Ignite Creation Date:
2025-12-26 @ 11:46 AM
Ignite Modification Date:
2026-03-04 @ 10:50 PM
Study NCT ID:
NCT02538016
Status:
COMPLETED
Last Update Posted:
2020-07-10
First Post:
2015-07-21
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003555', 'term': 'Cystinuria'}, {'id': 'D007669', 'term': 'Kidney Calculi'}], 'ancestors': [{'id': 'D000608', 'term': 'Renal Aminoacidurias'}, {'id': 'D015499', 'term': 'Renal Tubular Transport, Inborn Errors'}, {'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': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D053040', 'term': 'Nephrolithiasis'}, {'id': 'D052878', 'term': 'Urolithiasis'}, {'id': 'D014545', 'term': 'Urinary Calculi'}, {'id': 'D002137', 'term': 'Calculi'}, {'id': 'D020763', 'term': 'Pathological Conditions, Anatomical'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077602', 'term': 'Tolvaptan'}], 'ancestors': [{'id': 'D001552', 'term': 'Benzazepines'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Alyssia.Venna@childrens.harvard.edu', 'phone': '617-919-6352', 'title': 'Alyssia Venna', 'organization': "Boston Children's Hospital"}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': '2 weeks', 'eventGroups': [{'id': 'EG000', 'title': 'Tolvaptan', 'description': 'Tolvaptan.', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 4, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Severe Thirst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Tolvaptan', 'description': 'Tolvaptan.'}], 'classes': [{'categories': [{'measurements': [{'value': '70', 'groupId': 'OG000', 'lowerLimit': '-3', 'upperLimit': '111'}]}]}], 'paramType': 'MEAN', 'timeFrame': '23 days', 'description': 'The primary outcome was urinary cystine supersaturation as measured by "cystine capacity". This proprietary test (Litholink Corp., Chicago IL) is reported as a value in mg/L above or below zero, with positive values indicating urine undersaturated with cystine, while negative values indicate that the urine is supersaturated with cystine. Four 24-hour urine samples were obtained during the study: one at baseline 3-6 days prior to the measurement, one on day 3-4 of the dosing period, one on day 7-8 of the dosing period, and one 3-6 days after the washout period. Each 24 hour urine sample was sent individually for analysis by Litholink Corp, which performs the cystine assays.', 'unitOfMeasure': 'mg/L', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Urine Osmolality at High Dose (Day 8)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Tolvaptan', 'description': 'Tolvaptan.'}], 'classes': [{'title': 'Sodium', 'categories': [{'measurements': [{'value': '141', 'groupId': 'OG000', 'lowerLimit': '139', 'upperLimit': '142'}]}]}, {'title': 'Potassium', 'categories': [{'measurements': [{'value': '4.05', 'groupId': 'OG000', 'lowerLimit': '3.7', 'upperLimit': '4.4'}]}]}, {'title': 'Chloride', 'categories': [{'measurements': [{'value': '102', 'groupId': 'OG000', 'lowerLimit': '101', 'upperLimit': '103'}]}]}], 'paramType': 'MEAN', 'timeFrame': '11 days', 'description': 'Secondary outcomes included serum sodium and other electrolyte levels. Serum electrolytes for each subject before, during, and after tolvaptan treatment (Sodium/Potassium/Chloride in mmol/L).', 'unitOfMeasure': 'mmol/L', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Tolvaptan', 'description': 'This was a single arm, unblinded study. All participants received half of the recommended dose (weight-dependent) on Days 1 through 4 and then the full recommended dose (weight-dependent) on Days 5 through 8\n\nExample of Dosing Schedule by Weight:\n\nParticipant Weight is between: 12.5 kg -37.49 kg Then their Dose on Days 1-4 = 7.5mg (.6mg/kg -.2mg/kg) Dose on Days 5-8 is doubled = 15mg (1.2mg/kg - .4mg/kg)\n\nMaximum dose administered was 60 mg.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Recruitment and Enrollment period was between August 2016 to December 2018. Subjects were recruited from our existing database of cystinuria patients, from new patients presenting during the research period, and via advertising through the International Cystinuria Foundation, Rare Kidney Stone Consortium, and social media.', 'preAssignmentDetails': 'Of 6 eligible and interested participants, 5 met inclusion criteria and were consented, and of these, 1 withdrew prior to travel to our hospital for drug administration.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Tolvaptan', 'description': 'This was a single arm, unblinded study. All participants received half of the recommended dose (weight-dependent) on Days 1 through 4 and then the full recommended dose (weight-dependent) on Days 5 through 8\n\nExample of Dosing Schedule by Weight:\n\nParticipant Weight is between: 12.5 kg -37.49 kg Then their Dose on Days 1-4 = 7.5mg (.6mg/kg -.2mg/kg) Dose on Days 5-8 is doubled = 15mg (1.2mg/kg - .4mg/kg) Maximum dose administered was 60 mg.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}]}], 'categories': [{'title': '<=18 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-09-23', 'size': 326036, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2020-05-19T11:51', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 4}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-10'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-05', 'completionDateStruct': {'date': '2018-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-06-25', 'studyFirstSubmitDate': '2015-07-21', 'resultsFirstSubmitDate': '2020-05-01', 'studyFirstSubmitQcDate': '2015-08-31', 'lastUpdatePostDateStruct': {'date': '2020-07-10', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-05-19', 'studyFirstPostDateStruct': {'date': '2015-09-02', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-06-04', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8)', 'timeFrame': '23 days', 'description': 'The primary outcome was urinary cystine supersaturation as measured by "cystine capacity". This proprietary test (Litholink Corp., Chicago IL) is reported as a value in mg/L above or below zero, with positive values indicating urine undersaturated with cystine, while negative values indicate that the urine is supersaturated with cystine. Four 24-hour urine samples were obtained during the study: one at baseline 3-6 days prior to the measurement, one on day 3-4 of the dosing period, one on day 7-8 of the dosing period, and one 3-6 days after the washout period. Each 24 hour urine sample was sent individually for analysis by Litholink Corp, which performs the cystine assays.'}], 'secondaryOutcomes': [{'measure': 'Urine Osmolality at High Dose (Day 8)', 'timeFrame': '11 days', 'description': 'Secondary outcomes included serum sodium and other electrolyte levels. Serum electrolytes for each subject before, during, and after tolvaptan treatment (Sodium/Potassium/Chloride in mmol/L).'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Cystinuria', 'Kidney Stones'], 'conditions': ['Cystinuria']}, 'descriptionModule': {'briefSummary': 'The goal of this research project is to establish that in short-term use, tolvaptan is a safe and potentially effective new therapy for cystinuria, by conducting a short-term pilot study of the safety and tolerability of this drug, and assess impact on urinary stone risk parameters, among adolescent and young adult patients with clinical cystinuria.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '29 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males and females age 12 - 29 years\n* Weight ≥ 25kg (55 lbs)\n* Confirmed cystinuria diagnosis\n* Specific blood test levels (done within the past 6 months)\n\nExclusion Criteria:\n\n* Concurrent non-renal disease that might increase risk of complications due to aquaresis\n* Liver or biliary disease (chronic or acute)\n* Malabsorption syndrome or other gastrointestinal condition that may interfere with response to therapy\n* Non-cutaneous malignancy within last 5 years\n* History of adverse reaction or allergy to Tolvaptan or other arginine vasopressin V2-receptor antagonists'}, 'identificationModule': {'nctId': 'NCT02538016', 'acronym': 'TCUPS', 'briefTitle': 'TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study', 'organization': {'class': 'OTHER', 'fullName': "Boston Children's Hospital"}, 'officialTitle': 'Use of Tolvaptan, a Vasopressin Antagonist, to Increase Urine Dilution and Reduce Cystine Urolithiasis Among Patients With Homozygous Cystinuria: a Pilot Investigation', 'orgStudyIdInfo': {'id': '16726'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Tolvaptan', 'interventionNames': ['Drug: Tolvaptan']}], 'interventions': [{'name': 'Tolvaptan', 'type': 'DRUG', 'armGroupLabels': ['Tolvaptan']}]}, 'contactsLocationsModule': {'locations': [{'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}], 'overallOfficials': [{'name': 'Caleb Nelson, MD, MPH', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Boston Children's Hospital"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Caleb Nelson', 'class': 'OTHER'}, 'collaborators': [{'name': 'Otsuka America Pharmaceutical', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'MD, MPH', 'investigatorFullName': 'Caleb Nelson', 'investigatorAffiliation': "Boston Children's Hospital"}}}}