Clinical Trial: Early-Stage Polycystic Kidney Disease Biomarkers Repository Study
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]
Official Title: Polycystic Kidney Disease (PKD) Biomarkers Repository Study
Brief Summary: This observational study will collect blood and urine and clinical information from individuals with early-stages of polycystic kidney disease (PKD), their unaffected siblings and normal volunteers to create a biobank, also called a biorepository. The long-term goal is to develop new knowledge on biological markers or biomarkers that indicate changes in the disease progression. An understanding of biomarkers for early renal cyst growth will benefit PKD patients as new therapies are being developed and tested.
Detailed Summary:
In polycystic kidney disease (PKD), renal cysts form in utero and progressively enlarge due to aberrant proliferation of the cyst-lining cells and accumulation of fluid within the expanding cyst cavity. Over decades of unrelenting cyst growth, renal function declines due to the loss of functional tissue, eventually leading to kidney failure and the need for renal replacement therapy, such as dialysis or kidney transplantation.
Effective therapies for the treatment of PKD will need to be delivered as early as possible, before a measurable decline in kidney function, to preserve functional tissue. Currently, it is difficult to make an accurate prognosis of the progression of early-stage PKD since the growth of microscopic cysts is difficult to detect by standard imaging modalities and changes in total kidney volume measured within a reasonable time period are too small to be informative. Even though early cysts may not cause detectable changes in total kidney volume, their progressive enlargement damages the surrounding tissue and is a prelude to chronic kidney disease.
Current blood and urine tests provide important information on the decline of kidney function; however, these tests are not useful for monitoring early events of PKD such as initial cyst growth and damage to neighboring tissue. Clearly, novel biomarkers of early cystic disease need to be discovered to develop appropriate clinical tests to monitor the progression of early stage PKD. These tests will be important to identify patients at risk of rapid progression and of need of therapeutic intervention and to monitor the effectiveness of the therapeutic drug. The PKD Biomarkers Repository will allow approved researchers to obtain blood and urine samples for biomarker discovery and development of appropriate biomarker assays for prognosis of early PKD.
Sponsor: University of Kansas Medical CenterCurrent Primary Outcome: Collect blood and urine samples from affected and unaffected cohorts for basic and translational research [ Time Frame: 5 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
Original Secondary Outcome:
Information By: University of Kansas Medical Center
Dates:
Date Received: October 10, 2016
Date Started: April 2016
Date Completion: June 2020
Last Updated: October 17, 2016
Last Verified: October 2016
