Additional Informatics support on U54 Clinical Core

项目摘要--总体 堪萨斯州PKD研究和翻译核心中心(RTCC)由一个行政部门组成 核心和三个创新的生物医学研究核心：核心1：生物标志物、生物材料和 细胞模型；核心2：啮齿动物模型；核心3：临床研究。堪萨斯州RTCC还提供了一个 教育丰富计划和与KU合作的内部试点助学金计划 癌症中心。PKD中心的使命是通过提供这两项服务来促进PKD研究 创新和必要的试剂、生物材料和为国家PKD研究界提供的服务 为了促进我们对疾病机制的理解，生物标志物和 治疗目标和临床试验的发展，以改善患者的结果。具体的 堪萨斯州PKD RTCC的目标是：目标1)通过开发 创新的生物医学研究核心。这将通过发展专业知识来实现， 将最好的新技术引入PKD研究所需的试剂和资源 更大的PKD研究界的需求；利用现有的机构共享资源 扩展我们的能力、效率和效果；并通过开发工具来支持临床 研究和建立区域和国家合作，以发展多机构 ADPKD临床试验网络。目标2)开发熟练、协作、多学科的基础 科学和临床PKD研究调查员基地。这将通过建立在我们的 广泛的多学科研究基础，并继续从外部招聘新的调查人员 PKD领域通过提供创新的试点赠款计划，将吸引新的研究人员进入该领域， 通过在全国范围内为新的调查人员提供核心服务的研究支持，新的 技术、教育机会以及开放和协作的研究环境 帮助他们取得成功。目的3)通过教育方案加强PKD研究环境，以 支持正在进行的项目并开发新的研究方向。研究环境将是 通过研讨会计划、暑期学生充实计划、PKD实验室会议、 PKD方法研讨会、研究方法和先进技术计划、专业人员 发展研讨会系列，以患者为中心的活动，并通过参与整个财团 年度研讨会。目标4)在区域和国家发展伙伴关系，并建立团队以 作为更大的PKD研究社区的资源。机构间伙伴关系将 提供地方和国家基础设施支持、新的研究技术、增加 参与临床试验的患者和开发新研究的研究合作者 通过吸引来自其他领域的研究人员从合作网站进入PKD研究的机会 在PKD研究联盟内并与全国合作机构合作，扩大 任何一家机构都无法提供的机会。 项目摘要-临床研究核心 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。

Project Summary – Overall The Kansas PKD Research and Translation Core Center (RTCC) is comprised of an Administrative Core and three innovative Biomedical Research Cores: Core 1: Biomarkers, Biomaterials, and Cellular Models; Core 2: Rodent Models Core 3: Clinical Research. The Kansas RTCC also offers an Educational Enrichment Program and an internal Pilot Grant Program in partnership with the KU Cancer Center. The mission of the PKD Center is to promote PKD research by providing both innovative and essential reagents, biomaterials, and service to the national PKD research community for the advancement of our understanding of disease mechanisms, the discovery of biomarkers and therapeutic targets and the development of clinical trials to improve patient outcomes. The Specific Aims of the Kansas PKD RTCC are: Aim 1) To support PKD researchers nationally by developing innovative biomedical research cores. This will be accomplished by developing the expertise, reagents and resources required to bring the best new technologies to PKD research to meet the needs of the greater PKD research community; by leveraging existing institutional shared resources to expand our capability, efficiency, and effectiveness; and by developing tools to support clinical research and establish collaborations both regionally and nationally to develop a multi-institutional ADPKD clinical trials network. Aim 2) To develop a skilled, collaborative, multidisciplinary basic science and clinical PKD research investigator base. This will be accomplished by building on our broad multidisciplinary research base and continuing to recruit new investigators from outside the PKD field by offering an innovative pilot grant program that will attract new investigators to the field, and by providing new investigators, nationally, with research support for core services, new technologies, educational opportunities, and an open and collaborative research environment that will help them succeed. Aim 3) To enhance the PKD research environment with educational programs to support ongoing projects and to develop new research directions. The research environment will be enhanced through a seminar program, Summer Student Enrichment Program, PKD lab meetings, PKD methods workshops, Research Methods and Advanced Technologies Program, professional development seminar series, patient-oriented activities, and by participating in the consortium-wide annual symposium. Aim 4) To develop partnerships regionally and nationally and to build teams to serve as a resource for the greater PKD research community. Inter-institutional partnerships will provide local and national infrastructure support, new research technologies, increased numbers of patients involved in clinical trials, and research collaborators for the development of new research opportunities by attracting investigators from other fields into PKD research from collaborating sites within the PKD Research Consortium and with cooperating institutions nationwide, broadening opportunities beyond what any single institution can provide. Project Summary - Clinical Research Core 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.