Ignite Creation Date:
2025-12-25 @ 2:32 AM
Ignite Modification Date:
2025-12-31 @ 1:07 PM
Study NCT ID:
NCT04818034
Status:
COMPLETED
Last Update Posted:
2025-10-16
First Post:
2021-03-17
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
The Effect of Sodium-glucose Cotransporter (SGLT) 2 Inhibitors on Cystine Stone Formation: A Preliminary Study
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003555', 'term': 'Cystinuria'}], '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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C529054', 'term': 'dapagliflozin'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'victoria.hogue@ucsf.edu', 'phone': '415-514-0918', 'title': 'Marshall Stoller', 'organization': 'University of California, San Francisco'}, 'certainAgreement': {'piSponsorEmployee': True}, 'limitationsAndCaveats': {'description': 'Our study has several important limitations. First, this was a retrospective study with a limited follow up duration on SGLT2 inhibitors. Second, treating the stone event rate as its own control may introduce healthcare utilization bias as interfacing with the medical system could have led to improved dietary habits which had a major impact on stone event rates. Third, patients with cystinuria are known to develop symptomatic calculi in clusters for unknown reasons. The sample size is small.'}}, 'adverseEventsModule': {'timeFrame': 'From enrollment until the end of follow-up, 4 weeks.', 'eventGroups': [{'id': 'EG000', 'title': 'Study Drug', 'description': 'The study drug Dapagliflozin', 'otherNumAtRisk': 10, 'deathsNumAtRisk': 10, 'otherNumAffected': 0, 'seriousNumAtRisk': 10, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': '24hr Urine Measurements of the Effect of SGLT-2 Inhibitor Therapy on Cystine Production in Urine in Patients With Cystinuria', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Study Drug', 'description': 'The study drug Dapagliflozin'}], 'classes': [{'categories': [{'measurements': [{'value': '999.3', 'spread': '1.1', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.965', 'groupIds': ['OG000'], 'paramType': 'paired t-test', 'ciNumSides': 'ONE_SIDED', 'ciPctValue': '96', 'paramValue': '0.965', 'dispersionType': 'STANDARD_DEVIATION', 'dispersionValue': '37', 'statisticalMethod': 'paired t-test', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'paired t-test'}], 'paramType': 'MEAN', 'timeFrame': '1 month', 'description': 'The primary efficacy endpoint will be assessed by comparing previous 24hr urine cystine concentration data collected prior to study participation and 24hr urine concentration data collected after being on the study drug, specifically examining the content of cystine in the urine but not exclusively.', 'unitOfMeasure': '24hr urine cystine concentration (mg)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The analysis was specifically looking at cystine production in urine but other products were analyzed as well as shown in the table.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Study Drug', 'description': 'The study drug Dapagliflozin'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': "The study team will identify the patients being treated for cystinuria within the study team's clinic. The study team will inform these patients of the study and provide them with the study details to allow further discussion and to answer any questions the patients might have.", 'preAssignmentDetails': 'There is only one arm for this study. Any patient willing to participate in this study will be in that arm and will be given the study drug Dapagliflozin.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Study Drug', 'description': 'The study drug Dapagliflozin'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '10', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-08-17', 'size': 493658, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2025-09-19T13:59', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 10}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-08-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2022-04-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-10-08', 'studyFirstSubmitDate': '2021-03-17', 'resultsFirstSubmitDate': '2025-09-19', 'studyFirstSubmitQcDate': '2021-03-23', 'lastUpdatePostDateStruct': {'date': '2025-10-16', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2025-09-19', 'studyFirstPostDateStruct': {'date': '2021-03-26', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-10-08', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2021-12-13', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': '24hr Urine Measurements of the Effect of SGLT-2 Inhibitor Therapy on Cystine Production in Urine in Patients With Cystinuria', 'timeFrame': '1 month', 'description': 'The primary efficacy endpoint will be assessed by comparing previous 24hr urine cystine concentration data collected prior to study participation and 24hr urine concentration data collected after being on the study drug, specifically examining the content of cystine in the urine but not exclusively.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystinuria']}, 'descriptionModule': {'briefSummary': 'Cystinuria is an inherited autosomal recessive disorder of the kidney that is the result of an inability to reabsorb cystine from the urine. Supersaturation of cystine in the urine produces crystals that precipitate and form stones in the kidney, which can be a cause of obstruction, infection, and chronic kidney disease. Cystine stones constitute a major health challenge for affected individuals with cystinuria because of the frequent recurrence of painful symptoms and the current absence of effective, patient-accepting treatment.\n\nA mainstay of therapy is breaking or preventing the cystine bond on the molecular level such that cystine (which is formed from the joining of two cysteine amino acids and their corresponding sulfur atoms) cannot precipitate in the urine. It is hypothesized that a glucose molecule may be able to do this if introduced into the urine. SGLT-2 inhibitors are a class of drug that are FDA approved to treat diabetes mellitus (DM) and heart failure by inhibiting an enzyme in the kidney that allows for reabsorption of glucose from the urine. This effectively increases the concentration of glucose in the urine. Our hypothesis suggests that administration of this drug to patients with cystine will introduce sufficient glucose into the urine to prevent the formation of cystine stones. To date, there has been no published data on the effectiveness of this therapy for this indication, although the dosage and administration would be identical to that already approved by the FDA for the treatment of DM and heart failure.', 'detailedDescription': 'This is a single center, proof of concept prospective cohort trial designed to assess the effect of daily oral administration of dapagliflozin on cystine formation in freshly voided urine. Five subjects are planned, each with previously diagnosed cystinuria and without current treatment except with potassium citrate medication.\n\nTotal duration of subject participation with be up to four weeks. Total duration of the study is expected to be 6 months.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* males and females age 18 or older\n* documented cystinuria on prior 24-hour urine collection and/or stone analysis\n* history of previous cystine kidney stones\n* able and willing to provide consent\n\nExclusion Criteria:\n\n* prior diagnosis of diabetes mellitus (type I or type II)\n* current SGLT-2 inhibitor administration at the time of screening\n* SGLT-2 inhibitor administration within the last year prior to screening\n* vulnerable populations including incarceration status\n* anticipation of pregnancy during the study duration\n* unable to give informed consent\n* non-English primary language\n* pregnancy, lactation, or child- bearing age without birth control devices\n* serious illness likely to cause death within the next 5 years.'}, 'identificationModule': {'nctId': 'NCT04818034', 'briefTitle': 'The Effect of Sodium-glucose Cotransporter (SGLT) 2 Inhibitors on Cystine Stone Formation: A Preliminary Study', 'organization': {'class': 'OTHER', 'fullName': 'University of California, San Francisco'}, 'officialTitle': 'The Effect of Sodium-glucose Cotransporter (SGLT) 2 Inhibitors on Cystine Stone Formation: A Preliminary Study', 'orgStudyIdInfo': {'id': 'SGLT2'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '10 mg Dapagliflozin taken orally once daily for 4 weeks', 'description': '10 mg of the study drug Dapagliflozin taken orally once daily for 4 weeks', 'interventionNames': ['Drug: Dapagliflozin']}], 'interventions': [{'name': 'Dapagliflozin', 'type': 'DRUG', 'otherNames': ['FARXIGA'], 'description': 'Dapagliflozin is to lower blood sugar levels in adults with type 2 diabetes.', 'armGroupLabels': ['10 mg Dapagliflozin taken orally once daily for 4 weeks']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94143', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}], 'overallOfficials': [{'name': 'Marshall Stoller', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of California, San Francisco'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of California, San Francisco', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}