PKD Clinical Trials
NOTE: The clinical trial descriptions below are for informational purposes only. Individuals with PKD should discuss with their health care providers whether or not participation in a clinical trial is appropriate and advisable. You or your health care provider can also discuss this with the Principal Investigator of the study in order to determine if you are eligible and if it is advisable for you to enroll.
Understanding Clinical Trials
Choosing to participate in a clinical trial is an important personal decision. To help make the decision, this website provides detailed information about clinical trials in general. Individuals with PKD should discuss with their health care providers whether or not participation in a clinical trial is appropriate and advisable for them. After identifying some trial options, the next step is to contact the study research staff and ask questions about specific trials.
In order to participate in one of the clinical trials listed below, an individual must have a diagnosis of PKD. Making the decision to seek an official diagnosis, which then becomes part of an individual's medical records, can be difficult. For more information about making a clinical diagnosis of PKD, please click here.
Definitions
Interventional Study Drugs prescribed and their effect on PKD disease progression monitored
Observational Study No drugs given. PKD disease progression and/or other parameters monitored.
Large Multi-site Clinical Trials for ADPKD Patients
HALT PKD Progression of PKD (HALT PKD)
Intervention: Drugs -- lisonopril, telmisartan
Sponsors: NIDDK, Boehringer-Ingelheim Pharmaceuticals, Merck, PKD Foundation
Status: Recruiting
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.
Website
Contact: Robin Woltman at 314-362-1318 or robinw@wubios.wustl.edu
Gigi Flynn at 314-362-3737, or gigi@wubios.wustl.edu
Heart Disease and PKD: DID YOU KNOW ... ?
Tolvaptan Tempo 3 / 4 Trial
Intervention: Drug -- Tolvaptan
Sponsors: Otsuka Pharmaceutical Development & Commercialization, Inc.; Otsuka Pharmaceutical Co., Ltd.
Study Phase: III
Status: Recruiting
Enrollment goal: 1,500
Description: To evaluate the long-term effectiveness and safety of Tolvaptan by monitoring total kidney volume and cyst volume by magnetic resonance imaging and measuring a composite of clinical markers in ADPKD patients.
Website
Contact: Within North America: Kristin Cornwell at 866-712-5837 or kristin.cornwell@mayo.edu .
Outside North America: 800-712-58370
Study sites:
Link
Link
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: Recruiting
Enrollment goal: 300
Description: To evaluate the anti-proliferative drug Everolimus as a means to slow disease progression in PKD by measuring total kidney and cyst volumes and changes in renal function.
Website
Contact: Novartis, Freiburg, Germany 41-61-324-1111
Pilot Studies for ADPKD patients
These studies involve a small number of participants and are usually limited to those living near the study site.
Endothelial Dysfunction in PKD
Observational: Looking for patients with early ADPKD, i.e., preserved kidney function and normal blood pressure.
Sponsor: NIH
Status: Recruiting
Enrollment goal: 50
Description: To determine whether PKD, in the absence of high blood pressure and with preserved kidney function, is associated with cardiovascular risk factors such as endothelial dysfunction.
Contact: Vandana Menon, M.D., Ph.D., at 617-636-8791.
ADPKD Pain Study
Intervention: Procedure: Videothracoscopic Splanchnicectomy (VSPL)
Sponsor: Mayo Clinic
Study Phase: II
Status: Recruiting
Enrollment goal: 20
Description: To evaluate the effectiveness of VSLP procedure for pain management in patients with ADPKD
Website
Contact: Beth B. Bjornson at 507-255-7768 or bjornson.beth@mayo.edu
Pilot Study of Rapamycin as Treatment for ADPKD
Intervention: Drug -- Rapamycin
Sponsor: The Cleveland Clinic
Study Phase: I & II
Status: Recruiting
Enrollment goal: 30
Description: To compare the effects of an anti-proliferative, anti-angiogenesis and tumor-progression-blocking agent on disease progression in ADPKD. Due to the intensive and frequent testing schedule, enrollment is limited to local patients only.
Website
Contact: Susan Wirth at 216-444-0124 or wirths@ccf.org
Sirolimus (Rapamune) for ADPKD
Intervention: Drug -- Rapamycin
Sponsor: The University of Zurich
Study Phase: III
Status: Recruiting
Enrollment goal: 100
Description: To investigate whether a low dose of sirolimus retards cyst growth and slows renal functional deterioration in patients with ADPKD.
Website
Contact: Andreas Serra, MD at +41 44 255 33 84 or andreas.serra@usz.ch
Sirolimus Treatment in Patients with ADPKD: Renal Efficacy and Safety
Intervention: Drug -- Sirolimus
Sponsor: Mario Negri Institute for Pharmacological Research
Study Phase: II
Status: Recruiting
Enrollment goal: 16
Description: To determine if the addition of sirolimus to conventional high blood pressure therapy can prevent a further increase in or can reduce total kidney and cyst volumes in ADPKD adults with normal and mild to moderate renal insufficiency.
Website
Contact: Norberto Perico, MD at 0039 035-45351 or perico@marionegri.it
Somatostatin in Polycystic Kidney: A Long-term Three Year Follow-up Study
Intervention: Drug -- Long-acting somatostatin
Sponsor: Mario Negri Institute for Pharmacological Research
Study Phase: III
Status: Recruiting
Enrollment goal: 66
Description: To compare the effects on disease progression of a three year treatment regimen using long-acting somatostatin or placebo in patients with ADPKD and normal renal function or mild to moderate renal insufficiency.
Website
Contact: Norberto Perico, MD at 0039 035-45351 or perico@marionegri.it
Octreotide in Severe Polycystic Liver Disease
Intervention: Drug: Octreotide (long-acting somatostatin)
Sponsor: Mayo Clinic; Novartis
Study Phase: II & III
Status: Recruiting
Enrollment goal: 42
Description: To evaluate the effect of Octreotide LAR on liver volumes of patients with severe polycystic liver disease who are not candidates for or decline surgical treatments such as liver cyst fenestration or liver transplantation.
Website
Contact: Marie Hogan, M.D., Ph.D. at 507-538-9851 or Linda Page at 507-538-9851
Lanreotide as Treatment of Polycystic Liver Disease
Intervention: Lanreotide
Sponsor: Radboud University
Study Phase: II & III
Status: Active, but not recruiting
Enrollment goal: 38
Description: To assess the efficacy of lanreotide in controlling total liver volume in patients with polycystic livers.
Website
Contact: info@livercyst.org
Effect of Statin Therapy on Disease Progression in ADPKD
Intervention: Drug -- Pravastatin
Sponsor: University of Colorado at Denver and Health Sciences Center
Study Phase: III
Status: Recruiting
Enrollment goal: 100
Description: To determine the effect of pravastatin treatment on renal and cardiovascular disease over a 3-year study period in children and young adults aged 8-21 years with ADPKD.
Website
Contact: Melissa Cadnapaphornchai, M.D. at 303-724-1690 or pkd.nurse@uchsc.edu
Linda Perkins at 303-724-1690 or pkd.nurse@uchsc.edu. You may also call toll-free at 1-877-765-9297.
The Effect of High and Low Sodium Intake on Urinary Aquaportin-2 in ADPKD
Intervention: Drug -- High or low sodium diet
Sponsor: Holstebro Hospital, Denmark
Status: Recruiting
Enrollment goal: 25
Description: To examine the effect of high and low diets on kidney function and on hormones that affect the kidney.
Website
Contact: Carolina Cannillo, M.D. at 1145099125428 or cacan@ringamt.dk
CCB Safety Study in Treatment of Hypertension in ADPKD
Intervention: Drugs -- Candesartan; Candesartan + Cilnidipine; Candesartan + non-calcium channel blocker (CCB)
Sponsor: Kyorin University, Japan
Study Phase: IV
Status: Recruiting
Enrollment goal: 150
Description: To evaluate the safety and efficacy of calcium channel blocker Candesartan in treatment of hypertension in ADPKD patients.
Website
Contact: Eiji Higashihara, M.D. at +81-422-47-5511, ext. 5813 or ehigashi@kyorin-u.ac.jp
Current Clinical Trials for ARPKD patients
Evaluation of ARPKD and Congenital Hepatic Fibrosis
Observational
Sponsor: National Human Genome Project (NHGRI)
Status: Recruiting
Enrollment goal: 200
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.
Website
Contact: Patient Recruitment and Public Liaison Office at 1- 800-411 1222 or prpl@mail.cc.nih.gov
Chronic Kidney Disease in Children Prospective Cohort Study (CKiD)
Observational
Sponsors: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institute of Neurological Disorders and Stroke (NINDS), National Institute of Child Health and Human Development (NICHD) and National Heart, Lung, and Blood Institute (NHLBI)
Status: Recruiting
Enrollment goal: 540
Description: To determine the risk factors for decline in kidney function and to define how a progressive decline in kidney function impacts neurocognitive function and behavior; the risk factors for cardiovascular disease; growth failure and its associated morbidity.
Website
Contact: Susan Furth, M.D., P.D. at 410-502-7964 or sfurth@jhmi.edu
Brad Warady, M.D. at 816-234-3812 or bwarady@cmh.edu
The ARPKD Clinical and Genetic Resource
Observational
Sponsors: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Status: Recruiting
Enrollment goal: 200
Description: The Core has three primary objectives: To extend the observational study of ARPKD initiated by the North American ARPKD Database; to provide a mechanism for genetic evaluation of patients with both classic ARPKD and unusual phenotypes of recessive PKD; and to develop educational tools for physicians and patients regarding the natural history, cause, development and effects of the disease, genetic testing, and clinical trials applicable to ARPKD.
Website
Contact: Lisa M. Guay-Woodford, M.D. at 205-934-7308 or lgw@uab.edu
Teresa Chacana, B.S.N. at 205-934-7649 or tchacana@uab.edu
Transplant-related Studies
Transplant Registry: Patients Who May Require Transplantation and Those Who Have Undergone Transplantation of Liver, Kidney and/or Pancreas
Observational
Sponsor: Virginia Commonwealth University
Status: Recruiting
Enrollment goal: 2,000
Description: To make a systematic review of medical records of patients (with patient consent) in the Virginia commonwealth University Health System who have received a liver, kidney or pancreas or who may require one and to enter the data into a secure database.
Website
Contact: Robert Fisher, M.D. at 804-828-2461
Cheryl Rodgers, BSN, RN 804-828-2463
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