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.

Updated: 03/13/2013

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)
Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease (OVERTURE)
Sirolimus in ADPKD and Severe Renal Insufficiency
Polycystic Liver Disease in Kidney Transplant
Bosutinib for ADPKD
Open-Label Tolvaptan Study in Subjects With ADPKD
8-Week Study of Tolvaptan Dose Forms in ADPKD (NOCTURNE)
Randomized Clinical Trial of Triptolide Woldifii for ADPKD
ADPKD Pain Study
Evaluation of ARPKD and Congenital Hepatic Fibrosis
Everolimus on CKD Progression ADPKD
Lanreotide Augogel in the Treatment of Symptomatic Polycystic Liver Disease (LOCKCYST)
Efficacy Study of Water Drinking on PKD Progression (ESWP)
Core A: The Hepato/Renal Fibrocystic Diseases Translational Resource
Somatostatin Analogues as a Volume Reducing Treatment of Polycystic Livers (RESOLVE)
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)

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

Interventional
Sponsor:	Kadmon Corporation, LLC
Study Phase:	I & II
Status:	Recruiting
Age Group:	18 to 50
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)

Interventional
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

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

Observational
Sponsor:	Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Phase:
Status:	Unknown
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:	OVERTURE Study Info: OVERTUREStudyinfo@mmgct.com
Website:	NIH Study Details

Sirolimus in ADPKD and Severe Renal Insufficiency (SIRENA - II)

Intervention:	Drug - Sirolimus
Sponsor:	Mario Negri Institute for Pharmacological Research
Study Phase:	II & III
Status:	Recruiting
Age Group:	18-80
Enrollment goal:	40
Description:	The general aim of this study in adults with ADPKD and severe renal insufficiency (GFR 40-15 ml/min/1.73 m2) is to evaluate the safety and efficacy of sirolimus in slowing renal functional decline as compared to conventional therapy.
Contact:	Norberto Perico, M.D. 0039-035-45351 or norbertoperico@marionegri.it
Website:	NIH Study Details

Polycystic Liver Disease in Kidney Transplant

Intervention:	Drugs - Tacrolimus, sirolimus
Sponsor:	Mayo Clinic/Wyeth
Study Phase:	II
Status:	Recruiting
Age Group:	18 years and older
Enrollment goal:	68
Description:	The purpose of this study is to see if one kind of immunosuppressive drug is better for the patient’s polycystic liver disease than another type.
Contact:	Qi Qian, M.D. 507-284-2511 or qian.qi@mayo.edu Aleisha Chappell 507-266-8493 or chappell.aleisha@mayo.edu
Website:	NIH Study Details

Bosutinib for ADPKD

Intervention:	Drug - Bosutinib
Sponsor:	Pfizer
Study Phase:	II
Status:	Recruiting
Age Group:	18-50
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

Open-Label Tolvaptan Study in Subjects With ADPKD

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

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

Observational
Sponsors:	Otsuka Pharmaceutical Development & Commercialization, Inc.; Otsuka Pharmaceutical Co., Ltd.
Status:	Currently Enrolling
Age Group:	12-70
Enrollment goal:	3,000
Description:	The purpose of this study is to collect characteristics of patients with ADPKD across a broad population over time to better undertand disease progression (signs, symptoms and outcomes). Association of 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.
Websites:	NIH Study Details

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

Intervention:	Drug - Tolvaptan
Sponsors:	Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Phase:	II
Status:	Active
Age Group:	18-50
Enrollment goal:	180
Description:	To compare the short-term effects of two tolvaptan formulations in patients with ADPKD.
Websites:	NIH Study Details

Randomized Clinical Trial of Triptolide Woldifii for ADPKD

Intervention:	Drugs - Tripterygium wilfordii, Emodin
Sponsor:	Nanjing University School of Medicine, China
Study Phase:	II
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, Master 00862586030066 or yaoxiadan@hotmail.com
Website:	NIH Study Details

ADPKD Pain Study

Intervention:	Procedure - Videothoracoscopic 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:	Marie C. Hogan, M.D., Ph.D. 507-266-1963 or hogan.marie@mayo.edu
Website:	NIH Study Details

Evaluation of ARPKD and Congenital Hepatic Fibrosis

Observational
Sponsor:	National Human Genome Project (NHGRI)
Status:	Recruiting
Age Group:	6 months – 80 years
Enrollment goal:	500
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 1- 800-411 1222 or prpl@mail.cc.nih.gov
Website:	NIH Study Details

Everolimus on CKD Progression ADPKD

Interventional
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

Lanreotide Augogel in the Treatment of Symptomatic Polycystic Liver Disease (LOCKCYST)

Interventional
Sponsor:	Univerity of Ziekenhuizen Leven / Ipsen
Study Phase:	II and III
Status:	Recruiting
Age Group:	18 and older
Enrollment goal:	62
Description:	To evaluate the efiicacy and safety of laneotide autogel 90 mg/4 weeks in the treatment of symptomatic polycystic liver disease, including a dose escalation at month six to 120mg/4 weeks for non-responders
Website:	NIH Study Details

Efficacy Study of Water Drinking on PKD Progression (ESWP)

Observational
Sponsor:	Kyorin University
Status:	Recruiting
Age Group:	20-65
Enrollment goal:	30
Description:	To evaluate the long-term effect of water diuresis induced by oral water intake on kidney volume and renal function in ADPKD patients
Contact:	Eiji Higashihara, MD +81-422-47-5511, ext 5813 or ehigashi@ks.kyorin-u.ac.jp Kikuo Natahara, MD +81-422-47-5511, ext 7445 or kinuta@ks.kyorin-u.ac.jp
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 Analogues as a Volume Reducing Treatment of Polycystic Livers (RESOLVE)

Observational	Drug - Lanreotide
Sponsor:	Radboud University
Collaborator:	Ipsen Pharmaceutical
Status:	Recruiting
Age Group:	18 to 70
Enrollment goal:	43
Description:	To determine the effect of Lanreotide on polycystic liver and kidneys in patients with ADPKD.
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)

Interventional
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 wherther 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)

Interventional
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 ADPKA patients with massive polycystic liver.
Contact:	Curie Ahn, MD, PhD 82-2-2072-2222 or curie@snu.ac.kr
Website:	NIH Study Details