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: 3/25/2014

Active, Recruiting

Observational Phase I Phase II Phase III Phase IV
A New Diet for Patients with Autosomal Dominant Polycystic Disease (ADPKD)    
A Safety, Pharmacokinetic and Dose-Escalation Study of KD019 in Subjects With ADPKD    
Pasireotide LAR in Severe Polycystic Liver Disease (SOM230)    
Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP II)        
Open-Label Tolvaptan Study in Subjects With ADPKD  
Everolimus on CKD Progression in ADPKD  
Core A: The Hepato/Renal Fibrocystic Diseases Translational Resource        
Somatostatin in Patients with ADPKD and Moderate to Severe Renal Insufficiency (ALADIN 2)  
Study of Lanreotide to Treat Polycystic Kidney Disease (DIPAK1)  
Sirolimus for Massive Polycystic Liver (SILVER)  
Radiofrequency Ablation for ADPKD Blood Pressure and Disease Progression Control (RAFALE)    
PKD Clinical and Translationl Core Study (PKD Core)        
Clinical and Molecular Investigations Into Ciliopathies        

 

Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease - The Vienna RAP Study

 

Interventional

Sirolimus

Sponsor:

Medical University of Vienna

Status:

Not yet recruiting

Age Group:

18 and older

Enrollment goal:

68

Description:

Sirolimus (SIR) has lead to a reduction of overall kidney size, a decrease in cyst density and general tubular cell proliferation in animal models, and to a reduction of the increase in creatinine and blood urea nitrogen by 34 and 39 percent respectively, as well as a reduction of cyst proliferation, expressed by a 30 percent reduction of overall kidney enlargement, a reduction in general cyst volume, and a reduction of the cyst volume density in the renal cortex in humans.

However, despite promising data from animal- and in vivo studies, most mammalian target of rapamycin inhibitor (mTOR-I) studies in patients with autosomal-dominant polycystic kidney disease (ADPKD) produced only subtle if any clinically relevant effects on cyst growth and the preservation of renal function.

In this study we will investigate if pulsed administration of SIR in a fixed weekly oral dose of 3 mg over 24 months compared to placebo significantly reduces cyst growth and preserves excretory renal function in patients with ADPKD and an estimated glomerular filtration (eGFR) rate below 60 mL/min per 1.73m2.

Contact:

Markus Riegersperger, MD 0043140400 ext 4391 markus.riegersperger@meduniwien.ac.at
Gere Sunder-Plassmann, MD 0043140400 ext 4391

gere.sunder-plassmann@meduniwien.ac.at

Website:

NIH Study Details

 

Renal Sympathetic Denervation for Reduction of Pain and Improvement of Insulin Sensitivity in Adult Polycystic Kidney Disease

 

Interventional

Renal Denervation

Sponsor:

Odense University Hospital

Status:

Enrolling

Age Group:

18 and older

Enrollment goal:

12

Description:

In patients with polycystic kidney disease, pain may be resistant to drug therapy and may reduce quality of life. This study investigate the effect of renal denervation on this pain.

Contact:

Hans Dieperink, MD +4520598851 hans.dieperink@rsyd.dk  
Marie Blicher, MD + 45 65415305 marie.blicher@rsyd.dk  

Website:

NIH Study Details

 

A New Diet for Patients with Autosomal Dominant Polycystic Disease (ADPKD)

 

Interventional

ADPKD Diet 

Sponsor:

University of Kansas
Study Phase: II

Status:

Enrolling

Age Group:

18 to 65

Enrollment goal:

10

Description:

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 water intake sufficient to lower plasma vasopressin levels. This study will examine the effect of a novel diet (ADPKD diet) on kidney function.

Additional Info:

All patients will follow their regular diet for 8 days. Then they will be asked to follow the ADPKD diet for a total of 4 weeks.

Website:

NIH Study Details

 

A Safety, Pharmacokinetic and Dose-Escalation Study of KD019 in Subjects With ADPKD

 

Intervention:

Drug: KD019

Sponsor:

Kadmon Corporation, LLC
Study Phase: I & II

Status:

Recruiting

Age Group:

18 to 55

Enrollment goal:

110

Description:

The primary purpose of this study is to determine the safety, tolerability and plasma pharmacokinetics of KD019 in ADPKD patients. This is a 28 day daily dosing study with an option to continue through 6 months of KD019 dosing. All participants receive active KD019 study drug. KD019 is an oral, once-daily 50 mg strength tablet. Participants will enroll in three sequential dosing cohort levels (50 mg, 100 mg and 150 mg). Study participants will have an MRI of the abdomen at Screening and at 6 month visit to explore the effects of KD019. An echocardiogram will be performed Screening, Day 28 and Months 2-6, if option of continuation past Day 28 is accepted.

Contact:

Mayo Clinic, Rochester MN, Lisa Bungum 507-266-4616 or Bungum.Lisa2@mayo.edu
New York University School of Medicine, Casey Santana 212-263-6411 or casey.santana@nyumc.org

Website:

NIH Study Details

 

Pasireotide LAR in Severe Polycystic Liver Disease (SOM230)

 

Intervention:

Drug: Pasireotide LAR

Sponsor:

Mayo Clinic
Study Phase: II

Status:

Recruiting

Age Group:

18 and older

Enrollment goal:

48

Description:

The purpose of this study is to compare SOM230 treatment to placebo in patients with severe polycystic liver disease. The investigators will also assess the effectiveness and safety of SOM230 in reducing total liver volume and improving quality of life.

Contact:

Marie Hogan, MD, PhD 507-284-2500 or hogan.marie@mayo.edu
Angela Ihrke 507-538-2902 or ihtkr.angela@mayo.org

Website:

NIH Study Details

 

Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP II)

 

Observational

 

Sponsor:

University of Pittsburgh

Collaborators:

NIH, NIDDK

Study Phase:

Continuation of CRISP I

Status:

Enrolling by invitation only

Age Group:

15-45

Enrollment goal:

211

Description:

This study is a continuation of CRISP I to develop and implement studies to test whether imaging techniques can provide accurate and reproducible markers of disease progression in ADPKD patients.

Website:

NIH Study Details

 

Open-Label Tolvaptan Study in Subjects With ADPKD (TEMPO 4/4)

 

Intervention:

Drug - Tolvaptan

Sponsors:

Otsuka Pharmaceutical Development & Commercialization, Inc.

Study Phase:

III

Status:

Enrolling by invitation only

Enrollment goal:

1,500

Description:

To demonstrate whether Tolvaptan modifies ADPKD progression as measured by changes from baseline in total kidney volume (TKV) and renal function.

Website:

NIH Study Details

 

Everolimus on CKD Progression in ADPKD

 

Intervention:

Drug: Everolimus
Drug: Standard Therapy

Sponsor:

A. Manzoni Hospital

Study Phase:

II and III

Status:

Recruiting

Age Group:

18 and older

Enrollment goal:

90

Description:

To evaluate whether the administration of everolimus (1/5 mg/day) can slow the progression of chronic kidney disease in ADPKD patients.

Website:

NIH Study Details

 

Core A:  The Hepato/Renal Fibrocystic Diseases Translational Resource

 

Observational

 

Sponsor:

University of Alabama at Birmingham
Collaborator: National Institutes of Diabetes and Digestive and Kidney Diseases

Status:

Recruiting

Age Group:

Up to 35 years

Enrollment goal:

200

Description:

  • To expand their comprehensive clinical database to include information from patients with hepato/renal cystic diseases.
  • To identify genetic mutations in children with ARPKD and other hepato/renal cystic diseases.
  • To establish a human tissue resource from those with hepato/renal cystic diseases for research studies.
  • To develop a comprehensive informational resource for ARPKD and other hepato/renal cystic disease Families.

Website:

NIH Study Details

 

Somatostatin in Patients with ADPKD and Moderate to Severe Renal Insufficiency (ALADIN 2)

 

Intervention:

Octreotide-LAR

Sponsor:

Mario Negri Institute for Pharmacological Research
Study Phase: III
Status: Recruiting
Age Group: 18-75

Enrollment goal:

80

Description:

To assess the efficacy of one year treatment with long-acting somatostatin analogue (Octreotide LAR)
compared to placebo in slowing kidney and liver growth rate in patients with ADPKD and moderate/severe
renal insufficiency.
Contact: Norberto Perico, Mario Negri Institute for Pharmacological Research
Website: NIH Study Details

 

Study of Lanreotide to Treat Polycystic Kidney Disease (DIPAK1)
 

 

Intervention:

Drug: Lanreotide

Sponsor:

University Medical Centre Groningen

Collaborators

Leiden University Medical Center, Erasmus Medical Center, Radboud University

Study Phase: III

Status:

Recruiting

Age Group:

18 Years to 60 Years

Enrollment goal:

300

Description:

The purpose of this study is to investigate whether the somatostatin analogue Lanreotide slows progression of polycystic and liver disease in ADPKD patients.

Contact:

Esther Meijer, MD, PhD esther.meijer@umcg.nl
Ron Gansevoort, MD, PhD r.t.gansevoort@umcg.nl

Website:

NIH Study Details

 

Sirolimus for Massive Polycystic Liver (SILVER)
 

Intervention:

Drug: Sirolimus

Sponsor:

Seoul National University Hospital
Study Phase: II & III

Status:

Recruiting

Age Group:

18 Years to 65 Years

Enrollment goal:

44

Description:

This is a open-label, prospective study to evaluate the effectiveness and safety of Sirolimus to reduce cyst growth in ADPKD patients with massive polycystic liver.

Contact:

Curie Ahn, MD, PhD 82-2-2072-2222 or curie@snu.ac.kr

Website:

NIH Study Details

 

Radiofrequency Ablation for ADPKD Blood Pressure and Disease Progression Control (RAFALE) 

 

Intervention:

Procedure: renal sympathetic denervation;   Drug: antihypertensive drugs

Sponsor:

Mei changlin, Shanghai Changzheng Hospital
Study Phase: II

Status:

Recruiting

Age Group:

20 Years to 60 Years

Enrollment goal:

100

Description:

A randomized, open-label single-center study investigates the efficacy and safety of bilateral renal artery sympathetic denervation by catheter-based radiofrequency ablation on blood pressure and disease progression control in autosomal dominant polycystic kidney disease (ADPKD).

Contact:

Changlin Mei, MD +86 21 81885391 chlmei1954@126.com  
Yiyi Ma, Master +8613661679863 dukemm@126.com

Website:

NIH Study Details

 

PKD Clinical and Translational Core Study (PKD Core)

 

Observational

 

Sponsor:

University of Maryland
Status: Recruiting

Age Group:

18 years and older

Enrollment goal:

200

Description:

The Polycystic Kidney Disease Research Clinical and Translational Core (P30) aims to establish an infrastructure that will assist investigators in designing and conducting highest quality clinical and translational research focused on a diverse group of patients with ADPKD.

Objective 1: To establish a Mid-Atlantic cohort of ADPKD patients (N=200) with baseline clinical phenotyping performed at the General Clinical Research Unit of the University of Maryland School of Medicine.

Objective 2: To establish a state-of-the-art biobank of specimens from the ADPKD cohort including serum, plasma, urine and DNA.

Objective 3: To develop a collaborative network of physicians and practices in the Mid-Atlantic region who will contribute to the ADPKD cohort and will be willing to refer patients for future studies and trials.

Objective 4: To establish a web-based registry of ADPKD patients in the Mid-Atlantic area.

Contact:

Charalett E Diggs, RN, MSN    410-328-0207    cdiggs@medicine.umaryland.edu

Karkleen Schuhart    410-328-3727    kschuhart@medicine.umaryland.edu

Website:

NIH Study Details

 

Clinical and Molecular Investigations Into Ciliopathies

 

Observational

 

Sponsor:

National Human Genome Research Institute (NHGRI)
Status: Recruiting

Age Group:

6 months to 80 years

Enrollment goal:

500

Description:

This study will evaluate patients' ciliopathies.  People with ciliopathies develop fibrocystic disease of the kidneys and liver, retinal degeneration, obesity, structural and functional defects of the central nervous system and the eyes, abnormal bone growth, abnormal sidedness of internal organs and polydactyly. The goal of the study is to better understand the medical complications of these disorders and identify characteristics that can help in the design of new treatments.

Contact:

Contact: Meral Gunay-Aygun, M.D. (443) 286-1703 mg466r@nih.gov

Website:

NIH Study Details

 

Mineralocorticoid Antagonism and Endothelial Dysfunction in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

 

Intervention:

Drug - Spironolactone

Sponsor:

University of Colorado, Denver

Study Phase:

Status:

Not provided

Not Yet Recruiting

Age Group:

30 - 79 years

Enrollment goal:

60

Description:

The proposed research will determine the effectiveness of blocking aldosterone for improving the health and function of arteries in patients with autosomal dominant polycystic kidney disease (ADPKD).  The study also will provide insight into how blocking aldosterone improves artery health by determining the physiological mechanisms (biological reasons) involved.  Overall, the proposed research will provide important new scientific evidence upon which physicians can base recommendations to patients with ADPKD to decrease risk of developing cardiovascular diseases.

Contact:

Michel B Chonchol, MD 303-715-8423 Michel.Chonchol@ucdenver.edu

 

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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