Clinical Trials Current PKD Trials

There are currently many clinical trials for polycystic kidney disease patients – each providing help and hope to the 12.5 million people worldwide affected by one of the most common, life-threatening genetic diseases.

 

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Blood Pressure Regulation Clinical Trials

 

HALT PKD Progression of PKD   (HALT PKD)

 

Intervention:

Drugs - lisonopril, telmisartan

Sponsor:

University of Pittsburgh Medical Center
Study Phase:
III
Status: Active; enrollment complete

Enrollment goal:

1,018

Description:

To assess the effectiveness of anti-hypertensive drugs telmisartan and lisinopril on PKD progression and its cardiovascular complications in two different levels of kidney function, GFR more than 60 mL/min/1/73m^2 and GFR between 25-60 mL/min/1.73m^2.
Additional Information: The Data Collection Center is now located at the University of Pittsburgh Medical Center. 
Contact:  
Website: NIH Study Details

 

Effect of Statin Therapy on Disease Progression in ADPKD

 

Intervention:

Drug - Pravastatin

Sponsor:

University of Colorado at Denver and Health Sciences Center

Study Phase:

III
Status: Recruiting
Age Group: 8-21

Enrollment goal:

100

Description:

To determine the effect of pravastatin treatment on renal and cardiovascular disease over a 3-year study period in children and young adults aged 8-21 years with ADPKD.
Contact:

Melissa Cadnapaphornchai, M.D. at 303-724-1690 or pkd.nurse@uchsc.edu
Linda Perkins at 303-724-1690 or pkd.nurse@uchsc.edu.
You may also call toll-free at 1-877-765-9297.

Website:

NIH Study Details

 

The Effect of High and Low Sodium Intake on Urinary Aquaporin-2 in ADPKD

 

Intervention:

Drug - High or low sodium diet

Sponsor:

Holstebro Hospital, Denmark
Status: Recruiting
Age Group: 18-65

Enrollment goal:

25

Description:

To examine the effect of high and low diets on kidney function and on hormones that affect the kidney.
Contact: Carolina Cannillo, M.D. at 1145099125428 or cacan@ringamt.dk
Website: NIH Study Details

 

CCB Safety Study in Treatment of Hypertension in ADPKD

 

Intervention:

Drugs - Candesartan; Candesartan Cilnidipine; Candesartan non-calcium channel blocker (CCB)

Sponsor:

Kyorin University, Japan

Study Phase:

IV
Status: Recruiting
Age Group: 20-60

Enrollment goal:

150

Description:

To evaluate the safety and efficacy of calcium channel blocker Candesartan in treatment of hypertension in ADPKD patients.
Contact: Eiji Higashihara, M.D. at  81-422-47-5511, ext. 5813 or ehigashi@kyorin-u.ac.jp
Website: NIH Study Details

 

Study for the Second Line Treatment of Hypertension in Patients With ADPKD

 

Intervention:

Drug - Candesartan, Cilnidipine, Imidapril

Sponsor:

The Ministry of Health, Labour & Welfare, Japan; Teikyo University

Study Phase:

II
Status: Not yet Recruiting
Age Group: 20-60

Enrollment goal:

160

Description:

To compare the safety and effectiveness of combination therapy to control blood pressure in patients with ADPKD whose blood pressure is not controlled under 120/80 with ARB alone. Either ACE or CCB will be added to the regimen and results will be compared to those on ARB alone.

ARB – angiotensin receptor blocker ACE – angiotensin converting enzyme inhibitor CCB – Calcium channel blocker

Contact: Shigeo Horie, MD at +81339642497shorie@med.teikyo-u.ac.jp
Satoru Muto, MD, PhD at +81339642497muto@med.teiku-u.ac.jp
Website: NIH Study Details

 

mTOR Inhibitor Clinical Trials

 

Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease in Patients with ADPKD

 

Intervention:

Drug - Everolimus

Sponsor:

Novartis

Study Phase:

III
Status: Recruiting
Age Group: 18-65

Enrollment goal:

300

Description:

To evaluate the anti-proliferative drug Everolimus as a means to slow disease progression in PKD by measuring total kidney and cyst volumes and changes in renal function.
Contact: Novartis, Freiburg, Germany 41-61-324-1111
Website: NIH Study Details

 

Rapamycin as a Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy

 

Intervention:

Drug - Rapamycin

Sponsor:

Yale Center for Clinical Investigation

Study Phase:

II & III
Status: Recruiting
Age Group: 18-70

Enrollment goal:

45

Description:

To determine the safety & effectiveness of treatment with rapamycin for patients with ADPKD, patients will be observed for change in kidney (& cyst) size by following the patient’s through volumetric magnetic resonance imaging (MRI). To evaluate for change in biomarkers with treatment, blood and urine samples will be collected during the study.
Contact: Neera K. Dahl, MD, PhD at 203-785-4184 or neera.dahl@yale.edu
Website: NIH Study Details

 

Sirolimus Treatment in Patients with ADPKD:  Renal Efficacy and Safety

 

Intervention:

Drug - Sirolimus

Sponsor:

Mario Negri Institute for Pharmacological Research

Study Phase:

II
Status: Recruiting
Age Group: 18-80

Enrollment goal:

16

Description:

To determine  if the addition of sirolimus to conventional  high blood pressure therapy can prevent a further increase in or can reduce total kidney and cyst volumes in ADPKD adults with normal and mild to moderate renal insufficiency.
Contact: Norberto Perico, MD at 0039 035-45351 or perico@marionegri.it
Website: NIH Study Details

 

Sirolimus (Rapamune) for ADPKD

 

Intervention:

Drug - Rapamycin

Sponsor:

The University of Zurich

Study Phase:

III
Status: Active, enrollment complete
Age Group: 18-40

Enrollment goal:

100

Description:

To investigate whether a low dose of sirolimus retards cyst growth and slows renal functional deterioration in patients with ADPKD.
Contact: Andreas Serra, MD at 41 44 255 33 84 or andreas.serra@usz.ch
Website: NIH Study Details

 

Pilot Study of Rapamycin as Treatment for ADPKD

 

Intervention:

Drug - Rapamycin

Sponsor:

The Cleveland Clinic

Study Phase:

I & II
Status: Recruiting
Age Group: 18-75

Enrollment goal:

30

Description:

To compare the effects of an anti-proliferative, anti-angiogenesis and tumor-progression-blocking agent on disease progression in ADPKD.  Due to the intensive and frequent testing schedule, enrollment is limited to local patients only.
Contact: Susan Wirth at 216-444-0124 or wirths@ccf.org
Website: NIH Study Details

 

Somatostatin Clinical Trials




 

Somatostatin in Polycystic Kidney: A Long-term Three Year Follow-up Study

 

Intervention:

Drug - Long-acting somatostatin

Sponsor:

Mario Negri Institute for Pharmacological Research

Study Phase:

III
Status: Recruiting
Age Group: 18-75

Enrollment goal:

66

Description:

To compare the effects on disease progression of a three year treatment regimen using long-acting somatostatin or placebo in patients with ADPKD and normal renal function or mild to moderate renal insufficiency.
Contact: Norberto Perico, MD at 0039 035-45351 or perico@marionegri.it
Website: NIH Study Details

 

Octreotide in Severe Polycystic Liver Disease

 

Intervention:

Drug - Octreotide (long-acting somatostatin)

Sponsor:

Mayo Clinic; Novartis

Study Phase:

II & III
Status: Recruiting
Age Group: 18-80

Enrollment goal:

42

Description:

To evaluate the effect of Octreotide LAR on liver volumes of patients with severe polycystic liver disease who are not candidates for or decline surgical treatments such as liver cyst fenestration or liver transplantation.
Contact: Marie Hogan, M.D., Ph.D. at 507-538-9851 or Linda Page at 507-538-9851
Website: NIH Study Details

 

Lanreotide as Treatment of Polycystic Liver Disease

 

Intervention:

Lanreotide

Sponsor:

Radboud University

Study Phase:

II & III
Status: Completed
Age Group: 18-88

Enrollment goal:

38

Description:

To assess the effectiveness of lanreotide in controlling total liver volume in patients with polycystic livers.
Contact: info@livercyst.org
Website: NIH Study Details

 

Tolvaptan Clinical Trial

 

Tolvaptan Tempo 3 / 4 Trial

 

Intervention:

Drug - Tolvaptan

Sponsors:

Otsuka Pharmaceutical Development & Commercialization, Inc.; Otsuka Pharmaceutical Co., Ltd.

Study Phase:

III
Status: Active; enrollment complete
Age Group: 18-50

Enrollment goal:

1,500

Description:

To evaluate the long-term effectiveness and safety of Tolvaptan by monitoring total kidney volume and cyst volumes by magnetic resonance imaging and measuring a composite of clinical markers in ADPKD patients.
Contact: Within North America: Kristin Cornwell at 866-712-5837 or kristin.cornwell@mayo.edu
Websites: NIH Study Details

 

Triptolide Clinical Trial

 

Randomized Clinical Trial of Triptolide Woldifii for ADPKD

 

Intervention:

Drugs - Tripterygium wilfordii, Emodin, Losartan

Sponsor:

Nanjing University School of Medicine, China
Status: Recruiting
Age Group: 15-70

Enrollment goal:

150

Description:

To evaluate the effectiveness of Triptolide Woldifii to slow disease progression in ADPKD by monitoring total kidney volume and changes in renal function by magnetic resonance imaging.
Contact: Yao Xiaodan at +00862586030066 or yaoxiaodan@hotmail.com
Website: NIH Study Details

 

Water Therapy Clinical Trials

 

Water as Therapy in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

 

Intervention:

Water Prescription

Sponsor:

University of Kansas
Status: Completed
Age Group: 18-50

Enrollment goal:

10

Description:

To formulate a water prescription for use in clinical trials to determine the effect of sustained diuresis on the progression of ADPKD.  Enrollment is limited to local patients only.
Contact: Connie Wang, M.D. at  913-588-6074 or cwang@kumc.edu
Website: NIH Study Details

 

High Water Intake to Slow Progression of PKD

 

Intervention:

Water

Sponsor:

New York University School of Medicine
Status: Recruiting
Age Group: 18-65

Enrollment goal:

20

Description:

To evaluate the changes in urine composition in PKD patients versus healthy individuals with high water intake by monitoring urinary biomarkers.
Contact: Irina Barash, MD  at  212-263-5851  or   irina.barash@nyumc.org
Website: NIH Study Details

 


 

ARPKD Clinical Trials

Evaluation of ARPKD and Congenital Hepatic Fibrosis

 

Observational

 

Sponsor:

National Human Genome Project (NHGRI)
Status: Recruiting
Age Group: 6 months – 80 years

Enrollment goal:

200

Description:

To collect comprehensive data on kidney and liver disease in ARPKD/CHF and follow patients over time to provide the groundwork for more focused studies and novel therapeutic interventions.
Contact: Patient Recruitment and Public Liaison Office at 1- 800-411 1222 or prpl@mail.cc.nih.gov
Website: NIH Study Details

 

The ARPKD Clinical and Genetic Resource

 

Observational

 
Sponsors: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Status: Recruiting
Age Group: Up to 35 years

Enrollment goal:

200

Description:

The Core has three primary objectives: To extend the observational study of ARPKD initiated by the North American ARPKD Database; to provide a mechanism for genetic evaluation of patients with both classic ARPKD and unusual phenotypes of recessive PKD; and to develop educational tools for physicians and patients regarding the natural history, cause, development and effects of the disease, genetic testing, and clinical trials applicable to ARPKD.
Contact: Lisa M. Guay-Woodford, M.D. - 205-934-7308 or lgw@uab.edu
Teresa Chacana, B.S.N. - 205-934-7649 or tchacana@uab.edu
Website: NIH Study Details

 


 

Transplant-Related Clinical Trials

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

 

Observational

 

Sponsor:

Virginia Commonwealth University
Status: Recruiting
Enrollment goal: 2,000

Description:

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.

Contact:

Robert Fisher, M.D. - 804-828-2461
Cheryl Rodgers, BSN, RN - 804-828-2463
Website: NIH Study Details

 

CMV Glycoprotein B Vaccine in Allograft Recipients

 

Intervention:

Biological -- CMV gB vaccine, placebo

Sponsor:

National Institute of Allergy and Infectious Diseases (NIAID)
Status: Recruiting
Age Group: 18 and older

Enrollment goal:

140

Description:

Post-transplant use of immune-suppressive drugs to prevent rejection of the transplanted organ suppresses the recipient’s immune system.  This can result in infection by Cytomegalovirus (CMV), a common infectious agent.  The purpose of this study is to determine if a CMV vaccine administered to patients awaiting transplant can produce immunity to CMV in the post-transplant period.
Contact: Paul Griffiths at +44 20 7830 2997
Website: NIH Study Details

 

Polycystic Liver Disease in Kidney Transplant

 

Intervention:

Drug -- Tacrolimus, Sirolimus, Mycophenolate Mofetil + Prednisone

Sponsor:

Mayo Clinic
Status: Recruiting
Age Group: 18 and older

Enrollment goal:

68

Description:

The purpose of this study is to see if one kind of anti-rejection drug has better effects for the patient's polycystic liver disease than another type. Tacrolimus and Sirolimus are the two anti-rejection drugs that will be compared for this study.
Contact: Qi Qian, MD at 507-284-2511 or qian.qi@mayo.edu
Stacy Anderson at 507-538-9522 or anderson.stacy@mayo.edu
Website: NIH Study Details

 

Pain Clinical Trial

 

ADPKD Pain Study

 

Intervention:

Procedure - Videothracoscopic Splanchnicectomy (VSPL)

Sponsor:

Mayo Clinic

Study Phase:

II
Status: Recruiting
Age Group: 18 and older

Enrollment goal:

20

Description:

To evaluate the effectiveness of VSLP procedure for pain management in patients with ADPKD
Contact: Beverly Tietje at 507-255-0401 or Tietje.beverly@mayo.edu
Website: NIH Study Details