Viewing Study NCT02599961

Home Icon Home Search Icon Search Certify Doc Icon Certify Doc Certify Doc Icon Genes Certify Doc Icon Clinical Trials Certify Doc Icon CompTox Processes Icon DrugMatrix Open Targets Icon Open Targets Processes Icon Products Support Icon Support Bugs Icon Bugs
Main Search Make This Study a Gene Therapy Make This Study a Not Gene Therapy Report Bug
Ignite Creation Date: 2025-12-24 @ 12:54 PM
Ignite Modification Date: 2025-12-27 @ 9:33 PM
Study NCT ID: NCT02599961
Status: TERMINATED
Last Update Posted: 2020-06-11
First Post: 2015-11-03
Is Gene Therapy: True
Has Adverse Events: True
Brief Title: Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
Sponsor:
Organization:
Overview Dates Organization Conditions Design Enrollment Locations Outcomes Modules Raw JSON

Raw JSON

{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C536830', 'term': 'Glut1 Deficiency Syndrome'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C531010', 'term': 'triheptanoin'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@ultragenyx.com', 'phone': '1-888-756-8657', 'title': 'Medical Information', 'organization': 'Ultragenyx Pharmaceutical Inc'}, 'certainAgreement': {'otherDetails': 'The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.', 'eventGroups': [{'id': 'EG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.', 'otherNumAtRisk': 15, 'deathsNumAtRisk': 15, 'otherNumAffected': 13, 'seriousNumAtRisk': 15, 'deathsNumAffected': 0, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Abdominal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Abdominal Pain Upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Breath Odour', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Flatulence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Haematochezia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Thirst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Seasonal Allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Ear Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Otitis Media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Otitis Media Acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pharyngitis Streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Upper Respiratory Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Viral Upper Respiratory Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Head Injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Ligament Sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Procedural Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Alanine Aminotransferase Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Blood Glucose Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Weight Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypoglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hyponatraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Back Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Muscle Spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Muscular Weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Musculoskeletal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pain In Extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Disturbance In Attention', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Dysarthria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Head Titubation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Petit Mal Epilepsy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Tremor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Dysmenorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Vulvovaginal Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Nasal Congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Oropharyngeal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hair Growth Abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hot Flush', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}], 'seriousEvents': [{'term': 'Intestinal Obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Croup Infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Ear Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Otitis Media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Otitis Media Acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'classes': [{'title': 'TEAEs', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': 'Serious TEAEs', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Related TEAEs', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': 'Serious and Related TEAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Grade 3 or 4 TEAEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Gastrointestinal TEAEs', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'TEAEs Leading to Treatment Discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'TEAEs Leading to Study Discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'TEAEs Leading to Death', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.', 'description': 'An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'classes': [{'title': 'Change at Month 0-3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-64.22', 'spread': '185.564', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 4-6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-64.19', 'spread': '142.460', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 7-9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-61.81', 'spread': '164.770', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 10-12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-91.93', 'spread': '216.834', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 13-18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-75.56', 'spread': '206.406', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 19-24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-110.12', 'spread': '233.492', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 25-30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-135.60', 'spread': '249.871', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 31-36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-51.88', 'spread': '101.378', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-82.73', 'ciLowerLimit': '-105.00', 'ciUpperLimit': '-60.46', 'pValueComment': 'From the GEE model which includes the reduction from baseline for each visit period as the response variable, baseline as covariates, visit period as a factor, identity link function and exchangeable within subject working correlation matrix.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '11.363', 'groupDescription': 'Month 0-3', 'statisticalMethod': 'generalized estimating equation (GEE)', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.0006', 'groupIds': ['OG000'], 'paramType': 'LS mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-54.91', 'ciLowerLimit': '-86.46', 'ciUpperLimit': '-23.36', 'pValueComment': 'From the GEE model which includes the reduction from baseline for each visit period as the response variable, baseline as covariates, visit period as a factor, identity link function and exchangeable within subject working correlation matrix.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '16.097', 'groupDescription': 'Month 4-6', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.0019', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-52.53', 'ciLowerLimit': '-85.62', 'ciUpperLimit': '-19.45', 'pValueComment': 'From the GEE model which includes the reduction from baseline for each visit period as the response variable, baseline as covariates, visit period as a factor, identity link function and exchangeable within subject working correlation matrix.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '16.882', 'groupDescription': 'Month 7-9', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-82.65', 'ciLowerLimit': '-113.13', 'ciUpperLimit': '-52.17', 'pValueComment': 'From the GEE model which includes the reduction from baseline for each visit period as the response variable, baseline as covariates, visit period as a factor, identity link function and exchangeable within subject working correlation matrix.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '15.550', 'groupDescription': 'Month 10-12', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36', 'description': 'The number of observable seizures were recorded by the subject or caregiver via diary throughout the study. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence.', 'unitOfMeasure': 'seizures per 4 weeks', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with a baseline and postbaseline assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in CNS Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'classes': [{'title': 'Change at Month 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.85', 'spread': '20.331', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.36', 'spread': '18.495', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.64', 'spread': '21.371', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.38', 'spread': '3.092', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': 'NA', 'comment': '1 participant assessed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36', 'description': 'The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance \\& Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone \\& Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with a baseline and postbaseline assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'classes': [{'title': 'Change at Month 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.25', 'spread': '16.917', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.56', 'spread': '21.522', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.67', 'spread': '9.475', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-9.35', 'spread': '12.702', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-8.96', 'spread': '20.075', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.74', 'spread': '2.273', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30', 'description': 'The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.', 'unitOfMeasure': 'T-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Pediatric participants with a baseline and postbaseline assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'classes': [{'title': 'Change at Month 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.59', 'spread': '11.049', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.29', 'spread': '12.366', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-7.43', 'spread': '15.514', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.60', 'spread': '14.987', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-8.25', 'spread': '15.316', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.91', 'spread': '12.599', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30', 'description': 'The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.', 'unitOfMeasure': 'T-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Pediatric participants with a baseline and postbaseline assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in SF-12v2 Health Survey PCS Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'classes': [{'title': 'Change at Month 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.25', 'spread': '5.077', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12.37', 'spread': '5.367', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.09', 'spread': '1.619', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.35', 'spread': 'NA', 'comment': '1 participant assessed.', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18', 'description': 'SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.', 'unitOfMeasure': 'T-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Adult participants with a baseline and postbaseline assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in SF-12v2 Health Survey MCS Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'classes': [{'title': 'Change at Month 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.63', 'spread': '2.249', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.84', 'spread': '3.316', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.90', 'spread': '5.706', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '16.96', 'spread': 'NA', 'comment': '1 participant assessed.', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18', 'description': 'SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.', 'unitOfMeasure': 'T-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Adult participants with a baseline and postbaseline assessment at given time point.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Subject Non-Compliance', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Discontinuation of Study by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '9'}]}, {'type': 'Other, Not Specified', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}]}], 'recruitmentDetails': 'Study enrolled pediatric, adolescent, and adult glucose transporter type 1 deficiency syndrome (Glut1 DS) participants who completed the UX007G-CL201 study (NCT01993186; rollover participants). No non-rollover participants (those from other clinical studies, investigator sponsored trials, or expanded access/compassionate use treatment) enrolled.', 'preAssignmentDetails': 'For continuing UX007G-CL201 participants, the Week 52 visit of that study may have been conducted in conjunction with the Baseline visit for this study to avoid duplication of assessments.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'UX007 (Triheptanoin)', 'description': 'UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '15.24', 'spread': '6.106', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Age, Customized', 'classes': [{'title': '2 to < 12 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}, {'title': '12 to < 18 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}, {'title': '18 to < 65 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '1', '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': '1', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'American Indian or Alaska Native', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Asian', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Black or African American', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'White', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'BG000'}]}]}, {'title': 'Other, Not Specified', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Overall Seizure Frequency Per 4 Weeks', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '312.17', 'spread': '528.057', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'The number of observable seizures were recorded by the subject or caregiver via diary. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence.', 'unitOfMeasure': 'seizures per 4 weeks', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Baseline (NCT01993186) Columbia Neurological Score (CNS) Total Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '38.63', 'spread': '25.428', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance \\& Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone \\& Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'participants with a baseline assessment'}, {'title': 'Baseline (NCT01993186) Short Form (SF) 10 (SF-10) Health Survey for Children Physical Summary Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '32.63', 'spread': '17.027', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.', 'unitOfMeasure': 'T-score', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'participants with a baseline assessment'}, {'title': 'Baseline (NCT01993186) SF-10 Health Survey for Children Psychosocial Summary Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '48.29', 'spread': '10.196', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.', 'unitOfMeasure': 'T-score', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'participants with a baseline assessment'}, {'title': 'Baseline (NCT01993186) SF12 Version 2 (SF-12v2) Health Survey Physical Component Summary (PCS) Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '39.37', 'spread': '0.523', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'SF-12v2 was assessed for adults aged \\>=18 years. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to US general population T-scores. Therefore, all scores obtained that are \\< 50 can be interpreted as below the US general population T-score and scores \\> 50 can be interpreted as above the US general population T-score. Higher global scores are associated with better quality of life.', 'unitOfMeasure': 'T-score', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'participants with a baseline assessment'}, {'title': 'Baseline (NCT01993186) SF-12v2 Health Survey MCS Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '43.49', 'spread': '4.356', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'SF-12v2 was assessed for adults aged \\>=18 years. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to US general population T-scores. Therefore, all scores obtained that are \\< 50 can be interpreted as below the US general population T-score and scores \\> 50 can be interpreted as above the US general population T-score. Higher global scores are associated with better quality of life.', 'unitOfMeasure': 'T-score', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'participants with a baseline assessment'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-09-20', 'size': 1771087, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-04-01T15:11', 'hasProtocol': True}, {'date': '2018-04-25', 'size': 529154, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-04-01T15:12', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'whyStopped': 'Study was halted prematurely due to lack of efficacy', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2015-09-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'nctId': 'NCT03773770', 'statusForNctId': 'AVAILABLE', 'hasExpandedAccess': True}, 'statusVerifiedDate': '2020-06', 'completionDateStruct': {'date': '2019-10-22', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-06-03', 'studyFirstSubmitDate': '2015-11-03', 'resultsFirstSubmitDate': '2020-04-17', 'studyFirstSubmitQcDate': '2015-11-05', 'lastUpdatePostDateStruct': {'date': '2020-06-11', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-04-17', 'studyFirstPostDateStruct': {'date': '2015-11-09', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-04-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-10-22', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths', 'timeFrame': 'From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.', 'description': 'An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks', 'timeFrame': 'Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36', 'description': 'The number of observable seizures were recorded by the subject or caregiver via diary throughout the study. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence.'}, {'measure': 'Change From Baseline Over Time in CNS Total Score', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36', 'description': 'The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance \\& Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone \\& Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function.'}, {'measure': 'Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30', 'description': 'The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.'}, {'measure': 'Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30', 'description': 'The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.'}, {'measure': 'Change From Baseline Over Time in SF-12v2 Health Survey PCS Score', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18', 'description': 'SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.'}, {'measure': 'Change From Baseline Over Time in SF-12v2 Health Survey MCS Score', 'timeFrame': 'Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18', 'description': 'SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Glut 1 DS'], 'conditions': ['Glucose Transporter Type 1 Deficiency Syndrome']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.ultragenyx.com/', 'label': 'Company Website'}]}, 'descriptionModule': {'briefSummary': 'The primary objective of the study is to evaluate the long-term safety of UX007 in Glut1 DS participants.', 'detailedDescription': 'The study will enroll up to 40 pediatric, adolescent and adult Glut 1 DS participants who have completed the UX007G-CL201 (NCT019933186) study and, at the discretion of the Sponsor, additional participants from other clinical studies, investigator sponsored trials (ISTs), or expanded access/compassionate use treatment.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of Glut 1 DS confirmed by cerebrospinal fluid glucose concentration. erythrocyte 3-O-methyl-D-glucose uptake assay, or solute carrier family 2 member 1 (SLC2A1) molecular genetic testing (Information obtained from Medical Records)\n* Males and females aged at least 1 year old at the time of informed consent\n* Completion of UX007G-CL201 study (NCT01993186). Glut1 DS patients who received UX007/triheptanoin treatment as apart of clinical studies, ISTs or expanded access/compassionate use treatment programs may be eligible at the discretion of the Sponsor\n* Provide written informed consent or verbal assent (if possible) with written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research related procedures\n* Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, and comply with accurate completion of the seizure diary\n* Females of childbearing potential must have a negative urine pregnancy test at Baseline and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have not experienced menarche, are post-menopausal (defined as having no menses for at least 12 months without an alternative medical cause), or are permanently sterile due to total hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.\n* Participants of child-bearing potential or fertile males with partners of child-bearing potential who are sexually active must consent to use a highly-effective method of contraception as determined by the investigator from the period following the signing of the informed consent through 30 days after last dose of study drug.\n\nExclusion Criteria:\n\n* Any known hypersensitivity to triheptanoin, that in the judgement of the investigator, places the subject at an increased risk for adverse effects\n* History of, or current suicidal ideation, behavior and/or attempts\n* Pregnant and/or breast feeding an infant\n* Unwilling or unable to discontinue use of prohibited medication (barbiturates, pancreatic lipase inhibitors) or other substance that may confound study objectives. Use of up to 3 concomitant antiepileptic drugs is allowed, provided dose has been stable at least 14 days prior to Baseline\n* Use of any Investigational Product, drug or supplement (other than UX007) within 30 days prior to Baseline, or at any time during the study\n* Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment\n* Has a concurrent disease or condition, or laboratory abnormality that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or introduce additional safety concerns'}, 'identificationModule': {'nctId': 'NCT02599961', 'briefTitle': 'Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ultragenyx Pharmaceutical Inc'}, 'officialTitle': 'An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome', 'orgStudyIdInfo': {'id': 'UX007G-CL202'}, 'secondaryIdInfos': [{'id': '2015-000389-69', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'UX007', 'description': 'UX007 dosing targeted and/or maintained at 35% of total daily caloric intake.', 'interventionNames': ['Drug: UX007']}], 'interventions': [{'name': 'UX007', 'type': 'DRUG', 'otherNames': ['Triheptanoin'], 'description': 'UX007 is a liquid intended for oral (PO) administration.', 'armGroupLabels': ['UX007']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado - University of Colorado, Denver, School of Medicine", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '33155', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': "Miami Children's Hospital", 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '76104', 'city': 'Fort Worth', 'state': 'Texas', 'country': 'United States', 'facility': "Cook Children's Medical Center", 'geoPoint': {'lat': 32.72541, 'lon': -97.32085}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '3084', 'city': 'Heidelberg', 'state': 'Victoria', 'country': 'Australia', 'facility': 'Melbourne Brain Centre', 'geoPoint': {'lat': -37.75, 'lon': 145.06667}}, {'zip': '2100', 'city': 'Copenhagen', 'country': 'Denmark', 'facility': 'Copenhagen University Hospital', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'zip': '08950', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan De Deu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': 'NE7 7DN', 'city': 'Newcastle upon Tyne', 'country': 'United Kingdom', 'facility': 'The Newcastle upon Tyne Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Ultragenyx Pharmaceutical Inc'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ultragenyx Pharmaceutical Inc', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}