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: 11/01/2013

Active, Not Recruiting

Observational Phase I Phase II Phase III Phase IV
HALT PKD Progression of PKD (HALT PKD)  
Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)    
A Study to Investigate the Long-term Safety and Efficacy of Tolvaptan in Patients with ADPKD (Extension of Trial 156-04-251)  
Efficacy, Safety and Tolerability of Everolimus in Preventing End-Stage Renal Disease in Patients with ADPKD  
Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease (OVERTURE)        
8 Week Study of Tolvaptan Dose Forms in ADPKD (NOCTURNE)    
ADPKD Pain Study    
Bosutinib for ADPKD    
Somatostatin Analogues as a Volume Reducing Treatment of Polycystic Livers (RESOLVE)        

 

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

   

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

 

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

 

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: Active, not recruiting
Age Group: 18-65
Enrollment goal: 400
Description: This study will assess whether everolimus (RAD001) is effective in preventing cyst and kidney expansion as well as worsening of renal function in patients with ADPKD and whether the application of 5 mg/day everolimus as monotherapy is safe and well tolerated.
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

 

8 Week Study of Tolvaptan Dose Forms in ADPKD (NOCTURNE)

 

Intervention:

Drug - Tolvaptan

Sponsor:

Otsuka Pharmaceutical Development & Commercialization, Inc.

Study Phase:

II
Status: Active, not recruiting
Age Group: 18-50
Enrollment goal: 180
Description:

To compare the short-term effects of two tolvaptan formulations in patients with ADPKD.

Website: NIH Study Details

 

ADPKD Pain Study

 

Intervention:

Procedure - Videothoracoscopic Splanchnicectomy (VSPL)

Sponsor:

Mayo Clinic

Study Phase:

II
Status: Active, not 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: Marie C. Hogan, M.D., Ph.D.  507-266-1963  or  hogan.marie@mayo.edu
Website: NIH Study Details

 

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

 

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

 

Your Organization

2000 Daniel Island Drive, Charleston SC 29492
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.

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
©2014 PKD Foundation. Privacy Policy | Terms & Conditions

Founded in 1982, our vision is that one day,
no one will suffer the full effects of polycystic kidney disease.


International: Canada Canada  France France  Germany Germany  Italy Italy  Japan Japan  United Kingdom United Kingdom