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: 10/14/2014

Active, Not Recruiting

Observational Phase I Phase II Phase III Phase IV
Bosutinib for ADPKD    
Clinical and Molecular Description of PKD1 and PKD2 Mutation Negative Carriers in ADPKD (GeneQuest)    
The Efficacy of Everolimus in Reducing Total Native Kidney Volume in PKD Transplanted Recipients (EVERKYSTE)  
HALT PKD Progression of PKD (HALT PKD)  
Lanreotide in Polycystic Kidney Disease Study (LIPS)    
A New Diet for Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)    
New Quantitative MRI Parameters in Assessing Kidneys of Autosomal Dominant Polycystic Kidney Disease        
Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease (OVERTURE)        
Open-label Trial to Evaluate the Long-term Safety of Titrated Immediate-release Tolvaptan in Subjects with ADPKD    
Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)    
Somatostatin Analogues as a Volume Reducing Treatment of Polycystic Livers (RESOLVE)        
A Study to Investigate the Long-term Safety and Efficacy of Tolvaptan in Patients with ADPKD (Extension of Trial 156-04-251)  

 

Bosutinib for ADPKD

 

Intervention:

Drug - Bosutinib

Sponsor:

Pfizer

Study Phase:

II
Status: Active, not recruiting
Age Group: 18 - 50 years
Enrollment goal: 190
Description:

To determine if bosutinib reduces the rate of kidney enlargement in subjects with ADPKD entering the study with a total kidney volume greater than or equal to 750 cc and eGFR greater than or equal to 60 ml/min/1.73 m2;  To identify a safe and effective dose of bosutinib to be used in future studies.

Contact: Pfizer Call Center  1.800.718.1021
Website: NIH Study Details

 

Clinical and Molecular Description of PKD1 and PKD2 Mutation Negative Carriers in ADPKD (GeneQuest)

 

Observational and Interventional

Intervention: Blood collection

Sponsor:

University Hospital, Brest, France

Status:

Not yet recruiting
Age Group: 16 and older
Enrollment goal: 1,450
Description:
  • Inclusion of ADPKD patients in 20 different centers of Nephrology in the Western part of France
  • Characterization of the Phenotype
  • Collect DNA sample
  • Analysis of PKD1 and PKD2 genes first
  • Analysis of HNFIb and UMOD for PKD1 and PKD2 negative patients
  • Recruitment of affected and non-affected relatives of PKD1 and PKD2 negative ADPKD patients

Identify new genes involved in ADPKD using exome sequencing in PKD1 and PKD2 negative pedigrees.

Contact: Emilie Cornec-Le Gall, MD 298347061 ext. +33 emilie.cornec-legall@chu-brest.fr
Website: NIH Study Details

 

The Efficacy of Everolimus in Reducing Total Native Kidney Volume in Polycystic Kidney Disease (PKD) Transplanted Recipients (EVERKYSTE)

 

Interventional

Drug: Everolimus
Drug: Calcineurin inhibitors maintenance

Sponsor:

Assistance Publique - Hôpitaux de Paris

Study Phase:

III

Collaborator: Novartis
Status: Not yet recruiting
Age Group: 18 to 75
Enrollment goal: 40
Description:

This multi-center randomized open-labelled study will investigate the efficacy of an everolimus based immunosuppression in reducing total native kidney volume in kidney recipients with autosomal dominant polycystic kidney disease, compared to a calcineurin inhibitor-based immunosuppression. The primary objective will be the reduction of total native kidney volume after a 2-years treatment period.

Contact: Hélène François, MD, PhD+33(1)45 21 27 22helene.francois@bct.aphp.fr
Website: NIH Study Details

 

HALT PKD Progression of PKD (HALT PKD)

 

Intervention:

Drugs - lisonopril, telmisartan

Sponsor:

University of Pittsburgh Medical Center
Study Phase: III
Status: Active; enrollment complete
Age group: 15 - 64

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 Info: The Data Collection Center is now located at the University of Pittsburgh Medical Center. 
Website: NIH Study Details

 

Lanreotide in Polycystic Kidney Disease Study (LIPS)

 

Intervention:

Drugs: Lanreotide, saline

Sponsor:

Assistance Publique - Hôpitaux de Paris

Collaborator:

IPSEN pharmaceutical company, Boulogne-Billancourt, France
Study Phase: III
Status: Not yet recruiting
Age group: 18 and older

Enrollment goal:

180

Description:

LIPS study (Lanreotide In Polycystic kidney disease Study) is a prospective randomized double blind placebo controlled study. The main objective is to prove that lanreotide, a somatostatin analog, is able to reduce the glomerular filtration rate decline over 3 years by at least 30%. Cardiovascular outcomes, blood pressure, quality of life and safety are among the secondary outcomes. The study, which will include 180 ADPKD patients, is scheduled to start in early 2014.

An equal number of patients with chronic kidney disease stage 2 (90 patients with GFR 89 to 60 ml/mn/1.73 m2) and chronic kidney disease stage 3 (90 patients with GFR 59 to 30 ml/mn/1.73 m2) will be included. The primary endpoint (GFR decline) will be assessed by repeated measures, in the overall population as well as in the two GFR stratus.

Contact:

Dominique Joly, MD, PhD 1 44 49 54 15 ext. +33 dominique.joly@nck.aphp.fr
Laurence Lecomte, PhD 1 71 19 64 94 ext. +33 laurence.lecomte@nck.aphp.fr

Website: NIH Study Details

 

A New Diet for Patients with Autosomal Dominant Polycystic Kidney Disease

 

Intervention:

ADPKD Diet

Sponsor:

University of Kansas
Study Phase: II
Status: Active, not recruiting
Age group: 18 - 65

Enrollment goal:

10

Description:

Recent evidence has shown that kidney volume predicts the likelihood of developing renal insufficiency over a finite length of time in ADPKD, suggesting a linkage between the growth of cysts and the harm they do to kidney function. Recent studies indicate that the rate of kidney volume increase is hastened by excess dietary protein, salt, and potential net acid precursors, and slowed by increased water intake sufficient to lower plasma vasopressin levels.

Diets are commonly prescribed to treat ADPKD and other renal patients with disease near the end-stage, but there is currently no specific diet prescription that takes potentially harmful dietary elements into account for ADPKD patients in the earliest stages of the disease. This study will examine a novel diet for ADPKD created by the researcher termed the ADPKD diet.

Website:

NIH Study Details

 

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

 

Observational

 

Sponsor:

Mayo Clinic
Status: Not yet recruiting
Age group: 18 - 30 years

Enrollment goal:

20

Description:

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.

Contact:

Sara L. Osborn (507) 266-1385 osborn.sara@mayo.edu
Kathy J. Brown (507) 538-1321 brown.kathy@mayo.edu

Website:

NIH Study Details

 

Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease (OVERTURE)

 

Observational

 

Sponsor:

Otsuka Pharmaceutical Development & Commercialization, Inc.
Status: Active, not recruiting
Age Group: 12 - 70
Enrollment goal: 3000
Description:

To collect characteristics of patients with ADPKD across a broad population, over time to better understand disease progression (signs, symptoms and outcomes).  Association with total kidney volume changes and other measures of disease progression will be determined in order to identify a population at increased risk for disease progression.  The economic and quality life impact of ADPKD will be assessed.  Subjects who terminated participation early from clinical trials with tolvaptan may also be followed.

Contact: OVERTUREStudyinfo@mmgct.com
Website: NIH Study Details

 

Open-label Trial to Evaluate the Long Term Safety of Titrated Immediate-release Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease

 

Interventional:

Drug:  Tolvaptan (OPC-41061) 

Sponsor:

Otsuka Pharmaceutical Development & Commercialization, Inc.

Study Phase:

IIIb
Status: Not yet recruiting - limited to participants in prior Tolvaptan clinical trials
Age Group: 18 and older
Enrollment goal: 2,500
Description:

This trial is a phase 3b to evaluate and describe the long-term safety of tolvaptan treatment in ADPKD patients with CKD (chronic kidney disease). Eligible subjects will enter the trial from Trial 156-08-271 or other Tolvaptan interventional trials. Renal function will be assessed during screening by using historical laboratory values for serum creatinine levels to calculate the estimated glomerular filtration rate (eGFR). General safety endpoints will be assessed and also relevant safety endpoints for the trial as Serum transaminase elevations in frequency & Serum sodium excursions.

Contact: No contact information provided
Website: NIH Study Details

 

Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

 

Intervention:

Drug: Rapamycin

Sponsor:

The Cleveland Clinic

Study Phase:

I/II
Status: Active, not recruiting
Age Group: 18-75

Enrollment goal:

30

Description:

This study is a prospective, randomized,open label, pilot clinical trial designed to compare the effects of an agent that has antiproliferative (1,2), antiangiogenesis (3),and tumor-progression blocking capabilities (4), namely, rapamycin (Rapamune®), in the treatment of autosomal-dominant polycystic kidney disease (ADPKD).
Website: NIH Study Details

 

Somatostatin Analogues as a Volume Reducing Treatment of Polycystic Livers (RESOLVE)

 

Observational

 

Intervention:

Drug - Lanreotide

Sponsor:

Radboud University

Collaborator:

Ipsen Pharmaceutical
Status: Active, not recruiting
Age Group: 18-70
Enrollment goal: 43
Description:

To determine the effect of Lanreotide on polycystic liver and kidneys in patients with ADPKD.

Website: NIH Study Details

 

A Study to Investigate the Long-term Safety and Efficacy of Tolvaptan in Patients with ADPKD
(Extension of Trial 156-04-251)

 

Intervention:

Drug: tolvaptan 

Sponsor:

Otsuka Pharmaceutical Co., Ltd.

Study Phase:

III

Status:

Active, not recruiting

Age Group:

23 to 53

Enrollment goal:

150

Description:

To continue ADPKD patients enrolled in Trial 156-04-251 on oral Tolvaptan twice daily.

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.