Core E: Clinical and Translational Core

临床与转化核心（Core E）

Abstract – Clinical and Translational Core (Core E) Despite recent significant and important advances in characterizing pathways of disease progression in ADPKD, the management and treatment of the disease remains largely supportive, without proven disease- modifying interventions. Additional clinical and translational investigations are clearly needed to identify promising new therapies. Significant challenges to such investigations include limited resources, limited access to sufficient numbers of well-characterized patients with ADPKD, incomplete characterization of human kidney tissue from ADPKD patients, lack of sensitive instruments for quantifying patient symptoms, and insufficient expertise in clinical study design. The Baltimore PKD Center Clinical and Translational Core has established a strong foundation of key resources for these investigations and begun to disseminate these resources to active investigators. In our next phase, we propose to promote and expand these resources through: 1) the longitudinal collection of repeated biorepository specimens and clinical measures among 250 participants with ADPKD, including total kidney and liver volume, renal function, and occurrence of acute ADPKD complications. 2) the creation of a novel human tissue bank of ADPKD kidneys among 40 patients receiving simultaneous nephrectomy and renal transplantation and control donor nephrectomy tissue. 3) the development of a web-based disease registry for recruitment into clinical trials and cohort studies. 4) the development and testing of a novel mobile health-informatics tool to prospectively collect patient- reported outcomes in a “real-world” setting. 5) Development of a structured consultation service for ADPKD investigators on the design of clinical and observational trials and methods of human subjects protection. These resources will be disseminated among a robust user base of accomplished internal and external clinical and translational investigators. With these Aims, the support of the University of Maryland Clinical and Translational Science Institute (CTSI) and in close collaboration with the Administrative Core and the other Scientific Cores, the Clinical and Translational Core will provide the resources and expertise to greatly enhance clinical and translational research in ADPKD throughout the Mid-Atlantic region and nationally.