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Trial record 1 of 1 for:    NCT01680250
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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

Tracking Information
First Submitted Date  ICMJE August 30, 2012
First Posted Date  ICMJE September 7, 2012
Last Update Posted Date September 7, 2012
Study Start Date  ICMJE September 2011
Estimated Primary Completion Date September 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 3, 2012)
Total liver volume [ Time Frame: 12 months ]
Change in total liver volume
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: September 3, 2012)
  • Total liver volume [ Time Frame: 24 months ]
    Change in total liver volume
  • Total kidney volume [ Time Frame: 12 month ]
    Change in total kidney volume
  • Estimated glomerular filtration rate [ Time Frame: 12 month ]
    Change in estimated glomerular filtration rate
  • Urinary biomarker [ Time Frame: 12 month ]
    Urinary biomarker level
  • Total kidney volume [ Time Frame: 24 month ]
    Change in total kidney volume
  • Estimated glomerular filtration rate [ Time Frame: 24 month ]
    Change in estimated glomerular filtration rate
  • Urinary biomarker [ Time Frame: 24 month ]
    Urinary biomarker level
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: September 3, 2012)
  • Abdominal pain [ Time Frame: 12month ]
    Abdominal pain quantified by Visual Analog Scale
  • Abdominal pain [ Time Frame: 24 month ]
    Abdominal pain quantified by Visual Analog Scale
  • Infection [ Time Frame: 24 month ]
    Incidence of infection event during study time
  • Hospitalization [ Time Frame: 24 month ]
    Incidence of hospitalization due to adverse events during study time
  • Drop out [ Time Frame: 24 month ]
    Incidence of discontinuation of study drug due to serious adverse events during study time
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Sirolimus for Massive Polycystic Liver
Official Title  ICMJE 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
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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Polycystic Kidney Diseases
Intervention  ICMJE Drug: Sirolimus
Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months
Other Name: Rapamune
Study Arms  ICMJE Experimental: Sirolimus
Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL
Intervention: Drug: Sirolimus
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: September 3, 2012)
44
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2015
Estimated Primary Completion Date September 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Korea, Republic of
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01680250
Other Study ID Numbers  ICMJE SILVER
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Seoul National University Hospital
Study Sponsor  ICMJE Seoul National University Hospital
Collaborators  ICMJE Wyeth is now a wholly owned subsidiary of Pfizer
Investigators  ICMJE
Principal Investigator: Curie Ahn, MD, PhD Seoul National University Hospital
PRS Account Seoul National University Hospital
Verification Date September 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP