期ADPKD和对照对象的队列，并收集生物标志物的生物测量 发现; 3）创建用于REDCAP和软件的PKD研究的标准数据收集工具 自动映射到注册研究所需的标准数据格式； 4）使用我们的病人 参与资源以识别患者的研究问题的优先级，并使用此问题来为研究提供信息 PKD社区的策略和方向。