Early PKD Observational Cohort Study

Polycystic Kidney Disease Clinical Trial

— EPOC  

Official title:

Early PKD Observational Cohort Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02936791
• Other study ID #: STUDY00146013
• Secondary ID: U54DK126126
• Status: Recruiting
• Start date: April 2016
• Est. completion date: June 2025

Study information

• Verified date: June 2024
• Source: University of Kansas Medical Center
• Contact: Cathy Creed, RN
• Phone: 913-588-0053
• Email: ccreed[@]kumc.edu
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Description:

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.

This observational study is currently recruiting for three groups: 1) individuals diagnosed with relatively early PKD as defined by total kidney volume and estimated GFR, 2) unaffected or undiagnosed family members, preferably siblings, 3) normal volunteers with no family history of renal disease.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: June 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 4 Years to 35 Years

Eligibility

Inclusion Criteria:

• Inclusion for early stage autosomal dominant polycystic kidney disease (ADPKD):

• Family history of PKD

• All races and ethnic groups

• Glomerular filtration rate (GFR) >80 ml/min per 1.73 m2

• Inclusion for Healthy Volunteers:

• Male or female with no family history of kidney disease

• All races and ethnic groups

• Normal GFR

Exclusion Criteria:

• Non-insulin or insulin-dependent diabetes mellitus

• Systemic illness (i.e.systemic lupus erythematosus, vasculitis)

• Unable to provide written informed consent

• Unavailable for magnetic resonance imaging (MRI) and blood/urine collection

Related Conditions & MeSH terms

• Kidney Diseases
• Polycystic Kidney Disease
• Polycystic Kidney Diseases

Locations

Country	Name	City	State
United States	University of Chicago	Chicago	Illinois
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	University of Kansas Medical Center	Kansas City	Kansas

Sponsors (2)

Lead Sponsor	Collaborator
University of Kansas Medical Center	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Collect blood and urine samples from affected and unaffected cohorts for basic and translational research	The study will establish a repository of blood and urine from individuals with early stage PKD, unaffected/undiagnosed family members and normal volunteers.	10 years

External Links

•   PKD Biomarkers Repository Study