Additional informatics support on U54 Clinical Core

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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.