Conducting Clinical Trials
Clinical trial design: To guide investigators in the design of early-phase clinical trials of therapy in PKD, a consultation service is available, staffed by nephrologists with expertise in PKD clinical studies (Jared Grantham, MD, Alan Yu, MD), a specialist in early phase clinical trials (Raymond Perez, MD, Medical Director of the Clinical Research Center, KU Cancer Center), a biostatistician (Jonathan Mahnken, Ph.D.) and a highly experienced PKD research coordinator (Catherine Creed, RN).
Research staff: Research coordinators with experience in PKD research studies are available to assist in patient recruitment, study visits and calls.
Clinic rooms: Sites available for patient visits include an exam room in Nephrology clinical research space on the 2nd floor of the Delp Building on the KUMC campus. Freezers, refrigerators, centrifuges for sample handling, ultrasonic bladder scanner, and secure storage are all available at this site. The Frontiers Clinical and Translational Science Institute, which is a new, state-of-the-art, free-standing clinical trials facility located in Fairway, has 9 exam rooms available, a completely modern lab facility and hospital lab services, 11 drug infusion suites, an extended observation unit, exercise physiology lab, sleep lab, metabolic and demonstration kitchen, and investigational pharmacy.
Laboratory services: Standard lab testing is available through the Univ. of Kansas Hospital or as a send out to Quest laboratories.
Iothalamate GFR testing: Direct measurement of GFR is the gold standard for assessing renal function. In the case of PKD patients, the eGFR determined by the MDRD equation has been shown in the CRISP study to underestimate measured GFR by an average of 16 mL/min/1.73 m2, and predictors of renal function decline are most strongly associated with measured GFR. Our nurse coordinators are adept at performing iothalamate GFR measurements by the short (45 min) unlabeled iothalamate renal clearance test.
Urine soluble biomarker assays: The Biomarker Core offers two urinary biomarker panels to monitor disease progression: a standard ELISA panel to be performed at KU, and a Kidney Multi-Analyte Panel performed by Myriad RBM that uses microsphere-based multiplex immunoassay technology.
Urine exosome analysis: The Biomarker Core offers a service to isolate urine exosomes from PKD study subjects and assay for PC1/TMEM2 and other potential biomarkers.
Genotyping service: In collaboration with the Mayo Clinic PKD Center Molecular Genetics and Biomarker Core, we offer genetic screening for PKD1 and PKD2 mutations. Our Core will draw blood and perform DNA isolation, then send de-identified samples to the Mayo Clinic for Sanger sequencing and, if necessary, multiplex ligation-dependent probe amplification.
Research MRI facility: Total kidney and cyst volume are currently the best validated markers of PKD progression, and MRI is considered to be the most accurate method of measuring these. We offer kidney volume determination through the Hoglund Biomedical Imaging Center, which houses a whole body 3 Tesla Siemens Skyra MRI scanner. Staff and faculty at the Hoglund are experienced both at kidney imaging protocols and at volume determination by stereology.
Biostatistical support: The Core will have access to a portion of time of a dedicated biostatistician who will provide services to assist in the following activities:
- Design and analysis of investigator-initiated clinical trials, including innovative approaches such as adaptive design.
- Secondary analyses of datasets from existing trials (e.g. CRISP, HALT, etc.). Center members will have the capability of proposing ancillary studies to ongoing trials or requesting datasets from NIDDK for completed trials, using the Core to support the biostatistical analysis.
- Biostatistical support for observational research projects that emanate from use of the HERON database.
Patient-centered research services: Though not explicitly incorporated into this core, the following resources are available locally to support research at KUMC and Frontiers institutions. The Personalized Medicine and Outcomes Center (directed by Dr. John Spertus, Director for Health Outcomes Research at Saint Luke's Mid-America Heart Institute) provides expert guidance on survey design for patient-reported outcomes. Dr. Kim Kimminau (Research Director for the American Academy of Family Physicians National Research Network) designs patient engagement activities for patient-centered outcomes research. Please contact these individuals directly to learn more.