Clinical Trials

Thanks to research funded by the PKD Foundation, clinical trials are now exploring several new therapies for PKD. Patients wishing to participate or learn more will find specific information about the therapies below. To participate in a clinical trial, you will need to have a formal diagnosis of PKD. To learn more, click here.

 
 
       

 

Triptolide

Triptolide is thought to restore the function of the mutated PKD1/PKD2 protein complex which regulates cell growth.  When the one of the proteins is mutated in ADPKD, the cells to lose the "stop growing" signal, allowing them to keep growing, and thus generating cysts.

 

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

The naturally occurring hormone, arginine vasopressin (AVP), acts continuously in ADPKD patients to stimulate the formation of cAMP in renal cells, which causes both fluid secretion and cell growth.  Blocking the action of AVP dramatically slows cyst growth in animal models of cystic disease.  It is thought that drinking water evenly throughout the day will also block AVP in patients with ADPKD.

 

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

 


 

Pain

This study is limited to ADPKD patients with intractable pain that is not controlled by prescription drugs.  A surgical procedure blocks the pain impulses carried by the sphlancnic nerves to the brain. 

 

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

 


 

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
 
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