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Sirolimus for Massive Polycystic Liver (SILVER)

This study is currently recruiting participants.
Verified September 2012 by Seoul National University Hospital
Sponsor:
Collaborator:
Wyeth is now a wholly owned subsidiary of Pfizer
Information provided by (Responsible Party):
Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT01680250
First received: August 30, 2012
Last updated: September 3, 2012
Last verified: September 2012
History of Changes

August 30, 2012
September 3, 2012
September 2011
September 2014   (final data collection date for primary outcome measure)
Total liver volume [ Time Frame: 12 months ] [ Designated as safety issue: No ]
Change in total liver volume
Same as current
Complete list of historical versions of study NCT01680250 on ClinicalTrials.gov Archive Site
  • Total liver volume [ Time Frame: 24 months ] [ Designated as safety issue: No ]
    Change in total liver volume
  • Total kidney volume [ Time Frame: 12 month ] [ Designated as safety issue: No ]
    Change in total kidney volume
  • Estimated glomerular filtration rate [ Time Frame: 12 month ] [ Designated as safety issue: No ]
    Change in estimated glomerular filtration rate
  • Urinary biomarker [ Time Frame: 12 month ] [ Designated as safety issue: No ]
    Urinary biomarker level
  • Total kidney volume [ Time Frame: 24 month ] [ Designated as safety issue: No ]
    Change in total kidney volume
  • Estimated glomerular filtration rate [ Time Frame: 24 month ] [ Designated as safety issue: No ]
    Change in estimated glomerular filtration rate
  • Urinary biomarker [ Time Frame: 24 month ] [ Designated as safety issue: No ]
    Urinary biomarker level
Same as current
  • Abdominal pain [ Time Frame: 12month ] [ Designated as safety issue: Yes ]
    Abdominal pain quantified by Visual Analog Scale
  • Abdominal pain [ Time Frame: 24 month ] [ Designated as safety issue: Yes ]
    Abdominal pain quantified by Visual Analog Scale
  • Infection [ Time Frame: 24 month ] [ Designated as safety issue: Yes ]
    Incidence of infection event during study time
  • Hospitalization [ Time Frame: 24 month ] [ Designated as safety issue: Yes ]
    Incidence of hospitalization due to adverse events during study time
  • Drop out [ Time Frame: 24 month ] [ Designated as safety issue: Yes ]
    Incidence of discontinuation of study drug due to serious adverse events during study time
Same as current
 
Sirolimus for Massive Polycystic Liver
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

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

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.
Interventional
Phase 2
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Polycystic Kidney Diseases
Drug: Sirolimus
Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months
Other Name: Rapamune
Experimental: Sirolimus
Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL
Intervention: Drug: Sirolimus
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
44
August 2015
September 2014   (final data collection date for primary outcome measure)

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
Both
18 Years to 65 Years
No
Contact: Curie Ahn, MD, PhD 82-2-2072-2222 curie@snu.ac.kr
Korea, Republic of
 
NCT01680250
SILVER
No
Seoul National University Hospital
Seoul National University Hospital
Wyeth is now a wholly owned subsidiary of Pfizer
Principal Investigator: Curie Ahn, MD, PhD Seoul National University Hospital
Seoul National University Hospital
September 2012

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