Ignite Creation Date:
2025-12-24 @ 4:55 PM
Ignite Modification Date:
2025-12-29 @ 6:59 PM
Study NCT ID:
NCT01680250
Status:
UNKNOWN
Last Update Posted:
2012-09-07
First Post:
2012-08-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Sirolimus for Massive Polycystic Liver
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D007690', 'term': 'Polycystic Kidney Diseases'}], 'ancestors': [{'id': 'D052177', 'term': 'Kidney Diseases, Cystic'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D000072661', 'term': 'Ciliopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D020123', 'term': 'Sirolimus'}], 'ancestors': [{'id': 'D018942', 'term': 'Macrolides'}, {'id': 'D007783', 'term': 'Lactones'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 44}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2011-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2012-09', 'completionDateStruct': {'date': '2015-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2012-09-03', 'studyFirstSubmitDate': '2012-08-30', 'studyFirstSubmitQcDate': '2012-09-03', 'lastUpdatePostDateStruct': {'date': '2012-09-07', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2012-09-07', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Abdominal pain', 'timeFrame': '12month', 'description': 'Abdominal pain quantified by Visual Analog Scale'}, {'measure': 'Abdominal pain', 'timeFrame': '24 month', 'description': 'Abdominal pain quantified by Visual Analog Scale'}, {'measure': 'Infection', 'timeFrame': '24 month', 'description': 'Incidence of infection event during study time'}, {'measure': 'Hospitalization', 'timeFrame': '24 month', 'description': 'Incidence of hospitalization due to adverse events during study time'}, {'measure': 'Drop out', 'timeFrame': '24 month', 'description': 'Incidence of discontinuation of study drug due to serious adverse events during study time'}], 'primaryOutcomes': [{'measure': 'Total liver volume', 'timeFrame': '12 months', 'description': 'Change in total liver volume'}], 'secondaryOutcomes': [{'measure': 'Total liver volume', 'timeFrame': '24 months', 'description': 'Change in total liver volume'}, {'measure': 'Total kidney volume', 'timeFrame': '12 month', 'description': 'Change in total kidney volume'}, {'measure': 'Estimated glomerular filtration rate', 'timeFrame': '12 month', 'description': 'Change in estimated glomerular filtration rate'}, {'measure': 'Urinary biomarker', 'timeFrame': '12 month', 'description': 'Urinary biomarker level'}, {'measure': 'Total kidney volume', 'timeFrame': '24 month', 'description': 'Change in total kidney volume'}, {'measure': 'Estimated glomerular filtration rate', 'timeFrame': '24 month', 'description': 'Change in estimated glomerular filtration rate'}, {'measure': 'Urinary biomarker', 'timeFrame': '24 month', 'description': 'Urinary biomarker level'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Total liver volume', 'Liver cyst'], 'conditions': ['Polycystic Kidney Diseases']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the effectiveness and safety of Sirolimus in reducing liver volume in autosomal dominant polycystic kidney disease.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age 18 - 65\n* Patients diagnosed as ADPKD based upon the unified criteria for ultrasonographic diagnosis of ADPKD\n* Polycystic liver with total liver volume \\> 2500 mL or symptomatic polycystic liver\n* Estimated glomerular filtration rate (IDMS-traceable MDRD equation) \\>= 30 mL/min/1.73m2\n\nExclusion Criteria:\n\n* Concomitant systemic renal parenchymal or urinary tract disease (random urine albumin-to-creatinine ratio \\> 500 mg/g)\n* WBC \\< 4,000/uL, platelet \\< 100,000/uL, or hemoglobin \\< 10.0 g/dL\n* Diabetes mellitus, cancer, or psychiatric disorder\n* Increased liver enzymes (2-fold above normal value)\n* Hypercholesterolemia (fasting cholesterol \\> 200mg/dL) or hypertriglyceridemia (\\>150 mg/dL) not controlled by lipid lowering therapy\n* Infection with hepatitis B, C, HIV\n* Any condition that could prevent full comprehension of the purpose and risks of the study\n* Pregnant or lactating women or fertile women without effective contraception\n* History of intervention, such as cyst aspiration or embolization in past 1 year'}, 'identificationModule': {'nctId': 'NCT01680250', 'acronym': 'SILVER', 'briefTitle': 'Sirolimus for Massive Polycystic Liver', 'organization': {'class': 'OTHER', 'fullName': 'Seoul National University Hospital'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'SILVER'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Sirolimus', 'description': 'Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL', 'interventionNames': ['Drug: Sirolimus']}], 'interventions': [{'name': 'Sirolimus', 'type': 'DRUG', 'otherNames': ['Rapamune'], 'description': 'Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months', 'armGroupLabels': ['Sirolimus']}]}, 'contactsLocationsModule': {'locations': [{'zip': '110-744', 'city': 'Seoul', 'status': 'RECRUITING', 'country': 'South Korea', 'contacts': [{'name': 'Curie Ahn, MD, PhD', 'role': 'CONTACT', 'email': 'curie@snu.ac.kr', 'phone': '82-2-2072-2222'}], 'facility': 'Seoul National University Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}], 'centralContacts': [{'name': 'Curie Ahn, MD, PhD', 'role': 'CONTACT', 'email': 'curie@snu.ac.kr', 'phone': '82-2-2072-2222'}], 'overallOfficials': [{'name': 'Curie Ahn, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Seoul National University Hospital'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Seoul National University Hospital', 'class': 'OTHER'}, 'collaborators': [{'name': 'Wyeth is now a wholly owned subsidiary of Pfizer', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}