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Sirolimus for Massive Polycystic Liver (SILVER)

Primary Purpose

Polycystic Kidney Diseases

Status
Unknown status
Phase
Phase 2
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Sirolimus
Sponsored by
Seoul National University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Polycystic Kidney Diseases focused on measuring Total liver volume, Liver cyst

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Seoul National University HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Sirolimus

Arm Description

Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL

Outcomes

Primary Outcome Measures

Total liver volume
Change in total liver volume

Secondary Outcome Measures

Total liver volume
Change in total liver volume
Total kidney volume
Change in total kidney volume
Estimated glomerular filtration rate
Change in estimated glomerular filtration rate
Urinary biomarker
Urinary biomarker level
Total kidney volume
Change in total kidney volume
Estimated glomerular filtration rate
Change in estimated glomerular filtration rate
Urinary biomarker
Urinary biomarker level

Full Information

First Posted
August 30, 2012
Last Updated
September 3, 2012
Sponsor
Seoul National University Hospital
Collaborators
Wyeth is now a wholly owned subsidiary of Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT01680250
Brief Title
Sirolimus for Massive Polycystic Liver
Acronym
SILVER
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 Type
Interventional

2. Study Status

Record Verification Date
September 2012
Overall Recruitment Status
Unknown status
Study Start Date
September 2011 (undefined)
Primary Completion Date
September 2014 (Anticipated)
Study Completion Date
August 2015 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Seoul National University Hospital
Collaborators
Wyeth is now a wholly owned subsidiary of Pfizer

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polycystic Kidney Diseases
Keywords
Total liver volume, Liver cyst

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
44 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Sirolimus
Arm Type
Experimental
Arm Description
Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL
Intervention Type
Drug
Intervention Name(s)
Sirolimus
Other Intervention Name(s)
Rapamune
Intervention Description
Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months
Primary Outcome Measure Information:
Title
Total liver volume
Description
Change in total liver volume
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Total liver volume
Description
Change in total liver volume
Time Frame
24 months
Title
Total kidney volume
Description
Change in total kidney volume
Time Frame
12 month
Title
Estimated glomerular filtration rate
Description
Change in estimated glomerular filtration rate
Time Frame
12 month
Title
Urinary biomarker
Description
Urinary biomarker level
Time Frame
12 month
Title
Total kidney volume
Description
Change in total kidney volume
Time Frame
24 month
Title
Estimated glomerular filtration rate
Description
Change in estimated glomerular filtration rate
Time Frame
24 month
Title
Urinary biomarker
Description
Urinary biomarker level
Time Frame
24 month
Other Pre-specified Outcome Measures:
Title
Abdominal pain
Description
Abdominal pain quantified by Visual Analog Scale
Time Frame
12month
Title
Abdominal pain
Description
Abdominal pain quantified by Visual Analog Scale
Time Frame
24 month
Title
Infection
Description
Incidence of infection event during study time
Time Frame
24 month
Title
Hospitalization
Description
Incidence of hospitalization due to adverse events during study time
Time Frame
24 month
Title
Drop out
Description
Incidence of discontinuation of study drug due to serious adverse events during study time
Time Frame
24 month

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Curie Ahn, MD, PhD
Phone
82-2-2072-2222
Email
curie@snu.ac.kr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Curie Ahn, MD, PhD
Organizational Affiliation
Seoul National University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
110-744
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Curie Ahn, MD, PhD
Phone
82-2-2072-2222
Email
curie@snu.ac.kr

12. IPD Sharing Statement

Learn more about this trial

Sirolimus for Massive Polycystic Liver

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