PKD Biomarkers Study

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Brief Title

Early PKD Observational Cohort Study

Official Title

Early PKD Observational Cohort 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 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.
    


Study Type

Observational [Patient Registry]


Primary Outcome

Collect blood and urine samples from affected and unaffected cohorts for basic and translational research


Condition

Polycystic Kidney Disease


Study Arms / Comparison Groups

 Individuals diagnosed with PKD
Description:  Individuals that have been diagnosed and meet the study's definition of early stage PKD.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

250

Start Date

April 2016

Completion Date

June 2025

Primary Completion Date

June 2025

Eligibility Criteria

        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
      

Gender

All

Ages

4 Years - 35 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Alan SL Yu, MD, 913-588-0053, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02936791

Organization ID

STUDY00146013

Secondary IDs

U54DK126126

Responsible Party

Sponsor

Study Sponsor

University of Kansas Medical Center

Collaborators

 National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study Sponsor

Alan SL Yu, MD, Principal Investigator, University of Kansas Medical Center


Verification Date

May 2021