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Sirolimus for Massive Polycystic Liver (SILVER)

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ClinicalTrials.gov Identifier: NCT01680250
Recruitment Status : Unknown
Verified September 2012 by Seoul National University Hospital.
Recruitment status was:  Recruiting
First Posted : September 7, 2012
Last Update Posted : September 7, 2012
Sponsor:
Collaborator:
Wyeth is now a wholly owned subsidiary of Pfizer
Information provided by (Responsible Party):
Seoul National University Hospital

Study Description
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Brief Summary:
The purpose of this study is to evaluate the effectiveness and safety of Sirolimus in reducing liver volume in autosomal dominant polycystic kidney disease.

Condition or disease Intervention/treatment Phase
Polycystic Kidney Diseases Drug: Sirolimus Phase 2 Phase 3

Detailed Description:
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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 44 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Prospective Clinical Trial to Evaluate the Effectiveness and Safety of Sirolimus to Reduce Cyst Growth in ADPKD Patients With Massive Polycystic Liver
Study Start Date : September 2011
Estimated Primary Completion Date : September 2014
Estimated Study Completion Date : August 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Diseases
U.S. FDA Resources

Arms and Interventions
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Arm Intervention/treatment
Experimental: Sirolimus
Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL
 Drug: Sirolimus
Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months
Other Name: Rapamune


Outcome Measures
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Primary Outcome Measures :
  1. Total liver volume [ Time Frame: 12 months ]
    Change in total liver volume


Secondary Outcome Measures :
  1. Total liver volume [ Time Frame: 24 months ]
    Change in total liver volume

  2. Total kidney volume [ Time Frame: 12 month ]
    Change in total kidney volume

  3. Estimated glomerular filtration rate [ Time Frame: 12 month ]
    Change in estimated glomerular filtration rate

  4. Urinary biomarker [ Time Frame: 12 month ]
    Urinary biomarker level

  5. Total kidney volume [ Time Frame: 24 month ]
    Change in total kidney volume

  6. Estimated glomerular filtration rate [ Time Frame: 24 month ]
    Change in estimated glomerular filtration rate

  7. Urinary biomarker [ Time Frame: 24 month ]
    Urinary biomarker level


Other Outcome Measures:
  1. Abdominal pain [ Time Frame: 12month ]
    Abdominal pain quantified by Visual Analog Scale

  2. Abdominal pain [ Time Frame: 24 month ]
    Abdominal pain quantified by Visual Analog Scale

  3. Infection [ Time Frame: 24 month ]
    Incidence of infection event during study time

  4. Hospitalization [ Time Frame: 24 month ]
    Incidence of hospitalization due to adverse events during study time

  5. Drop out [ Time Frame: 24 month ]
    Incidence of discontinuation of study drug due to serious adverse events during study time


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 18 - 65
  • 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
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01680250


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

Locations
Korea, Republic of
Seoul National University Hospital Recruiting
Seoul, Korea, Republic of, 110-744
Contact: Curie Ahn, MD, PhD    82-2-2072-2222    curie@snu.ac.kr   
Sponsors and Collaborators
Seoul National University Hospital
Wyeth is now a wholly owned subsidiary of Pfizer
Investigators
Principal Investigator: Curie Ahn, MD, PhD Seoul National University Hospital
More Information
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party: Seoul National University Hospital
ClinicalTrials.gov Identifier: NCT01680250     History of Changes
Other Study ID Numbers: SILVER
First Posted: September 7, 2012    Key Record Dates
Last Update Posted: September 7, 2012
Last Verified: September 2012

Keywords provided by Seoul National University Hospital:
Total liver volume
Liver cyst

Additional relevant MeSH terms:
Kidney Diseases
Polycystic Kidney Diseases
Urologic Diseases
Kidney Diseases, Cystic
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Sirolimus
Everolimus
 Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs