Polycystic Kidney DiseaseEPOCNCT02936791
Early PKD Observational Cohort Study
An observational study of Polycystic Kidney Disease, run by University of Kansas Medical Center at 3 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 4 to 35 yearsHealthy volunteers can take part
- Conditions the study is for
- Polycystic Kidney Disease
- How many people
- 250 participants
- Where
- 3 sitesUnited States
- Study dates
- Apr 2016to Jun 2031
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 35 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: The Study Population will include individuals clinically diagnosed with PKD and meet the inclusion criteria, their family members with not been diagnosed or have been determined not to have cysts within their kidneys and normal volunteers that will serve as a control group for the study.
Who may be able to join
- 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
- Male or female with no family history of kidney disease
- All races and ethnic groups
- Normal GFR
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
University of Chicago
Chicago, Illinois, 60637
Arlene Chapman, MD, Achapman1@medicine.bsd.uchicago.edu
Arlene Chapman, MD
- Recruiting
University of Kansas Medical Center
Kansas City, Kansas, 66160
Cathy Creed, RN, 913-588-0053, ccreed@kumc.edu
Darren P Wallace, PhD, 913-588-3889, dwallace@kumc.edu
- Recruiting
Children's Mercy Hospital
Kansas City, Missouri, 64108
Megan Mains, BSN, 816-760-5918, mmmains@cmh.edu
Laurel Willig, MD, MS
About this study
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.
Read the 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.
- Conditions
- Polycystic Kidney Disease
- Keywords
- autosomal dominant polycystic kidney disease, ADPKD, PKD biomarkers, Kidney Institute, University of Kansas Medical Center
- Medical subject headings
- Polycystic Kidney Diseases, Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Individuals diagnosed with PKD | Individuals that have been diagnosed and meet the study's definition of early stage PKD. | |
| Individuals with a family history of PKD | Unaffected/ undiagnosed family members, preferably siblings, of participants with PKD | |
| Normal individuals for the comparison | Normal volunteers with no family history of PKD or other kidney diseases. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2016 (estimated)
- Study startApr 2016
- Primary completionJun 2030 (estimated)
- Study completionJun 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycystic Kidney DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 250 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alan SL Yu, MD, Principal investigator, University of Kansas Medical CenterDarren P Wallace, PhD, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02936791
- Sponsor's protocol ID
- STUDY00146013
- Other IDs
- U54DK126126
- Acronym
- EPOC
- Status last verified
- Apr 2026
- First submitted
- 10 Oct 2016
- Last updated
- 27 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early PKD Observational Cohort Study
- ClinicalTrials.gov ID
- NCT02936791
- Acronym / sponsor ID
- EPOC / STUDY00146013
- Conditions
- Polycystic Kidney Disease
- Keywords
- autosomal dominant polycystic kidney disease, ADPKD, PKD biomarkers, Kidney Institute, University of Kansas Medical Center
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- Apr 2016 (actual)
- Primary completion
- Jun 2030 (estimated)
- Study completion
- Jun 2031 (estimated)
- First posted
- 18 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Individuals diagnosed with PKD | – | –Individuals that have been diagnosed and meet the study's definition of early stage PKD. |
| Individuals with a family history of PKD | – | –Unaffected/ undiagnosed family members, preferably siblings, of participants with PKD |
| Normal individuals for the comparison | – | –Normal volunteers with no family history of PKD or other kidney diseases. |
Who may qualify
- Ages
- 4 to 35 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- The Study Population will include individuals clinically diagnosed with PKD and meet the inclusion criteria, their family members with not been diagnosed or have been determined not to have cysts within their kidneys and normal volunteers that will serve as a control group for the study.
Key inclusion criteria (7 listed)
- Inclusion for early stage autosomal dominant polycystic kidney disease (ADPKD):
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Collect blood and urine samples from affected and unaffected cohorts for basic and translational research
Time frame: 10 years
The study will establish a repository of blood and urine from individuals with early stage PKD, unaffected/undiagnosed family members and normal volunteers.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2026.
- PKD Biomarkers Repository StudyLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Alan SL Yu, MD principal investigator
University of Kansas Medical Center
Darren P Wallace, PhD principal investigator
University of Kansas Medical Center
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Polycystic Kidney Disease, run by University of Kansas Medical Center at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Cathy Creed, RN (913-588-0053, ccreed@kumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Collect blood and urine samples from affected and unaffected cohorts for basic and translational research (time frame: 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Apr 2026; AzTrials last refreshed its copy on 26 Oct 2026.