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.


You can also learn about potential treatmentsPKD and heart disease and the Accelerating Clinical Trials (ACT) program.

Updated: 2/27/2014

Completed Studies

Efficacy Study of Water Drinking on PKD Progression

 

Observational

 

Sponsor:

Kyorin University
Status: Completed

Results:

No information available at this time.
Website: NIH Study Details

 

Octreotide in Severe Polycystic Liver Disease

 

Intervention:

Drug - Octreotide (long-acting somatostatin)

Sponsor:

Mayo Clinic; Novartis
Status: Completed, Has Results

Results:

See NIH Study Details
Website: NIH Study Details

 

Tolvaptan Phase 3 Efficacy and Safety Study in ADPKD (Tempo 3/4)

 

Intervention:

Drug - Tolvaptan

Sponsor:

Otsuka Pharmaceutical Development & Commercialization, Inc.; Otsuka Pharmaceutical Co., Ltd.
Status: Completed
Results: The last patient has completed the TEMPO 3/4 Study, which was first listed on the FDA Clinical Trials website in January 2007. The data collected from all the study patients will now be reviewed and evaluated by the study directors. When the data review is complete, Otsuka will use this information to decide on the next steps in moving Tolvaptan through the drug approval process. It is not known at this time if additional studies will be necessary. Please watch our website for updates.
Website: NIH Study Details

 

Adrenal Functions in ADPKD

 

Intervention:

Drug - Tetracosactin

Sponsor:

Istanbul University
Status: Completed
Results: No information available at this time.
Website: NIH Study Details

 

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

 

Intervention:

Water Prescription

Sponsor:

University of Kansas
Status: Completed
Results:

In animal studies of ADPKD, giving the animal large amounts of water suppresses vasopressin levels in the blood and cyclic AMP levels in the kidneys, slowing cyst enlargement. This study tested whether water ingestion is a potential therapeutic strategy in people with ADPKD.

This study was designed so that water intake for each individual was calculated so that urine solute concentration reached a specific target level of 285 mOsm. Five of the eight individuals enrolled in the study were able to achieve a urine osmolality of 285 mOsm. (Osmolality is a measure of solutes in the urine.) The success of this study shows that a quantitative method to determine the amount of water intake needed for a given individual to achieve a target osmolality can be calculated.

Several limitations of the study were acknowledged by the researchers. First, the sample size was much smaller than required for a clinical trial because a methodology was being developed and validated. The effect of water intake on the course of the disease was not tested. Secondly, the small sample size included almost all Caucasian women. Daily solute intake, urine solute concentration and blood levels of vasopressin vary by race and gender, so these findings cannot be generalized to a more diverse population. Thirdly, study participants had normal blood pressure with GFRs in chronic kidney disease stages 1 and 2. NOTE: For those with advanced kidney disease (stages 3, 4 & 5) it is important to not increase fluid intake above normal.

Whether or not water intake can affect the course of ADPKD by lowering vasopressin levels in the kidney is a critical factor that must be studied further.

Website:

NIH Study Details

 

Lanreotide as Treatment of Polycystic Liver Disease

 

Intervention:

Drug - Lanreotide

Sponsor:

Radboud University
Status: Completed
Results: No information available at this time.
Website: NIH Study Details

  

Sirolimus Treatment in Patients with ADPKD:  Renal Efficacy and Safety (SIRENA)

 

Intervention:

Drug - sirolimus

Sponsor:

Mario Negri Institute for Pharmacological Research
Status: Completed
Results: This study was an 18-month, open-label, randomized, controlled trial in which researchers hoped to determine whether sirolimus halts the growth in kidney volume among patients with ADPKD. At the start of the study, total kidney volume for both control and sirolimus groups was approximately twice normal volume. Creatinine clearance was 70 ml/min in both groups. After 18 months of treatment, sirolimus did not halt polycystic kidney growth.
Website: NIH Study Details

 

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

 

Intervention:

High or low sodium diet

Sponsor:

Holstebro Hospital, Denmark

Status:

Completed

Results:

No information available at this time.

Website:

NIH Study Details

 

Pilot Study of RNA as a Biomarker for ADPKD

 

Observational

 

Sponsor:

The Rogosin Institute

Status:

Completed

Results:

No information available at this time.

Website:

NIH Study Details

 

Short-Term Renal Hemodynamic Effects of Tolvaptan in Subjects with ADPKD

 

Intervention:

Drug - Tolvaptan (OPC-41061)

Sponsor:

Otsuka Pharmaceutical Development & Commercialization, Inc.

Status:

Completed

Results:

No information available at this time.

Website:

NIH Study Details

 

The Vienna RAP Pilot Study

 

Intervention:

Drug - Sirolimus

Sponsor:

Medical University of Vienna

Status:

Completed

Results:

No information available at this time.

Website:

NIH Study Details

 

Dose-finding Study of New Tolvaptan Formulation in Subjects With ADPKD
 

Intervention:

Drug - Tolvaptan MR, Tolvaptan IR

Sponsor:

Otsuka Pharmaceutical Development & Commercialization, Inc.

Status:

Completed

Results:

No information available at this time.

Website:

NIH Study Details

 

Effect of Statin Therapy on Disease Progression in ADPKD

 

Intervention:

Drug - Pravastatin

Sponsor:

University of Colorado Denver

Status:

Completed

Results:

No information available at this time.

Website:

NIH Study Details

 

Somatostatin in Polycystic Kidney: A Long-Term Three Year Follow-Up Study
 

Intervention:

Drug - Long-acting somatostatin

Sponsor:

Mario Negri Institute for Pharmacological Research

Status:

Completed

Results:

No information available at this time.

Website:

NIH Study Details

 

Effects of Systemic NO-Inhibition on Renal Hemodynamics in Patients With Polycystic Kidney Disease and Chronic Glomerulonephritis

 

Intervention:

Drug - Ng-monomethyl-L-arginine

Sponsor:

Regional Hospital Holstebro, Denmark 

Status:

Completed

Results:

No information available at this time.

Website:

NIH Study Details

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©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.

The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.
National Headquarters: 8330 Ward Parkway, Suite 510, Kansas City, MO 64114. Phone: 1.800.PKD.CURE
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Founded in 1982, our vision is that one day,
no one will suffer the full effects of polycystic kidney disease.


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