Additional informatics support on U54 Clinical Core

U54 临床核心的附加信息学支持

• 批准号: 10685724
• 负责人: DARREN P. WALLACE
• 金额: $ 2.15万
• 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/10685724
• 关键词: Autosomal Dominant Polycystic Kidney; Basic Science; Biocompatible Materials; Biological Markers; Biomedical Research; Cancer Center; Cell model; Clinical; Clinical Research; Clinical Trials; Clinical Trials Network; Collaborations; Communities; Computer software; Data Collection; Data Sources; Development; Disease; Educational workshop; Effectiveness; Environment; Family; Functional disorder; Goals; Grant; Informatics; Infrastructure; Institution; Interdisciplinary Study; Kansas; Longitudinal cohort; Methods; Mission; Patient-Focused Outcomes; Patients; Reagent; Research; Research Methodology; Research Personnel; Research Priority; Research Support; Resource Sharing; Resources; Rodent Model; Series; Services; Site; Technology; Translating; Translations; base; biomarker discovery; clinical development; data format; data modeling; data standards; diverse data; improved; innovation; instrument; inter-institutional; meetings; multidisciplinary; new technology; novel therapeutics; patient engagement; patient oriented; programs; recruit; summer student; symposium; therapeutic target; tool; translational pipeline

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.

项目摘要 - 总体 堪萨斯州PKD研究与翻译核心中心（RTCC）已完成管理 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也提供 与KU合作，教育丰富计划和内部试点赠款计划 癌症中心。 PKD中心的任务是通过提供两者来促进PKD研究 为国家PKD研究界的创新和基本试剂，生物材料和服务 为了促进我们对疾病机制的理解，生物标志物的发现和 治疗靶标和临床试验的发展，以改善患者预后。具体 堪萨斯州PKD RTCC的目标是：目标1）通过开发全国范围内的PKD研究人员 创新的生物医学研究核心。这将通过发展专业知识来实现 将最佳新技术带入PKD研究所需的试剂和资源以满足PKD 更大的PKD研究社区的需求；通过利用现有的机构共享资源 扩大我们的能力，效率和有效性；并开发支持临床的工具 研究并在区域和全国建立合作，以开发多机构 ADPKD临床试验网络。目标2）开发熟练的，协作的，多学科的基本 科学与临床PKD研究者基础。这将通过在我们的 广泛的多学科研究基础，并继续从外部招募新的研究人员 PKD领域通过提供一项创新的试点赠款计划，该计划将吸引新的调查人员进入该领域 通过在全国范围内为新调查人员提供对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 帮助他们成功。目标3）通过教育计划增强PKD研究环境 支持正在进行的项目并开发新的研究方向。研究环境将是 通过开创性计划，夏季学生充实计划，PKD实验室会议，增强 PKD方法研讨会，研究方法和高级技术计划，专业 开发节目系列，以患者为导向的活动，并参与整个财团 年度研讨会。目标4）在区域和全国建立伙伴关系，并建立团队 充当大型PKD研究社区的资源。机构间合作伙伴关系 提供本地和国家基础设施支持，新的研究技术，增加数量 参与临床试验的患者和新研究的研究合作者 通过吸引其他领域的调查人员进入合作网站的PKD研究的机会 在PKD研究联盟和全国合作机构中，扩大 超越任何单个机构可以提供的机会。 项目摘要 - 临床研究核心 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，以协调潜水员的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，这些工具和资源将免费提供给 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 参与资源以确定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。