Updated: 12/18/2014

Observational Studies – Currently Recruiting

Alabama

Colorado

Georgia

Kansas

Maryland

Minnesota

New York

Pennsylvania

Virginia

International

Clinical Care of Autosomal Polycystic Kidney Disease: Retrospective Analysis and Prospective PKD Genotyping (ADPKD)

This is a retrospective analysis to include demographic, clinical, biochemical, and genetic data that will further explore the natural history of ADPKD. It will also assess the factors that are likely to be associated with the progression of disease and the incidence of complications, including progressive chronic kidney disease, cardiovascular disease, and cerebrovascular disease.

The goal of this project is to collect data from a large population of patients with PKD. Based upon the estimated prevalence of PKD (1:500 and 1:1000 live births), it is estimated that there may be 10,000 PKD patients in the New York City area. This sample size far exceeds any database established thus far. As many as 40% of affected PKD patients are reportedly unaware of a family history of this disease, in part because many patients may go undiagnosed until they present with a medical complication (e.g., hypertension, kidney failure). Furthermore, this initiative will provide an opportunity to compare data from racially diverse populations.

Age Group:
18 and older

Location(s):
New York, N.Y.

Participant Information:

  • Must be diagnosed with ADPKD
  • Need to be in the New York City area to participate

Contact Information:
Ines Chicos, CCRC
212.746.3541
inc9012@nyp.org

Website:
http://clinicaltrials.gov/ct2/show/NCT02161068

Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP II)

This is a continuation of the CRISP I study – enrollment is by invitation only. The purpose is to develop and implement studies to test whether imaging techniques can provide accurate and reproducible markers of disease progression in ADPKD patients.

Age Group:
15 - 45 years

Location(s):

  • University of Alabama – Birmingham, Ala.
  • Emory University – Atlanta, Ga.
  • University of Kansas – Kansas City, Kan.
  • Mayo Clinic – Rochester, Minn.
  • University of Pittsburgh – Pittsburgh, Pa.

Participant Information:
This study is enrolling participants by invitation only.

Contact Information:
Not Available as enrollment is by invitation only.

Website:
http://www.clinicaltrials.gov/ct/show/NCT01039987

Core A: The Hepato/Renal Fibrocystic Diseases Translational Resource

To expand their comprehensive clinical database to include information from patients with hepato/renal cystic diseases.

  • To identify genetic mutations in children with ARPKD and other hepato/renal cystic diseases.
  • To establish a human tissue resource from those with hepato/renal cystic diseases for research studies.
  • To develop a comprehensive informational resource for ARPKD and other hepato/renal cystic disease Families.

Age Group:
Up to 35 years

Location(s):
University of Alabama at Birmingham – Birmingham, Ala.

Participant Information:

Inclusion Criteria

  • Demonstrate hepato/renal fibrocystic disease by clinical information, imaging studies, biopsy, autopsy, or genetic testing

Exclusion Criteria

  • ADPKD urinary tract malformations
  • Major congenital anomalies of other systems

Contact Information:
Teresa J. Chacana, MSN, RN
205.934.7649
tchacana@uab.edu

Lisa M. Guay-Woodford, M.D. (principal investigator)
202.476.6439
lguaywoo@cnmc.org

Website:
http://clinicaltrials.gov/ct2/show/NCT01401998

The Eurocyst Initiative: Building a Network of ADPKD Reference Centers Across Europe

EuroCYST initiative aims to build a large, well-characterized cohort of Autosomal Dominant Polycystic Kidney Disease (ADPKD) subjects who are followed in a longitudinal observational cohort study. It has the potential to identify progression factors and biomarkers, and to assess disease stage specific mortality, morbidity and health care costs.

Overall, 1,100 patients will be enrolled in 14 study sites across Europe and will be followed up for at least three years. The ADPKD reference center network across Europe and the observational cohort study will enable European ADPKD researchers to gain insight into the natural history, heterogeneity, and associated complications of the disease, as well as how it affects the lives of patients across Europe.

Age Group:
18 and older

Location(s):

  • Belgium
  • Czech Republic
  • France
  • Germany
  • Italy
  • Switzerland
  • Turkey
  • United Kingdom

Participant Information:

  • Biospecimen samples will be collected (blood, spot urine, and 24-hour urine)

Inclusion Criteria

  • Clinical diagnosis of ADPKD based on kidney imaging and family history
  • Estimated Glomerular Filtration Rate (eGFR) ≥ 30 ml/min/1.73m2
  • Provide written informed consent

Exclusion Criteria

  • Dialysis before or within 12 months after enrollment

Contact Information:
Andreas L Serra, M.D.
+41 44 635 51 07
andreas.serra@usz.ch

Katja Petzold, Dipl.-Ing.
+41 44 635 51 07
katja.petzold@uzh.ch

Belgium
Cliniques universitaires Saint-Luc
Yves Pirson, Prof
yves.pirson@uclouvain.be

Czech Republic:
Charles University Prague
Vladimir Tesar, Prof
vladimir.tesar@lf1.cuni.cz

France:
Centre hospitalier universitaire de Brest
Yannick Le Meur, Prof
yannick.lemeur@chu-brest.fr

Hôpital de Rangueil
Dominique Chaveau, Prof
chauveau.d@chu-toulouse.fr

Germany:
Charité Universitätsmedizin Berlin
Klemens Budde, Prof
klemens.budde@charite.de

University Hospital Erlangen
Kai-Uwe Eckardt, Prof
Kai-Uwe.Eckardt@uk-erlangen.de

University Hospital Freiburg
Anna Koettgen, M.D.
anna.koettgen@uniklinik-freiburg.de

Italy:
Instituto di Ricerche Farmacologiche "Mario Negri"
Giuseppe Remuzzi, Prof
giuseppe.remuzzi@marionegri.it

Switzerland:
University Hospital Zurich
Andreas L Serra, M.D.
+41 44 635 51 07
andreas.serra@usz.ch

Turkey:
Istanbul School of Medicine
Tefvik Ecder, Prof
ecder@istanbul.edu.tr

United Kingdom:
University of Cambridge, Cambridge Cancer Center
Richard Sandford, Prof
rns13@medschl.cam.ac.uk

University of Sheffield Medical School Academic Unit of Nephrology Dept of Infection and Immunity
Albert CM Ong, Prof
a.ong@sheffield.ac.uk

Website:
http://clinicaltrials.gov/ct2/show/NCT02187432

Evaluation of Gut Bacteria in Patients With Polycystic Kidney Disease

To examine the impact of renal failure on the composition of gut microbiota, we are studying patients with renal failure due to polycystic kidney disease (PKD). PKD is the fourth leading cause of kidney failure, and is the most common genetic kidney disease. Compared to patients with renal failure due to diabetic nephropathy, hypertension, and glomerulonephritis, patients with PKD have virtually no major co-morbid medical conditions or associated medical interventions (i.e. antimicrobial or anti-inflammatory therapies) that could potentially alter the gut microbiota, and confound the interpretation of data.

Age Group:
18 and older

Location(s):
Icahn School of Medicine at Mount Sinai, New York, N.Y.

Participant Information:

Inclusion Criteria:

  • Diagnosed with PKD and be able to understand and give consent.

Exclusion Criteria:

  • On antibiotics or vitamin supplement (except Vit. D) in the last three months.
  • Advanced liver disease, advanced cardiovascular disease, heart failure with EF 30% and autoimmune disease.
  • Used chemotherapy, immunosuppressive medications, probiotics, steroid in last three months.
  • Used IV or oral iron supplementation, laxatives, kayexalate in last month.
  • History of intra abdominal surgery, small or large intestine resection or small bowel obstruction.
  • History of colon cancer or gastrointestinal bleed.

Contact Information:
Peter Y Chuang, M.D.
212.241.8004
peter.chuang@mssm.edu

Rabi Yacoub, M.D.
212.241.8004
rabi.yacoub@mountsinai.org

Website:
http://clinicaltrials.gov/ct2/show/NCT02142101

PKD Clinical and Translational Core Study (PKD Core)

The Polycystic Kidney Disease Research Clinical and Translational Core (P30) aims to establish an infrastructure that will assist investigators in designing and conducting highest quality clinical and translational research focused on a diverse group of patients with ADPKD.

Objective 1: To establish a Mid-Atlantic cohort of ADPKD patients (N=200) with baseline clinical phenotyping performed at the General Clinical Research Unit of the University of Maryland School of Medicine.

Objective 2: To establish a state-of-the-art biobank of specimens from the ADPKD cohort including serum, plasma, urine and DNA.

Objective 3: To develop a collaborative network of physicians and practices in the Mid-Atlantic region who will contribute to the ADPKD cohort and will be willing to refer patients for future studies and trials.

Objective 4: To establish a web-based registry of ADPKD patients in the Mid-Atlantic area.

Age Group:
18 and older

Location(s):
University of Maryland School of Medicine General Clinical Research Center – Baltimore, Md.

Participant Information:

  • Biosamples will be collected and stored (plasma, serum, urine, and DNA)

Inclusion Criteria:

  • ADPKD confirmed by genetic testing or ultrasound criteria using modified Ravine criteria: With family history= several cysts per kidney (3 by sonography, 5 if by computerized tomography or MRI) Without family history=10 cysts (by any radiologic method) per kidney and exclusion of other cystic kidney diseases
  • Ability to provide written informed consent prior to initiation of any study procedures and the ability in the opinion of the investigator to comply with all requirements of the study
  • Glomerular Filtration Rate (GFR) less than 20ml/min/1.73m2

Exclusion Criteria:

  • End stage renal disease, on dialysis or have had a prior kidney transplant
  • Pacemaker and defibrillator
  • Pregnant or lactating
  • Other systemic disease (diabetes, lupus)
  • Also participating in a drug treatment trial

Contact Information:
Charalett E. Diggs, RN MSN
410.328.0207
cdiggs@medicine.umaryland.edu

Karleen Schuhart
410.328.3727
kschuhart@medicine.umaryland.edu

Website:
http://clinicaltrials.gov/ct2/show/NCT01873235

Identifying Genetic Modifiers of Severity in Autosomal Dominant Polycystic Kidney Disease

The research study is being conducted to understand which genetic factors influence how severely an individual is affected by Polycystic Kidney Disease (PKD).

The study is funded by the National Institutes of Health (NIH) and recruitment is being conducted at 4 locations in the United States.

Age Group:
18 to 70

Location(s):

  • University of Colorado, Denver - Denver, Col.
  • Mayo Clinic – Rochester, Minn.
  • Emory University – Atlanta, Ga.
  • University of Kansas – Kansas City, Kan.

Participant Information:

Study participants will take part in one study visit lasting approximately 3 hours at the study location nearest to him or her.

At the visit, participants will provide a blood sample, and fill out questionnaires about family history, PKD symptoms, and a health survey form. Participants may also be asked to give permission to obtain related past medical records. If the participant has not had a transplant or dialysis, he or she may also receive an MRI.

Participation is also possible is some cases by mail and telephone without visiting the medical center.

Contact Information:
University of Colorado, Denver
Dr. Berenice Gitomer
303.724.1685
berenice.gitomer@ucdenver.edu

Mayo Clinic
Carly Banks
507.284.0944
Banks.Carly@mayo.edu

Emory
Diane Watkins
404.712.1354
dpwatki@emory.edu

University of Kansas
Cathy Creed, RN, BSN
913.588.0053
ccreed@kumc.edu

New Quantitative MRI Parameters in Assessing Kidneys of Autosomal Dominant Polycystic Kidney Disease (MRI Pilot)

The purpose of this study is to establish normal Magnetic Resonance quantitative values (tissue stiffness, Apparent Diffusion Coefficient values and Blood Oxygen Level Determination values for both renal cortex and medullary tissues and total renal blood flow) for young Autosomal Dominant Polycystic Kidney Disease patients with normal renal function, and normal young adult controls without Autosomal Dominant Polycystic Kidney Disease and normal renal function.

Hypothesis:
Newer Magnetic Resonance quantitative imaging parameters (tissue stiffness, Apparent Diffusion Coefficient, Blood Oxygen Level Determination levels, Magnetization Transfer and renal blood flow) will have different values in young adult ADPKD patients as compared to normal volunteers.

Age Group:
18 to 30 years

Location(s):
Mayo Clinic, Rochester, Minn.

Participant Information:

Inclusion Criteria ADPKD Participants:

  • Diagnosis of ADPKD
  • Glomerular Filtration Rate (GFR) of > or = to 60 mL/min (Chronic Kidney Disease-Epidemiology Collaboration equation)
  • Ability to provide written, informed consent

Exclusion Criteria for ADPKD Participants:

  • Clinically significant concomitant systemic disease
  • Subjects with Diabetes Mellitus
  • Urinary protein excretion
  • Abnormal urinalysis suggestive of concomitant glomerular disease
  • Subjects having contraindications to, or interference with MRI assessments
  • Subjects with supine blood pressure higher than 140/90 mm Hg or taking blood pressure medications

Inclusion Criteria for Normal (Healthy) Volunteers (non-ADPKD volunteers):

  • Glomerular Filtration Rate (GFR) of > or = to 60 mL/min (Chronic Kidney Disease-Epidemiology Collaboration equation)
  • Ability to provide written, informed consent

Exclusion Criteria for Normal (Healthy) Volunteers (non-ADPKD volunteers):

  • Previous personal or family history of kidney disease
  • Clinically significant concomitant systemic disease
  • Subjects with Diabetes Mellitus
  • Urinary protein excretion
  • Abnormal urinalysis suggestive of concomitant glomerular disease
  • Subjects having contraindications to, or interference with MRI assessments
  • Subjects with supine blood pressure higher than 140/90 mm Hg or taking blood pressure medications

Contact Information:
Sara L. Osborn
507.266.1385
osborn.sara@mayo.edu

Kathy J. Brown
507.538.1321
brown.kathy@mayo.edu

Website:
http://clinicaltrials.gov/ct2/show/NCT02250287

Development and Assessment of The Polycystic Liver Disease Questionnaire (PLD-Q)

This Pilot study will enable development & assessment of a Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q). Polycystic liver disease (PLD) is characterized by the formation of numerous cysts in the liver, and can lead to severe symptomatic hepatomegaly. It is common in patients with autosomal dominant polycystic liver disease (ADPLD) and autosomal dominant polycystic kidney disease (ADPKD).

Age Group:
18 and older

Location(s):
Mayo Clinic, Rochester, Minn.

Participant Information:

Inclusion Criteria:

  • Polycystic liver, defined by > 20 liver cysts on imaging

Exclusion Criteria:

  • Patients unable to speak or read the English properly
  • History of kidney or liver transplantation
  • Dialysis
  • Current use of experimental drugs (e.g. lanreotide, octreotide)
  • Recent liver resection or major surgery
  • Other comorbidities that can affect the outcome of the questionnaire, as deemed by the investigators

Contact Information:
Marie C. Hogan
507.266.1963
hogan.marie@mayo.edu

Website:
http://clinicaltrials.gov/ct2/show/NCT02173080

KoreaN Cohort Study for Outcome in Patients With Chronic Kidney Disease: A 10-year Longitudinal Cohort Study of Chronic Kidney Disease (KNOW-CKD)

The goals of the KNOW-CKD (KoreaN cohort study for Outcome in patients With Chronic Kidney Disease) study are 1) to establish a CKD cohort representing Korean CKD population for up to 10-year follow-up, and 2) to investigate the renal progression, mortality, complications, risk factors, role of biochemical parameters and the genetic influence.

Age Group:
20 to 75 years

Location(s):
Seoul National University Hospital - Seoul, Republic of Korea

Participant Information:

Inclusion Criteria:

  • CKD stage 1 through 5
  • Pre-dialysis

Exclusion Criteria:

  • Unable or unwilling to give consent
  • Previously received chronic dialysis
  • Previous any organ transplant
  • Heart failure
  • Known liver cirrhosis
  • Past or current cancer
  • Pregnant women
  • Single kidney due to trauma or donation

Contact Information:
Kook-Hwan Oh, M.D.
82.2.2072.0776

Seungmi Lee, BA
82.2.2072.1592

Website:
http://www.clinicaltrials.gov/ct2/show/NCT01630486

Polycystic Kidney Disease Data Repository

To collect information about PKD so that investigators may fully understand its complications, including high blood pressure, heart attack and stroke. This information may also aid in the development of improved treatment strategies.

Age Group:
18 and older

Location(s):

  • The Rockefeller University Hospital - New York, N.Y.
  • The Rogosin Institute - New York, New York, United States

Participant Information:

  • Patients enrolled in this study will have the diagnosis of ADPKD.
  • Diagnostic criteria for at-risk individuals (i.e., with a first degree family member with ADPKD):
    • The presence of at least two (unilateral or bilateral) renal cysts.
    • Two cysts in each kidney are considered sufficient for diagnosis in aged 15 to 29 years and in 30 to 59 years, respectively.
  • In families of unknown genotype:
    • The presence of three or more (unilateral or bilateral) renal cysts is sufficient for establishing the diagnosis in individuals aged 15 to 39 years.
    • Two or more cysts in each kidney is sufficient for individuals aged 40-59 years.
    • 4 or more cysts in each kidney is required in individuals ≥60 years of age.

Contact Information:
Stephanie Donahue, NP
212.746.1591
sld9001@nyp.org

Website:
http://www.clinicaltrials.gov/ct/show/NCT00792155

Repository Study of Autosomal Dominant Polycystic Kidney Disease

The goal of this project is to collect data from a large population of patients with PKD. Based upon the estimated prevalence of PKD (1:500 and 1:1000 live births), it is estimated that there may be 10,000 PKD patients in the New York City area. This sample size far exceeds any database established thus far. As many as 40% of affected PKD patients are reportedly unaware of a family history of this disease, in part because many patients may go undiagnosed until they present with a medical complication (e.g., hypertension, kidney failure). Furthermore, this initiative will provide an opportunity to compare data from racially diverse populations.

Age Group:
18 and older

Location(s):
The Rogosin Institute, New York, N.Y.

Participant Information:

Inclusion Criteria:

  • Previously diagnosed with ADPKD

Exclusion Criteria:

  • Inability to provide informed consent

Contact Information:
Ines Chicos, BS
212.746.3541
inc9012@nyp.org

Website:
http://www.clinicaltrials.gov/ct2/show/NCT01988038

Transplant Registry: Patients Who May Require Transplantation and Those Who Have Undergone Transplantation of Liver, Kidney and/or Pancreas

To make a systematic review of medical records of patients (with patient consent) in the Virginia Commonwealth University Health System who have received a liver, kidney or pancreas or who may require one and to enter the data into a secure database.

Age Group:
Not listed

Location(s):
Virginia Commonwealth University Health System – Richmond, Va.

Participant Information:

  • This trial is for patients already being followed in the transplant clinic at Virginia Commonwealth University Health System

Contact Information:
Robert Fisher, M.D.
804.828.2461

Mary Baldecchi, RN
804.828.7925

Website:
http://www.clinicaltrials.gov/ct/show/NCT00238693

Your Organization

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Phone: 800.443.9441 | Fax: 843.216.6100
info@yourdomain.org

©2014, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.

©2014, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.