Sirolimus for Massive Polycystic Liver (SILVER)
This study is currently recruiting participants.
Verified September 2012 by Seoul National University Hospital
Sponsor:
Seoul National University Hospital
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
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Polycystic Kidney Diseases |
Drug: Sirolimus |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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 |
Resource links provided by NLM:
Further study details as provided by Seoul National University Hospital:
Primary Outcome Measures:
- Total liver volume [ Time Frame: 12 months ] [ Designated as safety issue: No ]Change in total liver volume
Secondary Outcome Measures:
- 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
Other Outcome Measures:
- 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
| Estimated Enrollment: | 44 |
| Study Start Date: | September 2011 |
| Estimated Study Completion Date: | August 2015 |
| Estimated Primary Completion Date: | September 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
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
|
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.
Eligibility| Ages Eligible for Study: | 18 Years to 65 Years |
| Genders Eligible for Study: | Both |
| 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
Please refer to this study by its ClinicalTrials.gov identifier: 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
No publications provided
| Responsible Party: | Seoul National University Hospital |
| ClinicalTrials.gov Identifier: | NCT01680250 History of Changes |
| Other Study ID Numbers: | SILVER |
| Study First Received: | August 30, 2012 |
| Last Updated: | September 3, 2012 |
| Health Authority: | Korea: Food and Drug Administration Korea: Institutional Review Board |
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 Sirolimus Everolimus Antibiotics, Antineoplastic Antineoplastic Agents |
Therapeutic Uses Pharmacologic Actions Antifungal Agents Anti-Infective Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Anti-Bacterial Agents |
ClinicalTrials.gov processed this record on May 16, 2013