Ignite Creation Date:
2025-12-24 @ 9:37 PM
Ignite Modification Date:
2025-12-30 @ 9:54 PM
Study NCT ID:
NCT03301532
Status:
COMPLETED
Last Update Posted:
2024-01-17
First Post:
2017-08-29
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
Sponsor:
Organization:
Raw JSON
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Treatment: Triheptanoin (C7), orally, dose of 45% of the total daily calories divided in four equally spaced administrations (i.e., one dose every 4 hours to a total of four doses in one day).'}], 'classes': [{'categories': [{'measurements': [{'value': '3.82', 'spread': '1.9', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 1, Day 4', 'description': 'Change in Ketosis as measured by changes in blood beta-hydroxybutyric acid levels.', 'unitOfMeasure': 'mM', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Subjects with Glut1 deficency'}, {'type': 'SECONDARY', 'title': 'Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Fraction of Subjects With a Change in Seizure Rate', 'description': 'Ratio of subjects with seizure rate changes divided by total number of subjects. The seizure rate is based on changes in observable seizure numbers.'}], 'classes': [{'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline (Day 1) - Day 4', 'description': 'The percent change in observable seizure rate is measured as the change in observable seizure numbers from baseline to Day 4.\n\nSeizure rate is defined as count of seizures per patient per day.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects with Glut1 deficiency studied. Triheptanoin (C7 oil, liquid). Subjects consumed triheptanoin 4 times over the course of one day. The approach included substituting a fraction of ketogenic diet fat with C7, weight by weight, at the maximum tolerable dose (45% of their their daily calories) in individuals receiving a ketogenic diet prior to enrolment prescribed independently of this study.'}, {'type': 'SECONDARY', 'title': 'Change in Glycemia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Glycemia', 'description': 'Changes in blood glucose levels.'}], 'classes': [{'categories': [{'measurements': [{'value': '81.2', 'spread': '13.6', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 1 - Day 4', 'description': 'Change in Glycemia is measured as changes in blood glucose levels.', 'unitOfMeasure': 'mg/dl', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Experimental: Triheptanoin (C7)', 'description': 'Triheptanoin (C7 oil, liquid). Subjects consumed triheptanoin 4 times over the course of one day. The approach included substituting a fraction of ketogenic diet fat with C7, weight by weight, at the maximum tolerable dose (45% of their their daily calories) in individuals receiving a ketogenic diet prior to enrolment prescribed independently of this study.'}], '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'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Triheptanoin (C7 Oil, Liquid)', 'description': 'C7 oil was given at maximum tolerated dose (45% of total daily calories) to 10 Glut1 subjects receiving a ketogenic diet prior to enrolment as medically prescribed independently of this study.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '1', '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': '6', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', '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': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}]}]}, {'title': 'Argentina', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-01-22', 'size': 567774, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2023-11-03T13:01', 'hasProtocol': True}, {'date': '2021-01-22', 'size': 296117, 'label': 'Informed Consent Form', 'hasIcf': True, 'hasSap': False, 'filename': 'ICF_001.pdf', 'typeAbbrev': 'ICF', 'uploadDate': '2023-11-03T13:04', 'hasProtocol': False}]}}, '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': '2018-06-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-12', 'completionDateStruct': {'date': '2022-07-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-12-22', 'studyFirstSubmitDate': '2017-08-29', 'resultsFirstSubmitDate': '2023-11-09', 'studyFirstSubmitQcDate': '2017-09-28', 'lastUpdatePostDateStruct': {'date': '2024-01-17', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-12-22', 'studyFirstPostDateStruct': {'date': '2017-10-04', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-01-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-06-18', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Ketosis (Beta-hydroxybutyrate Levels)', 'timeFrame': 'Day 1, Day 4', 'description': 'Change in Ketosis as measured by changes in blood beta-hydroxybutyric acid levels.'}], 'secondaryOutcomes': [{'measure': 'Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate', 'timeFrame': 'Baseline (Day 1) - Day 4', 'description': 'The percent change in observable seizure rate is measured as the change in observable seizure numbers from baseline to Day 4.\n\nSeizure rate is defined as count of seizures per patient per day.'}, {'measure': 'Change in Glycemia', 'timeFrame': 'Day 1 - Day 4', 'description': 'Change in Glycemia is measured as changes in blood glucose levels.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['G1D, glucose, transporter'], 'conditions': ['GLUT1DS1']}, 'referencesModule': {'references': [{'pmid': '37268656', 'type': 'RESULT', 'citation': 'Avila A, Malaga I, Sirsi D, Kayani S, Primeaux S, Kathote GA, Jakkamsetti V, Kallem RR, Putnam WC, Park JY, Shinnar S, Pascual JM. Combination of triheptanoin with the ketogenic diet in Glucose transporter type 1 deficiency (G1D). Sci Rep. 2023 Jun 2;13(1):8951. doi: 10.1038/s41598-023-36001-x.'}]}, 'descriptionModule': {'briefSummary': 'To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.', 'detailedDescription': "This is a single site, open label proof of principle exploratory trial to investigate the compatibility of C7 oil with the ketogenic diet in subjects diagnosed with G1D. The ketogenic diet will have been previously described by the patient's treating physician independently of this study and for clinical reasons. The ketogenic diet supplies over 50% of calories from fat, subjects, who are already tolerating over this much fat as part of their previously prescribed ketogenic diet, will replace 45% of their daily caloric intake with the triheptanoin for 24 hours, during a 48 hour inpatient stay. Subjects will have a continuous EEG to monitor for any potential C7 related changes in seizure before, during, and after triheptanoin oil ingestion."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '35 Years', 'minimumAge': '30 Months', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical genotyping at a CLIA-certified laboratory.\n* Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have taken place for 2 months). The initiation of a ketogenic diet is previous to - and thus is not part of this study.\n* Males and females 30 months to 35 years and 11 months old inclusive.\n\nExclusion Criteria:\n\n* Subjects with evidence of independent, unrelated metabolic and/or genetic disease.\n* Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome, crohn's disease, or colitis that could increase the subject's risk of developing diarrhea or stomach pain.\n* Subjects with a BMI (body mass index) greater than or equal to 30.\n* Subjects currently not on ketogenic diet.\n* Women who are pregnant or breast feeding may not participate. Women who plan to become pregnant during the course of the study, or who are unwilling to use birth control to prevent pregnancy (including abstinence) may not participate. Females age 10 and over will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects will be asked to agree to abstinence or another form of birth control for the duration of the study.\n* Allergy/sensitivity to C7\n* Previous use of triheptanoin less than 1 month prior to study initiation.\n* Treatment with medium chain triglycerides in the last 24 hours.\n* Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain disorder (such as Alzheimer's disease) that would confound assessment of cognitive changes, in the opinion of the investigator.\n* Active drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with adherence to study requirements.\n* Inability or unwillingness of subject or legal guardian/representative to give written informed consent, or assent for children age 10-17,\n* Addition of a new antiseizure drug in the previous 3 months."}, 'identificationModule': {'nctId': 'NCT03301532', 'briefTitle': 'Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D', 'organization': {'class': 'OTHER', 'fullName': 'University of Texas Southwestern Medical Center'}, 'officialTitle': 'Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency', 'orgStudyIdInfo': {'id': 'STU 102015-091'}, 'secondaryIdInfos': [{'id': 'R01NS094257-01A1', 'link': 'https://reporter.nih.gov/quickSearch/R01NS094257-01A1', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Patients on a ketogenic diet', 'description': 'This is a one arm study were patients will be receiving an oil called triheptanoin. Patients will be consuming triheptanoin 4 times over the course of one day. The triheptanoin oil will take up 45% of their daily calories on the day the day they are taking the oil.', 'interventionNames': ['Drug: Triheptanoin']}], 'interventions': [{'name': 'Triheptanoin', 'type': 'DRUG', 'otherNames': ['C7 oil'], 'description': 'Dietary supplementation with triheptanoin', 'armGroupLabels': ['Patients on a ketogenic diet']}]}, 'contactsLocationsModule': {'locations': [{'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'UTexasSouthwestern', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}], 'overallOfficials': [{'name': 'Juan Pascual, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Study Principal Investigator'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Texas Southwestern Medical Center', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Institute of Neurological Disorders and Stroke (NINDS)', 'class': 'NIH'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor of Medicine', 'investigatorFullName': 'Juan Pascual', 'investigatorAffiliation': 'University of Texas Southwestern Medical Center'}}}}