Biomedical Research Core 3 - Clinical Research Core

临床研究核心-项目总结/摘要 PKD的首要挑战是如何将基础科学认识的许多进展转化为 PKD的病理生理学研究投入到有效新药的开发中。具体挑战包括： 可用于临床试验的患者人数减少，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，以协调不同的数据源，患者参与有限 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 全国范围内PKD研究人员从T1到T4的翻译谱研究。这将是 通过开发多种工具和资源， 研究社区。具体目标是：1）在中部地区建立一个学术中心联盟 和西部地区，形成ADPKD临床试验网络; 2）扩大并继续遵循纵向 早期ADPKD年轻患者和对照受试者队列，并收集生物标志物的生物标本 发现; 3）为RedCAP中的PKD研究创建标准化的数据收集工具，并为 自动映射到配准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级，并利用此为研究提供信息 在PKD社区的战略和方向。

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.