Updated: 5/9/2016

Interventional Studies – Currently Recruiting

















Not Listed

ADPKD Pain Study

Some patients with autosomal dominant polycystic kidney disease (ADPKD) have intractable disabling chronic kidney pain. Among methods used to manage these patients, removal of the nerve supply to the kidney by Videothoracoscopic excision of Splanchnic nerve (videothoracoscopic splanchnicectomy — VSPL) is one of the most promising procedures.

Procedure: Video Thorascopic Splanchnicectomy (VSPL)

Study Phase:
Phase 2

Age Group:
18 and older

Mayo Clinic, Rochester, Minn.

Participant Information:

  • Must be able to make multiple (3) visits to Mayo Clinic Rochester; urine and blood tests taken at each visit.
  • MRI of the kidneys will be performed
  • Multiple questionnaires must be completed
  • VSPL operation will be done within 1 week of initial visit and is considered part of your clinical care and not part of the research. In this procedure, the surgeon approaches the nerves supplying the kidneys through the chest wall using 3 small incisions. The surgeon locates the nerves, cuts a piece of them and removes it from the chest cavity. You will be released from the hospital when your pain is controlled on oral medications.
  • Must be able to cover the cost of surgery and post-op care

Inclusion Criteria

  • Diagnosis of ADPKD
  • History of debilitating kidney pain for at least 6 months other methods of pain management will have been appropriately considered and used when feasible and indicated; fit for general anesthesia; must be able to travel to Mayo Clinic Rochester for 3 visits; must be able to cover the cost of the surgery and post operative care.

Exclusion Criteria

  • Pregnant or nursing
  • Creatinine > 3mg/dl or be hemodialysis dependent
  • Cancer or other major systemic diseases
  • Uncontrolled hypertension (SBP>160; DBP>100)

Contact Information:
Marie C. Hogan, M.D., Ph.D., Principal Investigator


Mesenchymal Stem Cell Transplantation in Patients with Chronic Renal Failure Due to Polycystic Kidney Disease (PKD)

The investigators will assess the 18-month safety and potential efficacy of autologous mesenchymal stem cells (MSCs) as therapy for ADPKD. A total of 6 patients with ADPKD IV injection of high doses 2×106 of autologous mesenchymal stem cells / kg their weight, which will be derived from biopsies of their bone marrow. Assessments will be made at 1, 3, 6, 12 and 18 months after cell injection. Changes in GFR rate were evaluated by scan isotope.

Biological: Intravenous injection, autologous mesenchymal stem cells

Study Phase:
Phase 1

Age Group:
18 to 60 years

Royan Institute, Tehran, Islamic Republic of Iran

Participant Information:

Inclusion Criteria

  • ADPKD symptom
  • ADPKD confirmed with sonography and genetic testing
  • GFR 25-60 mL/min/1.73 m2
  • Ability to understand and willingness to sign consent form

Exclusion Criteria

  • Pregnant or lactating
  • Basis diseases such as diabetes, malignancy, autoimmune
  • Unable to follow post-operative exercise regimen or return for evaluations

Contact Information:
Nasser Aghdami, M.D.,Ph.D.
+98 23562000 ext. 516

Leila Arab, M.D.
+98 23562000 ext. 414


Pasireotide LAR in Severe Polycystic Liver Disease (SOM230)

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.


  • Drug: Pasireotide LAR
  • Drug: Placebo

Study Phase:
Phase 2

Age Group:
18 and older

Mayo Clinic, Rochester, Minn.

Participant Information:

  • Travel to Mayo Clinic, Rochester several times required
  • Primary endpoint assessed at 12 months
  • Patients who receive placebo will crossed over to SOM230, permitting all patients to receive SOM230 for the subsequent 2 years.
  • MRI will be used to assess liver volume at baseline and end of years 1 and 3

Inclusion Criteria

  • Male or female
  • Diagnosis of PLD associated with ADPKD (meeting the Modified Ravine's criteria) or isolated ADPLD (defined by the criteria described by Reynolds et al)
  • Severe PLD defined as a liver volume >4000mL or symptomatic disease due to mass effects from hepatic cysts (must be able to undergo MRI or CT scan to determine this)
  • Not a candidate for or declining surgical intervention
  • Capable of providing informed consent
  • Life expectancy ≥ 12 weeks
  • Patients with a known history of impaired fasting blood glucose (glucose >100 and <126) may be included at the discretion of the PI
  • Adequate end organ function
  • No evidence of significant liver disease
  • Estimated glomerular filtration rate (eGFR) >30 ml/min/m2
  • Serum amylase and lipase ≤ 1.5 x ULN
  • Alkaline phosphatase ≤ 2.5 x ULN
  • Written informed consent obtained prior to any screening procedures
  • Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests and other study procedures.

Exclusion Criteria

  • Known hypersensitivity to SST analogs or any component of the pasireotide LAR or SQ formulations
  • Known malabsorption syndrome, short bowel or chologenic diarrhea not controlled by specific therapeutic means
  • Abnormal coagulation (PT or a PTT elevated by 30% above normal limits)
  • On continuous anticoagulation therapy. Patients who were on anticoagulant therapy must complete a washout period of at least 10 days and have confirmed normal coagulation parameters before study inclusion
  • Symptomatic cholelithiasis
  • Not biochemically euthyroid
  • Known history of hypothyroidism are eligible if they are on adequate and stable re-placement thyroid hormone therapy for at least 3 months
  • Serum magnesium ≥ ULN
  • QT-related exclusion criteria:
  • QTcF at screening > 470 msec
  • History of syncope or family history of idiopathic sudden death
  • Have sustained or clinically significant cardiac arrhythmias
  • Risk factors for Torsades de Pointes such as hypokalemia, hypomagnesemia, cardiac failure, clinically significant/symptomatic bradycardia, or high-grade AV block
  • Concomitant disease(s) that could prolong QT such as autonomic neuropathy (caused by diabetes, or Parkinson's disease), HIV, cirrhosis, uncontrolled hypothyroidism or cardiac failure
  • Family history of long QT syndrome
  • Concomitant medications known to prolong the QT interval.
  • Potassium < or = to 3.5
  • Have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:
    • Uncontrolled diabetes as defined by HbA1c>8%* despite adequate therapy
    • Presence of active or suspected acute or chronic uncontrolled infection or with a history of immunodeficiency, including a positive HIV test result (ELISA and Western blot). An HIV test will not be required; however, previous medical history will be reviewed.
    • Non-malignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with this study treatment.
    • Liver disease such as cirrhosis, decompensated liver disease, chronic active hepatitis or chronic persistent hepatitis.
    • Baseline ALT or AST >3x ULN
    • Life-threatening autoimmune and ischemic disorders.
    • Uncontrolled hypertension
  • Have a history of a primary malignancy, with the exception of locally excised non-melanoma skin cancer and carcinoma in situ of uterine cervix. (Patients who have had no evidence of disease from primary cancer for 3 or more years are allowed to participate in the study.)
  • History of pancreatitis
  • Known history of hepatitis B or C
  • Hepatitis B surface antigen (HbsAg)
  • Hepatitis C antibody (anti-HCV)
  • History of, or current, alcohol misuse/abuse within the past 12 months
  • Known gallbladder or bile duct disease, acute or chronic pancreatitis
  • Have any current or prior medical condition that may interfere with the conduct of the study or the evaluation of its results in the opinion of the Investigator or the Sponsor's Medical Monitor
  • Use of an investigational drug within 1 month prior to dosing
  • History of non-compliance to medical regimens or who are considered potentially unreliable or will not be able to complete the entire study

Contact Information:
Marie Hogan, M.D., Ph.D.

Angela Ihrke


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

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.


  • Drug: Sirolimus
  • Drug: Placebo

Study Phase:
Phase 3

Age Group:
18 and older

Medical University of Vienna – Vienna, Austria

Participant Information:

  • Includes oral drug therapy (Sirolimus)

Inclusion Criteria

  • ADPKD, as confirmed by history, ultrasound, computed- or magnetic resonance tomography
  • Baseline eGFR below 60 mL/min per 1.73m2
  • Negative serum pregnancy test prior to administration of sirolimus and agreement to use contraception throughout the study and three months after
  • Written informed consent

Exclusion Criteria

  • Need for renal replacement therapy
  • Pregnant or lactating
  • Proteinuria as defined by protein:creatinine ratio > 1000 or >1g/d, respectively
  • History of life threatening complications of ADPKD
  • Active systemic or localized major infection
  • Infiltrate or consolidation on chest X-ray
  • Use of any investigational drug or treatment up to 4 weeks prior to enrollment and during study
  • Known allergy/hypersensitivity to sirolimus and/or its derivatives
  • Medication that will interfere with the cytochrome P450 (CYP3A4/CYP3A5) system
  • Total white blood cell count below or equal to 3000/mm3
  • Platelet count below or equal to 100.000/mm3
  • Fasting triglycerides above or equal to 400 mg/dL
  • Fasting total cholesterol above or equal to 300 mg/dL
  • Concomitant glomerular diseases
  • Psychiatric disorders and any condition that might prevent full comprehension of the purposes and risks of the study
  • History of malignancy, with the exception of adequately treated basal cell- and squamous cell carcinoma of the skin
  • HIV positivity

Contact Information:
Markus Riegersperger, M.D.
0043140400 ext. 4391

Gere Sunder-Plassmann, M.D.
0043140400 ext. 4391


Triptolide-Containing Formulation as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Triptolide was shown in experimental studies to inhibit the cyst formation and growth in ADPKD models, while triptolide-containing formulation was revealed to potentially slow the disease progression in several proteinuric ADPKD patients in our clinical practice. It remains to be shown the effect of triptolide-containing formulation on total kidney volume (TKV) enlargement and renal function protection in ADPKD patients. This study's primary outcome measure is renal volume measured by high-resolution MRI.


  • Drug: Triptolide – containing formulation
  • Drug: Placebo

Age Group:
40 to 75 years

Shanghai, China

Participant Information:

Inclusion Criteria

  • Diagnosed with ADPKD
  • Estimated glomerular filtration rate (eGFR) higher than 60 ml/min 1.73m2
  • Documented kidney volume progression with yearly increasing rate more than 6%
  • Informed consent

Exclusion Criteria

  • Female who is planning to become pregnant, who is pregnant or lactating, or who is unwilling to use effective means of contraception
  • Impaired liver function as increased liver enzymes (2-fold above normal values)
  • Uncontrolled hypercholesterolemia or hypertriglyceridaemia
  • Granulocytopenia or thrombocytopenia
  • Hepatitis B, C, or HIV infection
  • Malignancy
  • Nental illness that interfere with the patient ability to comply with the protocol
  • Drug or alcohol abuse
  • Known hypersensitivity to similar drugs as Triptolide – Containing Formulation

Contact Information:
Changlin Mei, M.D.


Ursodeoxycholic Acid as Treatment for Polycystic Liver Disease (CURSOR)

Polycystic liver disease (PLD) is a rare disorder characterized by >20 fluid-filled hepatic cysts. Polycystic livers are present in the combination with renal cysts as a manifestation of autosomal dominant polycystic kidney disease (ADPKD), or isolated in the absence of renal cysts as autosomal dominant polycystic liver disease (ADPLD or PCLD). PLD patients are confronted with symptoms caused by the mass effect of their polycystic liver every day for the rest of their life. There is no standard therapeutic option for symptomatic PLD patients. Current options are fairly invasive or their efficacy is only moderate.

The investigators hypothesize that ursodeosycholic acid (UDCA) is an effective therapeutic tool in reducing liver volume in PLD. The first objective of the study is to demonstrate whether UDCA-therapy is effective in reducing total liver volume in PLD patients. Also, the investigators want to assess whether UDCA modifies quality of life. Finally, the investigators want to assess safety and tolerability.

Ursodeoxycholic Acid

Age Group:
18 to 80 years


  • Radboud University Medical Centre Nijmegen – Nijmegen, Gelderland, Netherlands
  • Academic Medical Centre Amsterdam – Amsterdam, Netherlands
  • Donostia University Hospital – San Sebastian, Spain

Participant Information:

Inclusion Criteria

  • Polycystic liver disease with underlying diagnosis of (PCLD or ADPKD), defined as ≥ 20 liver cysts
  • Total liver volume ≥ 2500 mL
  • Symptomatic defined as ECOG-PS ≥ 1 (2), and having at least three out of ten PCLD symptoms
  • Informed consent, patients are willing and able to comply with the study drug regimen and all other study requirements

Exclusion Criteria

  • Use of oral anticonceptives or estrogen supplementation
  • Use of UDCA in 3 months before baseline
  • Females who are pregnant or breast-feeding or patients of reproductive potential not employing an effective method of birth control.
  • Intervention (aspiration or surgical intervention) within six months before baseline
  • Treatment with somatostatin analogues within six months before baseline
  • Renal dysfunction (MDRD-GFR < 30 ml/min/1.73m2)
  • Patients with a kidney transplant
  • Hypersensitivity reaction to UDCA or patients with galactose-intolerance, lactase deficiency or glucose-galactose malabsorption
  • Acute cholecystitis or frequent biliary colic attacks
  • Acute stomach or duodenal ulcers
  • Inflammation of small intestine or colon
  • Use of drugs that can interact with UDCA, such as colestyramine, aluminium hydroxide or cyclosporin
  • Enrolment in another clinical trial of an investigational agent while participating in this study
  • History or other evidence of severe illness or any other conditions which would make the patient, in the opinion of the investigator, unsuitable for the study
  • Mental illness that interferes with the patient ability to comply with the protocol

Contact Information:
Hedwig MA D'Agnolo


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

Cardiovascular complications are currently the major causes of mortality among patients with autosomal dominant polycystic kidney disease (ADPKD). Therefore, testing valid interventions to reduce morbidity and mortality within this population is of high priority. It is well documented that endothelial dysfunction coupled with abnormalities in markers of oxidative stress and inflammation develops early in ADPKD even before there is a significant decline in kidney function. Aldosterone levels are increased in patients with ADPKD and may contribute to cardiovascular disease by impairing endothelial function, and reducing vascular compliance. Of note, aldosterone antagonists have been shown to improve endothelial dysfunction in a number of studies in other patient populations. However, there have been no clinical interventional studies specifically targeting endothelial dysfunction in ADPKD.

The main goal of this trial is to establish the efficacy of an aldosterone antagonist (spironolactone) for treating vascular endothelial dysfunction and large elastic artery stiffness in ADPKD patients with preserve kidney function. A key secondary goal is to determine the integrative physiological (i.e., whole limb/artery tomolecular) mechanisms underlying the beneficial effects of spironolactone.


  • Drug: Spironolactone
  • Drug: Placebo

Age Group:
30 to 79 years

University of Colorado, Denver, Col.

Participant Information:

Inclusion Criteria

  • Aged 30-79 years
  • Adults with ADPKD diagnosis based on Ravine criteria aged ≥ 30 years
  • Estimated glomerular filtration rate ≥ 60 ml/min/1.73m2
  • Hypertension defined as a systolic BP > 130 mm Hg and/or diastolic BP > 80 mmHg based on 3 separate measurements within the past year and currently on a minimum dose of an angiotensin converting enzyme inhibitor (minimum dose 10 mg P.O qd) or angiotensin receptor blocker (i.e., Losartan 25 mg P.O qd)
  • If using antioxidants and/or omega-3 fatty acids, must discontinue 4 weeks prior to participation
  • Free from alcohol dependence or abuse
  • Mini-mental state examination score ≥ 24; ability to provide informed consent
  • BMI < 40 kg/m2 (FMD measurements can be inaccurate in severely obese patients)
  • Not taking medications that interact with agents administered during experimental sessions (e.g., sildenafil interacts with nitroglycerin)
  • Use of birth control in women of childbearing potential

Exclusion Criteria

  • Average serum potassium >5.5 millequivalents or any single serum potassium > 6.0 millequivalents within the previous 6 months
  • Receiving an aldosterone antagonist within the previous 6 months
  • Use of a potassium sparing diuretic
  • Uncontrolled hypertension
  • History of liver disease
  • History of heart failure (EF < 35%)
  • History of hospitalizations within the last 3 months
  • Active infection or antibiotic therapy
  • Warfarin use
  • Immunosuppressive therapy within the last year
  • Pregnancy

Contact Information:
Michel B Chonchol, M.D.


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

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.


  • Drug: Everolimus
  • Drug: Calcineurin inhibitors maintenance

Age Group:
18 to 75 years

Bicêtre Hospital - Le Kremlin-Bicêtre, France

Participant Information:

  • Both affected and non-affected relatives of PKD1 and PKD2 negative ADPKD patients will be recruited
  • DNA samples will be collected

Inclusion Criteria:

  • Recipients of a first kidney graft between 6 month and 5 years ago with a stable eGFR above 30 ml/min/1.73m2
  • Contraception for female recipients to avoid pregnancy
  • Valid health Insurance during the study period

Exclusion Criteria:

  • Signed informed consent not obtained

Contact Information:
Hélène François, M.D., Ph.D.
+33(1)45 21 27 22


Limited to participants in prior Tolvaptan clinical trials.

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

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.

Drug: Tolvaptan (OPC-41061)

Age Group:
18 and older

No locations listed – see website for details

Participant Information:

Inclusion Criteria:

  • Confirmed diagnosis of ADPKD (during participation in prior tolvaptan trials) who have:
    • Completed and transferred from the double-blind Trial 156-13-210 (12-month period including post treatment follow-up, regardless of whether this was on-treatment or off-treatment), or
    • Completed Trial 156-08-271 or a prior tolvaptan trial, or
    • Interrupted or discontinued treatment in a prior tolvaptan ADPKD trial other than Trial 156-13-210. Subjects may be enrolled with the medical monitor approval, and additional close monitoring may be required at the beginning of the study
    • Estimated glomerular filtration rate (eGFR) ≥ 20 mL/min/1.73m2 within 45 days of the baseline visit. Subjects who have an eGFR between 15 and 19 mL/min/1.73m2 may be enrolled with medical monitor approval
    • Diagnosis of ADPKD by modified Pei-Ravine criteria

Exclusion Criteria:

  • Need for chronic diuretic use
  • Hepatic impairment based on liver function assessments other than that expected for ADPKD with cystic liver disease
  • Women of childbearing potential (WOCBP) who do not agree to practice 2 different methods of birth control or remain abstinent during the trial and for 30 days after the last dose of IMP
  • Women who are breast-feeding and/or who have a positive pregnancy test result prior to receiving IMP
  • Subjects with contraindications to required trial assessments (contraindications to optional assessments, eg, MRI are not a limitation)
  • Subjects who in the opinion of the Investigator or the Medical Monitor, have a medical history or medical finding inconsistent with safety or trial compliance

Contact Information:
Not provided


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

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). The total number of study subjects will be 100. All of them have diagnosed with ADPKD and hypertension. Patients will be randomized 1:1 (50 with radiofrequency ablation (RFA), 50 only with drugs). Change in average office-based measurements of systolic blood pressure (SBP), average 24-hour systolic blood pressure by ambulatory blood pressure monitoring (ABPM) , incidence of office systolic blood pressure reductions of ≥10, ≥15 and ≥20 mm Hg , office diastolic blood pressure (DBP), number and dosage of blood pressure tablets, total kidney volume (TKV), total cyst volume (TCV), pain related to cystic kidneys and renal function, will be assessed at 12 months of follow-up. The safety variables will be assessed at every visit of follow-up.


  • Procedure: Renal Sympathetic Denervation
  • Drug: Antihypertensive Drugs

Age Group:
20 to 60 years

Shanghai Changzheng Hospital - Shanghai, China

Participant Information:

Inclusion Criteria:

  • Patients with ADPKD.
  • Having hypertension, defined as systolic blood pressure ≥140 mm Hg and/or diastolic blood pressure ≥90 mm Hg, and currently using 2 antihypertensive drugs and receiving a stable antihypertensive treatment regimen without change in dose or medication in the previous 30 days.
  • Glomerular Filtration Rate (GFR) ≥30 ml/min/1.73 m2, estimated from serum creatinine using the Chronic Kidney Disease Epidemiology collaboration(CKD-EPI) equation.
  • Have followed-up kidney and cyst volume at least 6 months in Shanghai Changzheng Hospital.
  • Signed Informed Consent after being informed.

Exclusion Criteria:

  • Documented renal vascular disease.
  • Congenital absence of a kidney.
  • Systemic illness with renal involvement.
  • Spot urine albumin-to-creatinine ratio of >0.5 g/g and/or findings suggestive of kidney disease other than ADPKD.
  • Exclusions specific to MRI acquisition and measurement: cardiac pacemaker, presence of MRI incompatible metallic clips or other material, excessive body weight, untreatable claustrophobia.
  • Contraindications to the catheter-based renal denervation procedure by RFA, including allergy to radioiodinated contrast agents. Anatomical abnormalities of the renal arteries which preclude RFA: presence in either kidneys of multiple main renal arteries, main renal artery stenosis >50%, or main renal arteries of
  • Contraindications on ethical grounds.
  • Women who are pregnant or breast feeding.
  • Intention to become pregnant during the course of the study.
  • Lack of safe contraception: Female subjects of childbearing potential, not using and not willing to continue using a medically reliable method of contraception for the entire study duration, such as oral, injectable, or implantable contraceptives, or intrauterine contraceptive devices, or who are not using any other method considered sufficiently reliable by the investigator in individual cases (Female subjects who are surgically sterilized/hysterectomized or post-menopausal for longer than 2 years are not considered as being of child bearing potential).
  • Other clinically significant concomitant disease states (hepatic dysfunction, cardiovascular disease, metastatic cancer).
  • Known or suspected non-compliance, drug or alcohol abuse.
  • Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia or confusional state of the subject.
  • Participation in another study with investigational drug within the 30 days preceding and during the present study.
  • Previous enrollment into the current study.
  • Enrollment of the investigator, his/her family members, employees and other dependent persons.

Contact Information:
Changlin Mei, M.D.
+86 21 81885391

Yiyi Ma, Master


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

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.

Procedure: Renal Denervation

Age Group:
18 years and older

Odense University Hospital - Odense, Denmark

Participant Information:

Inclusion Criteria:

  • Pain limiting daily activities in spite of pain killers, ADPKD

Exclusion Criteria:

  • Pregnancy

Contact Information:
Marie Blicher, M.D.
+ 45 65415305

Hans Dieperink, M.D.


Sirolimus for Massive Polycystic Liver (SILVER)

Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common causes of end stage renal disease (ESRD), affecting an estimated 0.2% of population. Of ADPKD patients, 58% in 15-24 year, 85% in 25-34 year, and 94% in 35-46 year olds suffer from polycystic liver in addition to polycystic kidneys. Several anti-proliferative drugs have been used in clinical trials to stop cyst growth both in liver and kidneys. Among them, octreotide and sirolimus have been shown to be one of the most promising drugs to reduce cyst volume. Sirolimus already has been used as one of the most potential oral immunosuppressants. Moreover, the serum trough level is quite easy to measure. Sirolimus is the mTOR inhibitor that has been proven to be effective in reducing cyst growth both in animal models. However, its efficacy and safety is not well proven in previous studies. 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.

Drug: Sirolimus

Age Group:
18 to 65 years

Seoul National University Hospital – Seoul, Korea

Participant Information:

Inclusion Criteria:

  • Patients diagnosed as ADPKD based upon the unified criteria for ultrasonographic diagnosis of ADPKD
  • Polycystic liver with total liver volume > 2500 mL or symptomatic polycystic liver
  • Estimated glomerular filtration rate (IDMS-traceable MDRD equation) >= 30 mL/min/1.73m2

Exclusion Criteria:

  • Concomitant systemic renal parenchymal or urinary tract disease (random urine albumin-to-creatinine ratio > 500 mg/g)
  • WBC < 4,000/uL, platelet < 100,000/uL, or hemoglobin < 10.0 g/dL
  • Diabetes mellitus, cancer, or psychiatric disorder
  • Increased liver enzymes (2-fold above normal value)
  • Hypercholesterolemia (fasting cholesterol > 200mg/dL) or hypertriglyceridemia (>150 mg/dL) not controlled by lipid lowering therapy
  • Infection with hepatitis B, C, HIV
  • Any condition that could prevent full comprehension of the purpose and risks of the study
  • Pregnant or lactating women or fertile women without effective contraception
  • History of intervention, such as cyst aspiration or embolization in past 1 year

Contact Information:
Curie Ahn, M.D., Ph.D.


Lanreotide in Polycystic Kidney Disease Study (LIPS)

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.


  • Drug: Lanreotide
  • Drug: Saline

Age Group:
18 and older

Necker Hospital - Paris, France

Participant Information:

Inclusion Criteria:

  • ADPKD (clinical, familial, imaging grounds)
  • Measured GFR: 30 to 89 ml/mn/1.73m2
  • Affiliated with health insurance
  • Written informed consent

Exclusion Criteria:

  • Iohexol /iodine allergy
  • Diabetes mellitus
  • Other associated nephropathy suspected
  • Previous malignant disease
  • Cholelithiasis
  • Uncontrolled hypertension (BP>160/100 mmHg)
  • Cardiac failurev
  • Liver failure
  • Psychiatric illness
  • Pregnancy, lactation, lack of contraception
  • Use of somatostatin analogs during the last 6 months

Contact Information:
Dominique Joly, M.D., Ph.D.
1 44 49 54 15 ext. +33

Laurence Lecomte, Ph.D.
1 71 19 64 94 ext. +33


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

The primary purpose of this study is to determine the safety, tolerability and plasma pharmacokinetics of KD019 in ADPKD patients. Changes in kidney function will also be evaluated. The phase 1b portion has been completed and the best selected daily dose was determined to be 100 mg daily. The phase 2a portion of the study is now enrolling using an alternate dosing schedule of 150 mg given every Monday, Wednesday and Friday. This is a 28 day daily dosing study with an option to continue through 24 months of KD019 dosing. All participants receive active KD019 study drug. KD019 is an oral tablet that comes in 50 mg, 100 mg, and 150 mg strength tablets. Study participants will have an MRI of the abdomen at screening and every 6 months to explore the effects of KD019. An echocardiogram will be performed at screening, Day 26, month 3, 6 and every 6 months thereafter.


Drug: KD019

Study Phase:
Phase 2a

Age Group:
18 to 55 years


  • UCLA Medical Center – Los Angeles, Calif.
  • Beth Israel Deaconess Medical Center – Boston, Mass.
  • Mayo Clinic, Rochester, Minn.
  • Washington University School of Medicine – St. Louis, Mo.
  • Cleveland Clinic – Cleveland, Ohio
  • University of Virginia - Charlottesville, Va.
  • Medical College of Wisconsin – Milwaukee, Wis.
  • University of Kansas Medical Center, Kansas City, Kan.

Participant Information:

  • Must be available to visit one of the study centers listed above
  • Will have various tests performed including MRI of the abdomen and echocardiogram

Inclusion Criteria

  • Confirmed diagnosis of ADPKD; GFR ≥ 50 mL/min/1.73 m2; cysts must be at least 1 cm in size
  • Adequate bone marrow, kidney, and liver function
  • Must agree to use two forms of birth control for those of child bearing potential

Exclusion Criteria

  • Previous partial or total nephrectomy
  • Tuberous sclerosis, Hippel-Lindau disease, or acquired cystic disease
  • Congenital absence of one kidney and/or need for dialysis
  • Presence of renal or hepatic calculi (stones) causing symptoms
  • Received any investigational therapy within 30 days prior to study entry
  • Active treatment (within 4 weeks of study entry) of urinary tract infection
  • Known to be immunocompromised
  • Pregnant or nursing

Contact Information:
University of California, Los Angeles, Los Angeles, Calif.
Fahad Sheckley

Beth Israel Deaconess Medical Center, Boston, Mass.
Alice Lee

Mayo Clinic, Rochester Minn.
Lisa Bungum

Washington University, St. Louis, Mo.
Alaina Thompson

Cleveland Clinic, Cleveland, Ohio
Kimberly Mackay

University of Virginia, Charlottesville, Va.
Lisa Johnson, BA, CCRC

Medical College of Wisconsin, Milwaukee, Wis.
Sonia Maldonado-Schmidt, RN

University of Kansas Medical Center
Cathy Creed, RN


Pilot Study of Niacinamide (Vitamin B3) in Polycystic Kidney Disease (NIAC-PKD2)

The purpose of this study is to observe the effects of niacinamide on markers of kidney injury, inflammation, kidney cyst growth and kidney function.

Watch a video of investigator Alan Yu, M.D. discuss the study.


  • Dietary Supplement: Niacinamide (Vitamin B3)
  • Drug: Placebo

Study Phase:
Phase 2

Age Group:
18 to 60 years

University of Kansas Medical Center, Kansas City, Kan.

Participant Information:

Inclusion Criteria

  • Confirmed diagnosis of autosomal dominant polycystic kidney disease (ADPKD)
  • Estimated glomerular filtration rate (eGFR) > 50 ml/min/1.73m2 as determined from the serum creatinine by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
  • Provide Informed consent

Exclusion Criteria

  • History of liver disease or abnormal liver function test
  • Heavy alcohol intake
  • Chronic diarrhea or malabsorption syndrome
  • Thrombocytopenia
  • Hypophosphatemia
  • Pregnancy or lactation or plan to become pregnant during the study
  • Treatment with anti-epileptic drugs
  • Treatment with tolvaptan, current or within 2 months prior to screening
  • Participation in another interventional trial currently or within 2 months prior to screening

Exclusions specific to magnetic resonance (MR) imaging acquisition and measurement

  • Partial or total nephrectomy or renal cyst reduction (including aspiration) done
  • Cardiac pacemaker
  • Presence of MR incompatible metallic clips (e.g. clipped cerebral aneurysm)
  • Body weight >159 kg (350 lbs) or untreatable claustrophobia

Contact Information:
Cathy Creed, Rn, BSN


Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney Disease (PIOPKD)

Clinical trial to test the safety and efficacy of low dose (15 mg/day) pioglitazone (Actos) on ADPKD progression. Pioglitazone is currently used in clinical practice to treat diabetes. This is double-bind, 2 year cross-over study. Each volunteer will be on placebo for one year and pioglitazone for one year. Volunteers will be randomized as to whether the first year will be drug or placebo. Primary efficacy end point will be kidney volume by MRI.

Currently enrolling volunteers with ADPKD.

Age Group:

Indiana University School of Medicine, Indianapolis

Participant Information:

Inclusion Criteria:

  • Male or female ADPKD patients aged 18-45
  • eGFR (kidney function)_at or above 60 ml/min
  • Normal liver enzyme (function) on blood tests
  • Fasting blood sugar levels between 70 and 120
  • For female patients, a willingness to use double contraception to avoid pregnancy while in study
  • Able to give informed consent
  • Abdominal discomfort or enlarged kidneys on previous tests

Exclusion Criteria:

  • Diabetes
  • Uncontrolled hypertension, defined as systolic > 150, diastolic > 90 despite an attempt by physician to titrate medications
  • History of impaired heart function or heart disease
  • Systemic illness requiring immunosuppressive or anti-inflammatory agents
  • Congenital absence of a kidney or history of a total nephrectomy
  • History of cyst reduction or partial nephrectomy
  • History of renal cyst aspiration within the previous year
  • History of bladder cancer, or blood in the urine
  • Inability to undergo MRI due to implantable devices (for example, hip replacement) or foreign objects that preclude MRI
  • Active renal transplant
  • Allergy or sensitivity to any of the components of the test materials
  • In nursing home or prison
  • Currently pregnant or plans to become pregnant during the study

Visits will be at least every 3 months over two years with a possibility of additional checkups if abnormal blood tests are found.

You will be compensated $10 at each completed visit for parking and your time.

Contact Information:
Kristen Ponsler-Sipes

Bonnie Blazer-Yost


Your Organization

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©2019, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.

©2019, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.