Ignite Creation Date:
2025-12-24 @ 11:16 PM
Ignite Modification Date:
2025-12-25 @ 8:54 PM
Study NCT ID:
NCT00286156
Status:
COMPLETED
Last Update Posted:
2015-04-06
First Post:
2006-02-01
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D007690', 'term': 'Polycystic Kidney Diseases'}, {'id': 'D016891', 'term': 'Polycystic Kidney, Autosomal Dominant'}], '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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'braunw@ccf.org', 'phone': '216/444/6995', 'title': 'Dr William Braun Consultant Staff Nephrology, Cleveland Clinic', 'organization': 'Cleveland Clinic'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Standard Rapamycin Dose (STD)', 'description': 'Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml\n\nRapamycin: Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml', 'otherNumAtRisk': 10, 'otherNumAffected': 10, 'seriousNumAtRisk': 10, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'Low Dose Rapamycin (LD)', 'description': 'Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml\n\nRapamycin: Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml', 'otherNumAtRisk': 10, 'otherNumAffected': 6, 'seriousNumAtRisk': 10, 'seriousNumAffected': 1}, {'id': 'EG002', 'title': 'Standard Care', 'description': 'Standard Care: fluid intake primarily water of 2500-3000ml/24hrs, low sodium diet of 2300mg/24 hrs, caffeine avoidance, control of hypertension', 'otherNumAtRisk': 10, 'otherNumAffected': 8, 'seriousNumAtRisk': 10, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Oral ulcerations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 10, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Immune system disorders'}, {'term': 'Nonserious infections', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 10, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Infections and infestations'}, {'term': 'Edema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders'}, {'term': 'Gastrointestinal symptoms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 10, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders'}, {'term': 'Dermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 10, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders'}, {'term': 'Miscellaneous/other', 'notes': 'The problems in this group were minor, extremely varied and transient.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 7, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 10, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'General disorders'}], 'seriousEvents': [{'term': 'pulmonary embolus', 'notes': 'Patient had massive polycystic kidneys (about 7500ml) and there was compression of the right iliac vein/IVC with clot formation. This has been reported in the literature, so it is not necessarily related to drug.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 10, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders'}, {'term': 'nephrotic range proteinuria', 'notes': 'Open renal biopsy showed FSGS. Primary glomerulopathies have been reported to be superimposed on underlying ADPKD.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 10, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders'}, {'term': 'Decrease visual acuity', 'notes': 'Patient developed transient decrease in visual acuity. Neuro-ophthalmologist did not find any association with drug. Vision improved without specific treatment.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 10, 'numAffected': 0}], 'organSystem': 'Eye disorders'}, {'term': 'Hypoglycemia', 'notes': 'Hypoglycemia associated with excessive beer consumption.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 10, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in GFR From Baseline to 12 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '9', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Standard Rapamycin Dose (STD)', 'description': 'Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml\n\nRapamycin: Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml'}, {'id': 'OG001', 'title': 'Low Dose Rapamycin (LD)', 'description': 'Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml\n\nRapamycin: Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml'}, {'id': 'OG002', 'title': 'Standard Care', 'description': 'Standard Care: fluid intake primarily water of 2500-3000ml/24hrs, low sodium diet of 2300mg/24 hrs, caffeine avoidance, control of hypertension'}], 'classes': [{'title': 'Baseline iGFR', 'categories': [{'measurements': [{'value': '72.8', 'spread': '25.7', 'groupId': 'OG000'}, {'value': '70.3', 'spread': '27.0', 'groupId': 'OG001'}, {'value': '73.1', 'spread': '20.3', 'groupId': 'OG002'}]}]}, {'title': '12 month iGFR', 'categories': [{'measurements': [{'value': '74.4', 'spread': '34.4', 'groupId': 'OG000'}, {'value': '78.0', 'spread': '35.0', 'groupId': 'OG001'}, {'value': '61.9', 'spread': '15.6', 'groupId': 'OG002'}]}]}, {'title': 'Change in iGFR', 'categories': [{'measurements': [{'value': '1.6', 'spread': '12.1', 'groupId': 'OG000'}, {'value': '7.7', 'spread': '12.5', 'groupId': 'OG001'}, {'value': '-11.2', 'spread': '9.1', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '<0.01', 'groupIds': ['OG000', 'OG001', 'OG002'], 'ciNumSides': 'TWO_SIDED', 'pValueComment': 'Pairwise comparisons adjusted for multiple testing (Tukey)', 'groupDescription': 'Null hypothesis: no difference between groups in iGFR', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': 'From baseline to 12 months', 'description': 'GFR (glomerular filtration rate) was measured by iothalamate. GFR is a key indicator of renal function.', 'unitOfMeasure': 'ml/min/1.73m^2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Total Kidney Volume as Measured by 3D-CT From Baseline to 12 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '9', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Standard Rapamycin Dose (STD)', 'description': 'Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml\n\nRapamycin: Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml'}, {'id': 'OG001', 'title': 'Low Dose Rapamycin (LD)', 'description': 'Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml\n\nRapamycin: Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml'}, {'id': 'OG002', 'title': 'Standard Care', 'description': 'Standard Care: fluid intake primarily water of 2500-3000ml/24hrs, low sodium diet of 2300mg/24 hrs, caffeine avoidance, control of hypertension'}], 'classes': [{'title': 'Baseline TKV', 'categories': [{'measurements': [{'value': '1454.1', 'spread': '801.5', 'groupId': 'OG000'}, {'value': '1919.1', 'spread': '903.6', 'groupId': 'OG001'}, {'value': '1907.1', 'spread': '1126.8', 'groupId': 'OG002'}]}]}, {'title': '12 month TKV', 'categories': [{'measurements': [{'value': '1537', 'spread': '864.3', 'groupId': 'OG000'}, {'value': '2115.8', 'spread': '1035.0', 'groupId': 'OG001'}, {'value': '2059.8', 'spread': '1236.0', 'groupId': 'OG002'}]}]}, {'title': 'Change in TKV', 'categories': [{'measurements': [{'value': '82.9', 'spread': '111.3', 'groupId': 'OG000'}, {'value': '197.7', 'spread': '201.2', 'groupId': 'OG001'}, {'value': '152.7', 'spread': '129.4', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From baseline to 12 months', 'description': 'Total kidney volume measured by CT from baseline to 12 months', 'unitOfMeasure': 'ml', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Standard Rapamycin Dose (STD)', 'description': 'Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml\n\nRapamycin: Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml'}, {'id': 'FG001', 'title': 'Low Dose Rapamycin (LD)', 'description': 'Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml\n\nRapamycin: Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml'}, {'id': 'FG002', 'title': 'Standard Care', 'description': 'Standard Care: fluid intake primarily water of 2500-3000ml/24hrs, low sodium diet of 2300mg/24 hrs, caffeine avoidance, control of hypertension'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}, {'groupId': 'FG001', 'numSubjects': '10'}, {'groupId': 'FG002', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '9'}, {'groupId': 'FG002', 'numSubjects': '9'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Missed 12 month visit', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'AE: pulmonary embolus', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'AE: nephrotic-range proteinuruia', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'AE: pneumonia', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}, {'value': '30', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'Standard Rapamycin Dose (STD)', 'description': 'Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml\n\nRapamycin: Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml'}, {'id': 'BG001', 'title': 'Low Dose Rapamycin (LD)', 'description': 'Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml\n\nRapamycin: Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml'}, {'id': 'BG002', 'title': 'Standard Care', 'description': 'Standard Care: fluid intake primarily water of 2500-3000ml/24hrs, low sodium diet of 2300mg/24 hrs, caffeine avoidance, control of hypertension'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '53.2', 'spread': '15.0', 'groupId': 'BG000'}, {'value': '44.9', 'spread': '8.6', 'groupId': 'BG001'}, {'value': '49.4', 'spread': '11.0', 'groupId': 'BG002'}, {'value': '49.3', 'spread': '12.0', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '13', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '17', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}, {'title': 'White', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}, {'value': '29', 'groupId': 'BG003'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}, {'value': '30', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Baseline characteristics and risk factors', 'classes': [{'title': 'Hypertension', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}, {'value': '11', 'groupId': 'BG003'}]}]}, {'title': 'Family history of ESRD', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '19', 'groupId': 'BG003'}]}]}, {'title': 'Initial iGFR 25-59 ml/min per 1.73 m^2', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}]}, {'title': 'Initial TKV>1500ml', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}, {'value': '19', 'groupId': 'BG003'}]}]}, {'title': 'Initial height-adjusted TKV>=600ml/m', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}, {'value': '20', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'description': 'ADPKD, autosomal dominant polycystic kidney disease; iGFR, 125I-iothalamate GFR; TKV, total kidney volume; Hypertension is a BP \\>= 140/90 mmHg before 35 years old; family history of ESRD occurs from ADPKD before 56 years old', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'FACTORIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 30}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-10'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-03', 'completionDateStruct': {'date': '2014-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-03-31', 'studyFirstSubmitDate': '2006-02-01', 'resultsFirstSubmitDate': '2015-03-31', 'studyFirstSubmitQcDate': '2006-02-01', 'lastUpdatePostDateStruct': {'date': '2015-04-06', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2015-03-31', 'studyFirstPostDateStruct': {'date': '2006-02-03', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-04-06', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in GFR From Baseline to 12 Months', 'timeFrame': 'From baseline to 12 months', 'description': 'GFR (glomerular filtration rate) was measured by iothalamate. GFR is a key indicator of renal function.'}], 'secondaryOutcomes': [{'measure': 'Change in Total Kidney Volume as Measured by 3D-CT From Baseline to 12 Months', 'timeFrame': 'From baseline to 12 months', 'description': 'Total kidney volume measured by CT from baseline to 12 months'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['ADPKD'], 'conditions': ['Polycystic Kidney Diseases']}, 'referencesModule': {'references': [{'pmid': '39356039', 'type': 'DERIVED', 'citation': 'St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.'}]}, 'descriptionModule': {'briefSummary': 'This study is a prospective, randomized, open-label, pilot clinical trial designed to compare the effects of an agent that has antiproliferative (1,2), antiangiogenesis (3),and tumor-progression blocking capabilities (4), namely, rapamycin (Rapamune®), in the treatment of autosomal-dominant polycystic kidney disease (ADPKD).\n\nUp to this time, only generic renal disease treatments for ADPKD have been in use, such as the treatment of hypertension, urinary tract infections, renal stones, renal call carcinomas, and replacement therapy with dialysis and/or renal transplantation. The fundamental aberrations in ADPKD are proliferation of cyst-forming tubuloepithelial cells, secretion of cytokine-rich fluid into those cysts, and progressive cyst expansion and release of inflammatory mediators that injure surrounding normal renal tissue. Consequently, therapy directed specifically at blocking the proliferation of tubuloepithelial cells and their tendency to malignant transformation, as well as impeding their blood supply, should have obvious merit.\n\nGeneral Procedures:\n\nIn Group I participants will have an iothalamate glomerular filtration rate (GFR) equal to or greater than 60 ml/min/1.73 m2, and in Group II participants will have a GFR less than 25-59 ml/min/1.73 m2. Both males and females with ADPKD who volunteer and qualify, will be randomly and prospectively assigned to treatment with rapamycin at either a high or low trough blood level or to standard care (each 1/3 of enrolled patients) for one year. The two treatment groups will receive rapamycin doses aimed at maintaining the 20- to 24-hour trough blood levels at either 2 to 5 ng/mL (low-dose), or greater than 5 to 8 ng/mL (high-dose). These trough levels are in the lower range of levels used when treating renal transplant recipients in whom trough levels are typically maintained between 5 and 15 ng/mL.', 'detailedDescription': 'This study is a prospective, randomized,open label, pilot clinical trial designed to compare the effects of an agent that has antiproliferative (1,2), antiangiogenesis (3),and tumor-progression blocking capabilities (4), namely, rapamycin (Rapamune®), in the treatment of autosomal-dominant polycystic kidney disease (ADPKD).\n\nUp to this time, only generic renal disease treatments for ADPKD have been in use, such as the treatment of hypertension, urinary tract infections, renal stones, renal call carcinomas, and replacement therapy with dialysis and/or renal transplantation. The fundamental aberrations in ADPKD are proliferation of cyst-forming tubuloepithelial cells, secretion of cytokine-rich fluid into those cysts, and progressive cyst expansion and release of inflammatory mediators that injure surrounding normal renal tissue. Consequently, therapy directed specifically at blocking the proliferation of tubuloepithelial cells and their tendency to malignant transformation, as well as impeding their blood supply, should have obvious merit.\n\nGeneral Procedures:\n\nIn Group I participants will have an iothalamate glomerular filtration rate (GFR) equal to or greater than 60 ml/min/1.73 m2, and in Group II participants will have a GFR less than 25-59 ml/min/1.73 m2. Both males and females with ADPKD who volunteer and qualify, will be randomly and prospectively assigned to treatment with rapamycin at either a high or low trough blood level or to standard care (each 1/3 of enrolled patients) for one year. The two treatment groups will receive rapamycin doses aimed at maintaining the 20- to 24-hour trough blood levels at either 2 to 5 ng/mL (low-dose), or greater than 5 to 8 ng/mL (high-dose). These trough levels are in the lower range of levels used when treating renal transplant recipients in whom trough levels are typically maintained between 5 and 15 ng/mL.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* ADPKD\n* \\> 18 y.o. GFR greater than or equal to 25. Willingness to be randomized to any treatment group Willingness to follow protocol requirements-frequent testing and follow-up required at Cleveland Clinic(Cleveland, OH) signed informed consent Willingness to use birth control(male and female)\n\nExclusion Criteria:\n\n* Pregnancy\n* post partum\n* lactating\n* system illness with renal involvement'}, 'identificationModule': {'nctId': 'NCT00286156', 'briefTitle': 'Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)', 'organization': {'class': 'OTHER', 'fullName': 'The Cleveland Clinic'}, 'officialTitle': 'Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease', 'orgStudyIdInfo': {'id': '7736'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '1', 'description': 'Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml', 'interventionNames': ['Drug: Rapamune']}, {'type': 'EXPERIMENTAL', 'label': '2', 'description': 'Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml', 'interventionNames': ['Drug: Rapamune']}, {'type': 'NO_INTERVENTION', 'label': '3', 'description': 'Standard Care'}], 'interventions': [{'name': 'Rapamune', 'type': 'DRUG', 'otherNames': ['Rapamycin'], 'description': 'Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml Group 3- Standard Care', 'armGroupLabels': ['1', '2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'The Cleveland Clinic- main campus', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}], 'overallOfficials': [{'name': 'William E. Braun, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'The Cleveland Clinic'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'The Cleveland Clinic', 'class': 'OTHER'}, 'collaborators': [{'name': 'Wyeth is now a wholly owned subsidiary of Pfizer', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}