Ignite Creation Date:
2025-12-25 @ 2:23 AM
Ignite Modification Date:
2026-03-06 @ 11:46 PM
Study NCT ID:
NCT04088734
Status:
TERMINATED
Last Update Posted:
2023-07-25
First Post:
2019-09-11
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-07-03', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D009084', 'term': 'Mucopolysaccharidosis III'}], 'ancestors': [{'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@ultragenyx.com', 'phone': '1-888-756-8567', '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': 'All-Cause Mortality: From enrollment to the end of study; mean of 18 months. Adverse Events: from first dose of study drug up to Day 454.', 'eventGroups': [{'id': 'EG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 5, 'seriousNumAtRisk': 5, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Mixed deafness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Cushingoid', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Gait disturbance', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Amylase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Blood creatine phosphokinase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Blood lactate dehydrogenase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Haematocrit increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Haemoglobin increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Platelet count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Red blood cell count increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Weight increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'White blood cell count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Increased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Bursitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Myositis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Synovial cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Ataxia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Cognitive disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Dyskinesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Epilepsy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Fine motor skill dysfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Abnormal behaviour', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Agitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Initial insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Rhinitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Dermatitis atopic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Ecchymosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}], 'seriousEvents': [{'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Gait disturbance', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Foreign body in gastrointestinal tract', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Cognitive disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Mental status changes', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Gastrostomy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': '< 30 days : Possible; Mild', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '< 30 days : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '< 30 days : Possible; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '< 30 days : Probable; Mild', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': '< 30 days : Probable; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '< 30 days : Probable; Severe', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '< 30 days : Definitely; Mild', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '< 30 days : Definitely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '< 30 days : Definitely; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Possible; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Possible; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Probable; Mild', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Probable; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Probable; Severe', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Definitely; Mild', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Definitely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '30 - < 60 days : Definitely; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Possible; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Possible; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Probable; Mild', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Probable; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Probable; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Definitely; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Definitely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Definitely; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Possible; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Possible; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Probable; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Probable; Moderate', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Probable; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Definitely; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Definitely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Definitely; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Possible; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Possible; Severe', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Probable; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Probable; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Probable; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Definitely; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Definitely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 - < 12 months : Definitely; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12', 'description': 'An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': '60 - < 90 days : Unrelated; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Unrelated; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Unrelated; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Unlikely; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Unlikely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Unlikely; Severe', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Possible; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Possible; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Probable; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Probable; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Probable; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Definite; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Definite; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '60 - < 90 days : Definite; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Unrelated; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Unrelated; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Unrelated; Severe', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Unlikely; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Unlikely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Unlikely; Severe', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Possible; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Possible; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Probable; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Probable; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Probable; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Definite; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Definite; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '90 - < 180 days : Definite; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Unrelated; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Unrelated; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Unrelated; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Unlikely; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Unlikely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Unlikely; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Possible; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Possible; Severe', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Probable; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Probable; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Probable; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Definite; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Definite; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '180 days - < 12 months : Definite; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Unrelated; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Unrelated; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Unrelated; Severe', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Unlikely; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Unlikely; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Unlikely; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Possible; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Possible; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Possible; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Probable; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Probable; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Probable; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Definite; Mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Definite; Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '>= 12 months : Definite; Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months)', 'description': 'An SAE is defined as any untoward medical occurrence that, at any dose:\n\n1. Results in death\n2. Is life threatening\n3. Requires inpatient hospitalization or prolongation of existing hospitalization\n4. Results in persistent disability/incapacity\n5. Is a congenital anomaly/birth defect\n6. Other situations such as important medical events that may not be immediately life threatening or result in death or hospitalization but may jeopardize the participant or may require medical or surgical intervention to prevent one of the other outcomes listed in the above definition.\n\nRelationship to study drug was defined as unrelated, unlikely, possible, probable, or definitely.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Liver volume: change from BL to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.4200', 'spread': '0.08876', 'groupId': 'OG000'}]}]}, {'title': 'Liver volume: change from BL to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.4543', 'spread': '0.31560', 'groupId': 'OG000'}]}]}, {'title': 'Liver volume: change from BL to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.1765', 'spread': '0.17466', 'groupId': 'OG000'}]}]}, {'title': 'Spleen volume: change from BL to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.0603', 'spread': '0.19257', 'groupId': 'OG000'}]}]}, {'title': 'Spleen volume: change from BL to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2253', 'spread': '0.53267', 'groupId': 'OG000'}]}]}, {'title': 'Spleen volume: change from BL to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.1330', 'spread': '0.18102', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, 180, Month 12', 'description': 'As Measured by Magnetic Resonance Imaging (MRI). Baseline value of multiple of normal is calculated using the baseline values of the Liver volume/Spleen Volume/Height and Weight.\n\n* Body Surface Area (BSA) (m2)=( Height(cm) \\* Weight (kg)/3600)1/2.\n* Normal Liver Volume=(689.9 \\* BSA (m)) - 24.7.\n* Normal Spleen Volume (mL)=(4.6 \\* Weight (kg)) + 0.7.\n* Liver Volume (multiples of normal)=Subject Liver Volume (mL)/Normal Liver Volume (mL).\n* Spleen Volume (multiples of normal)=Subject Spleen Volume (mL)/Normal Spleen Volume (mL).', 'unitOfMeasure': 'ratio', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at baseline and given time point'}, {'type': 'PRIMARY', 'title': 'Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from BL to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.120', 'spread': '0.0837', 'groupId': 'OG000'}]}]}, {'title': 'Change from BL to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.183', 'spread': '0.0289', 'groupId': 'OG000'}]}]}, {'title': 'Change from BL to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.100', 'spread': 'NA', 'comment': '1 participant analyzed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'description': 'Change from baseline in CSF heparan sulfate levels after treatment', 'unitOfMeasure': 'nmol/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Plasma Heparan Sulfate After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-31.0', 'spread': '9.62', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-25.0', 'spread': '7.07', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-17.5', 'spread': '17.68', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'unitOfMeasure': 'pmol/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Urine Glycosaminoglycans After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-14.5', 'spread': '9.33', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-13.7', 'spread': '9.81', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.0', 'spread': 'NA', 'comment': '1 participant analyzed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'unitOfMeasure': 'mg/mmol creatinine', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Urine Heparan Sulfate After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.95', 'spread': '0.676', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.63', 'spread': '0.651', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.60', 'spread': 'NA', 'comment': '1 participant analyzed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'unitOfMeasure': 'μmol/mmol creatinine', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.662', 'spread': '5.9524', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.297', 'spread': '5.7100', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.000', 'spread': 'NA', 'comment': '1 participant analyzed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, and Month 12', 'unitOfMeasure': 'nmol/17 h/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.578', 'spread': '1.1299', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.000', 'spread': '0.0000', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.025', 'spread': '0.0354', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'unitOfMeasure': 'nmol/17 h/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Plasma SGSH After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.50', 'spread': '8.682', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.88', 'spread': '1.825', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.05', 'spread': '0.495', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'unitOfMeasure': 'nmol/17 h/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Brain Volumes After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change in amygdala volume', 'categories': [{'measurements': [{'value': '-0.635', 'spread': '0.2333', 'groupId': 'OG000'}]}]}, {'title': 'Change in corpus callosum volume', 'categories': [{'measurements': [{'value': '0.285', 'spread': '0.5445', 'groupId': 'OG000'}]}]}, {'title': 'Change in CSF volume', 'categories': [{'measurements': [{'value': '1.290', 'spread': '0.5091', 'groupId': 'OG000'}]}]}, {'title': 'Change in total cerebellar gray matter volume', 'categories': [{'measurements': [{'value': '-7.210', 'spread': '7.2125', 'groupId': 'OG000'}]}]}, {'title': 'Change in total cerebellar white matter volume', 'categories': [{'measurements': [{'value': '0.985', 'spread': '4.0234', 'groupId': 'OG000'}]}]}, {'title': 'Change in total brain volume', 'categories': [{'measurements': [{'value': '-157.705', 'spread': '44.6114', 'groupId': 'OG000'}]}]}, {'title': 'Change in total cerebral volume', 'categories': [{'measurements': [{'value': '-152.365', 'spread': '59.1353', 'groupId': 'OG000'}]}]}, {'title': 'Change in total cerebral gray matter volume', 'categories': [{'measurements': [{'value': '-145.975', 'spread': '81.0981', 'groupId': 'OG000'}]}]}, {'title': 'Change in total cortical volume', 'categories': [{'measurements': [{'value': '-144.170', 'spread': '84.9235', 'groupId': 'OG000'}]}]}, {'title': 'Change in total cerebellar volume', 'categories': [{'measurements': [{'value': '-6.225', 'spread': '11.2218', 'groupId': 'OG000'}]}]}, {'title': 'Change in total cerebral white matter volume', 'categories': [{'measurements': [{'value': '-6.385', 'spread': '21.9698', 'groupId': 'OG000'}]}]}, {'title': 'Change in total gray matter volume', 'categories': [{'measurements': [{'value': '-153.190', 'spread': '73.8927', 'groupId': 'OG000'}]}]}, {'title': 'Change in total intercranial volume', 'categories': [{'measurements': [{'value': '-122.245', 'spread': '40.0576', 'groupId': 'OG000'}]}]}, {'title': 'Change in total white matter volume', 'categories': [{'measurements': [{'value': '-5.400', 'spread': '25.9932', 'groupId': 'OG000'}]}]}, {'title': 'Change in ventricular volumes', 'categories': [{'measurements': [{'value': '34.165', 'spread': '5.0417', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, 12 months', 'description': 'As measured by MRI', 'unitOfMeasure': 'mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Brain Volumes After Treatment: Average Total Cortical Thickness', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.455', 'spread': '0.2475', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, 12 months', 'description': 'As measured by MRI', 'unitOfMeasure': 'mm', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment'}, {'type': 'SECONDARY', 'title': "Change From Baseline in Sleep Pattern as Measured by the Modified Children's Sleep Habits Questionnaire (CSHQ) Subscore Total After Treatment", 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.8', 'spread': '4.76', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.5', 'spread': '3.54', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': 'CSHQ is a caregiver-completed, 35-item questionnaire that assesses the frequency of behaviors associated with common pediatric sleep difficulties. Eight domains of sleep, including Bedtime Resistance, Sleep Onset Delay, Sleep Duration, Sleep Anxiety, Night Awakenings, Parasomnias, Sleep Disordered Breathing, and Daytime Sleepiness are assessed, producing eight individual subdomain scores and an overall CSHQ subscore total. CSHQ total score is calculated by adding all the 8 subscores, and ranges from 36 to 108. A higher score is indicative of more disturbed sleep.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Core Generic Scales Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-11.60', 'spread': '23.218', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-23.80', 'spread': '6.647', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': 'PedsQL is a brief measure of health-related quality of life in children. The Peds QL Generic Core Scales was used in the study, consisting of 23 items applicable for healthy school and community populations, as well as pediatric populations with acute and chronic conditions. The four scales include Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Item scores are added together and averaged to produce a Core total score, where higher scores on a scale of 0-100 indicate better Health-related Quality of Life (HRQOL).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Parent Quality of Life, Using the Parenting Stress Index, 4th Edition (PSI-4) Total Stress Raw Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-14.0', 'spread': '24.33', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-9.0', 'spread': '4.24', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': 'The Parenting Stress Index, 4th Edition evaluates the magnitude and type of stress in a parent/child relationship. The short form version was used in the study, consisting of 36 items divided into three domains: Parental Distress (PD), Parent-Child Dysfunctional Interaction (P-CDI), and Difficult Child (DC), which combine together to form a Total Stress raw score. Total Stress raw scores can range from 36 - 180, with higher raw scores indicating higher levels of stress.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Gastrointestinal Symptoms Using the PedsQL™ Gastrointestinal (GI) Symptoms Scales Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5.88', 'spread': '9.261', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.15', 'spread': '6.010', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': "The PedsQL Gastrointestinal Symptoms Scale is a specific module of the PedsQL that measures gastrointestinal symptoms in patients with acute and chronic health conditions as well as healthy school and community populations. The Parent Report version was used on the study, consisting of 58 items across 10 dimensions, assessing parents' perceptions of their child's GI-specific symptoms. Item scores are added together and averaged to produce a GI symptoms scales score, where higher scores on a scale of 0-100 indicate better GI specific QOL.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Parent Global Impression (PGI) Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change at Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.0', 'spread': '6.48', 'groupId': 'OG000'}]}]}, {'title': 'Change at Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.0', 'spread': '6.56', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': "The Parent Global Impression scale evaluates all aspects of a patients' health and assesses if there has been an improvement or decline in clinical status, as reported by the parent/caregiver. This study used a modified version with symptoms relevant to the patient population in the trial. Nine symptoms were scored at each visit, using a 7-point rating scale where 3 = much better, 2 = better, 1 = slightly better, 0 = same, -1 = slightly worse, -2 = worse, and -3 = much worse. The nine symptom scores are added together to produce a PGI total score, ranging from -27 to 27.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Clinical Global Impression Improvement Scale at Day 180 and Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Global Improvement, Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'title': '0 = not assessed', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '1 = very much improved', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '2 = much improved', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '3 = minimally improved', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '4 = no change', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': '5 = minimally worse', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': '6 = much worse', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': '7 = very much worse', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Global Improvement, Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'title': '0 = not assessed', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '1 = very much improved', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '2 = much improved', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '3 = minimally improved', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': '4 = no change', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '5 = minimally worse', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': '6 = much worse', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': '7 = very much worse', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 180, Month 12', 'description': "The Clinical Global Impression of Improvement scale is a brief, stand-alone assessment of the clinician's view of the patient's global functioning prior to and after initiating a study medication, specifically looking at whether the patient has demonstrated improvement or not. Assessment of improvement was scored at each visit, using a 7-point rating scale where 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Parent Symptoms Score Questionnaire', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'timeFrame': 'Baseline, Day 180, Month 12', 'description': 'The Parent Symptoms Score Questionnaire contains 29 symptoms with an indicator of whether the symptom was present or absent.', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to the inconsistent use of forms across visits and sites for this study, the data collected for this endpoint was incomplete, unreliable, and could not be evaluated.'}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Body Mass Index After Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.581', 'spread': '4.4477', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.875', 'spread': '9.0517', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.873', 'spread': '6.1672', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Abnormalities in Standard Awake 45-Minutes-Electroencephalogram (EEG) Monitoring at Baseline and Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Overall EEG : Baseline', 'categories': [{'title': 'Abnormal, CS', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}, {'title': 'Abnormal, NCS', 'measurements': [{'value': '3', 'groupId': 'OG000'}]}, {'title': 'Normal', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Overall EEG : Day 180', 'categories': [{'title': 'Abnormal, CS', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': 'Abnormal, NCS', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': 'Normal', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, Day 180', 'description': 'NCS=not clinically significant CS=clinically significant', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Vector Shedding Analysis in Plasma, Saliva, Stool and Urine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'classes': [{'title': 'Plasma Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '45115.0', 'spread': '45413.33', 'groupId': 'OG000'}]}]}, {'title': 'Plasma Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2686.3', 'spread': '5372.50', 'groupId': 'OG000'}]}]}, {'title': 'Plasma Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2250.0', 'spread': '3181.98', 'groupId': 'OG000'}]}]}, {'title': 'Saliva Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '17472.5', 'spread': '18345.89', 'groupId': 'OG000'}]}]}, {'title': 'Saliva Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1900.3', 'spread': '2330.09', 'groupId': 'OG000'}]}]}, {'title': 'Saliva Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': 'NA', 'comment': 'Not applicable; 1 participant analyzed', 'groupId': 'OG000'}]}]}, {'title': 'Stool Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-277522.5', 'spread': '760910.43', 'groupId': 'OG000'}]}]}, {'title': 'Stool Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-800833.0', 'spread': '1132548.89', 'groupId': 'OG000'}]}]}, {'title': 'Stool Change from Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': 'NA', 'comment': 'Not applicable; 1 participant analyzed', 'groupId': 'OG000'}]}]}, {'title': 'Urine Change from Baseline to Day 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2250.0', 'spread': '2598.08', 'groupId': 'OG000'}]}]}, {'title': 'Urine Change from Baseline to Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1500.0', 'spread': '2598.08', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'description': 'Detection of the adeno-associated Virus 9 (AAV9) viral deoxyribonucleic acid (DNA) in plasma, saliva, urine and feces was analyzed. Per protocol, data were not collected for urine at Month 12.', 'unitOfMeasure': 'Copies per 1 mL of Specimen (copies/mL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with an assessment at given time point'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}], 'dropWithdraws': [{'type': 'Other, Not Specified', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'scAAV9.U1a.hSGSH, 3x1013 vg/kg', 'description': 'An intravenous injection of ABO-102 (scAAV9.U1a.hSGSH) via peripheral limb vein at a dose of 3.0 x 1013 vg/kg'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '69.02', 'spread': '34.692', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'months', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', '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': '4', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-09-22', 'size': 1732625, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-06-09T16:46', 'hasProtocol': True}, {'date': '2022-12-15', 'size': 1466045, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-06-09T16:46', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'whyStopped': 'Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-09-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-07', 'dispFirstSubmitDate': '2023-01-11', 'completionDateStruct': {'date': '2022-03-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-07-02', 'studyFirstSubmitDate': '2019-09-11', 'resultsFirstSubmitDate': '2023-06-09', 'studyFirstSubmitQcDate': '2019-09-11', 'dispFirstPostDateStruct': {'date': '2023-07-25', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2023-07-25', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-07-02', 'studyFirstPostDateStruct': {'date': '2019-09-13', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-07-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-03-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame', 'timeFrame': 'From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12', 'description': 'An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely.'}, {'measure': 'Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame', 'timeFrame': 'From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months)', 'description': 'An SAE is defined as any untoward medical occurrence that, at any dose:\n\n1. Results in death\n2. Is life threatening\n3. Requires inpatient hospitalization or prolongation of existing hospitalization\n4. Results in persistent disability/incapacity\n5. Is a congenital anomaly/birth defect\n6. Other situations such as important medical events that may not be immediately life threatening or result in death or hospitalization but may jeopardize the participant or may require medical or surgical intervention to prevent one of the other outcomes listed in the above definition.\n\nRelationship to study drug was defined as unrelated, unlikely, possible, probable, or definitely.'}, {'measure': 'Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment', 'timeFrame': 'Baseline, Day 30, 180, Month 12', 'description': 'As Measured by Magnetic Resonance Imaging (MRI). Baseline value of multiple of normal is calculated using the baseline values of the Liver volume/Spleen Volume/Height and Weight.\n\n* Body Surface Area (BSA) (m2)=( Height(cm) \\* Weight (kg)/3600)1/2.\n* Normal Liver Volume=(689.9 \\* BSA (m)) - 24.7.\n* Normal Spleen Volume (mL)=(4.6 \\* Weight (kg)) + 0.7.\n* Liver Volume (multiples of normal)=Subject Liver Volume (mL)/Normal Liver Volume (mL).\n* Spleen Volume (multiples of normal)=Subject Spleen Volume (mL)/Normal Spleen Volume (mL).'}, {'measure': 'Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'description': 'Change from baseline in CSF heparan sulfate levels after treatment'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Plasma Heparan Sulfate After Treatment', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12'}, {'measure': 'Change From Baseline in Urine Glycosaminoglycans After Treatment', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12'}, {'measure': 'Change From Baseline in Urine Heparan Sulfate After Treatment', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12'}, {'measure': 'Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment', 'timeFrame': 'Baseline, Day 30, Day 180, and Month 12'}, {'measure': 'Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12'}, {'measure': 'Change From Baseline in Plasma SGSH After Treatment', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12'}, {'measure': 'Change From Baseline in Brain Volumes After Treatment', 'timeFrame': 'Baseline, 12 months', 'description': 'As measured by MRI'}, {'measure': 'Change From Baseline in Brain Volumes After Treatment: Average Total Cortical Thickness', 'timeFrame': 'Baseline, 12 months', 'description': 'As measured by MRI'}, {'measure': "Change From Baseline in Sleep Pattern as Measured by the Modified Children's Sleep Habits Questionnaire (CSHQ) Subscore Total After Treatment", 'timeFrame': 'Baseline, Day 180, Month 12', 'description': 'CSHQ is a caregiver-completed, 35-item questionnaire that assesses the frequency of behaviors associated with common pediatric sleep difficulties. Eight domains of sleep, including Bedtime Resistance, Sleep Onset Delay, Sleep Duration, Sleep Anxiety, Night Awakenings, Parasomnias, Sleep Disordered Breathing, and Daytime Sleepiness are assessed, producing eight individual subdomain scores and an overall CSHQ subscore total. CSHQ total score is calculated by adding all the 8 subscores, and ranges from 36 to 108. A higher score is indicative of more disturbed sleep.'}, {'measure': 'Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Core Generic Scales Total Score', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': 'PedsQL is a brief measure of health-related quality of life in children. The Peds QL Generic Core Scales was used in the study, consisting of 23 items applicable for healthy school and community populations, as well as pediatric populations with acute and chronic conditions. The four scales include Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Item scores are added together and averaged to produce a Core total score, where higher scores on a scale of 0-100 indicate better Health-related Quality of Life (HRQOL).'}, {'measure': 'Change From Baseline in Parent Quality of Life, Using the Parenting Stress Index, 4th Edition (PSI-4) Total Stress Raw Score', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': 'The Parenting Stress Index, 4th Edition evaluates the magnitude and type of stress in a parent/child relationship. The short form version was used in the study, consisting of 36 items divided into three domains: Parental Distress (PD), Parent-Child Dysfunctional Interaction (P-CDI), and Difficult Child (DC), which combine together to form a Total Stress raw score. Total Stress raw scores can range from 36 - 180, with higher raw scores indicating higher levels of stress.'}, {'measure': 'Change From Baseline in Gastrointestinal Symptoms Using the PedsQL™ Gastrointestinal (GI) Symptoms Scales Score', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': "The PedsQL Gastrointestinal Symptoms Scale is a specific module of the PedsQL that measures gastrointestinal symptoms in patients with acute and chronic health conditions as well as healthy school and community populations. The Parent Report version was used on the study, consisting of 58 items across 10 dimensions, assessing parents' perceptions of their child's GI-specific symptoms. Item scores are added together and averaged to produce a GI symptoms scales score, where higher scores on a scale of 0-100 indicate better GI specific QOL."}, {'measure': 'Change From Baseline in Parent Global Impression (PGI) Total Score', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': "The Parent Global Impression scale evaluates all aspects of a patients' health and assesses if there has been an improvement or decline in clinical status, as reported by the parent/caregiver. This study used a modified version with symptoms relevant to the patient population in the trial. Nine symptoms were scored at each visit, using a 7-point rating scale where 3 = much better, 2 = better, 1 = slightly better, 0 = same, -1 = slightly worse, -2 = worse, and -3 = much worse. The nine symptom scores are added together to produce a PGI total score, ranging from -27 to 27."}, {'measure': 'Clinical Global Impression Improvement Scale at Day 180 and Month 12', 'timeFrame': 'Day 180, Month 12', 'description': "The Clinical Global Impression of Improvement scale is a brief, stand-alone assessment of the clinician's view of the patient's global functioning prior to and after initiating a study medication, specifically looking at whether the patient has demonstrated improvement or not. Assessment of improvement was scored at each visit, using a 7-point rating scale where 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse."}, {'measure': 'Change From Baseline in Parent Symptoms Score Questionnaire', 'timeFrame': 'Baseline, Day 180, Month 12', 'description': 'The Parent Symptoms Score Questionnaire contains 29 symptoms with an indicator of whether the symptom was present or absent.'}, {'measure': 'Percent Change From Baseline in Body Mass Index After Treatment', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12'}, {'measure': 'Number of Participants With Abnormalities in Standard Awake 45-Minutes-Electroencephalogram (EEG) Monitoring at Baseline and Day 180', 'timeFrame': 'Baseline, Day 180', 'description': 'NCS=not clinically significant CS=clinically significant'}, {'measure': 'Change From Baseline in Vector Shedding Analysis in Plasma, Saliva, Stool and Urine', 'timeFrame': 'Baseline, Day 30, Day 180, Month 12', 'description': 'Detection of the adeno-associated Virus 9 (AAV9) viral deoxyribonucleic acid (DNA) in plasma, saliva, urine and feces was analyzed. Per protocol, data were not collected for urine at Month 12.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['MPS IIIA', 'Sanfilippo', 'Gene therapy'], 'conditions': ['MPS IIIA', 'Sanfilippo Syndrome', 'Sanfilippo A', 'Mucopolysaccharidosis III']}, 'referencesModule': {'references': [{'pmid': '27331076', 'type': 'BACKGROUND', 'citation': 'Fu H, Cataldi MP, Ware TA, Zaraspe K, Meadows AS, Murrey DA, McCarty DM. Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery. Mol Ther Methods Clin Dev. 2016 Jun 8;3:16036. doi: 10.1038/mtm.2016.36. eCollection 2016.'}, {'pmid': '29064732', 'type': 'BACKGROUND', 'citation': 'Fu H, Meadows AS, Pineda RJ, Kunkler KL, Truxal KV, McBride KL, Flanigan KM, McCarty DM. Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. Hum Gene Ther Clin Dev. 2017 Dec;28(4):187-196. doi: 10.1089/humc.2017.109. Epub 2017 Oct 24.'}]}, 'descriptionModule': {'briefSummary': 'Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein', 'detailedDescription': 'This is an open-label, single dose clinical trial. All participants will receive 3 X 10\\^13 vg/kg of ABO-102 delivered one time through a venous catheter inserted into a peripheral limb vein. The target population includes MPS IIIA participants with a DQ lower than 60 in middle and advanced phases of the disease. Similar numbers of MPS IIIA participants with age equivalent above and below 18 months of age will be enrolled to ensure a representation of middle and advanced phases of the disease.\n\nThis study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Diagnosis of MPS IIIA confirmed by the following methods:\n\n 1. No detectable or significantly reduced SGSH enzyme activity by leukocyte assay and\n 2. Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene\n* Cognitive Development Quotient (DQ) lower than 60 (calculated by Bayley Scales of Infant and Toddler Development - Third Edition)\n* Must be ambulatory, though may receive assistance with ambulation\n* Age range of 2 years up to 18 years (excluded)\n\nExclusion Criteria:\n\n* Inability to participate in the clinical evaluation as determined by Principal Investigator\n* Identification of two nonsense or null variants on genetic testing of the SGSH gene\n* At least one S298P mutation in the SGSH gene\n* Has evidence of an attenuated phenotype of MPS IIIA\n* Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics\n* Active viral infection based on clinical observations\n* Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer, or precludes the child from participating in the protocol assessments and follow up\n* Participants with total anti-AAV9 antibody titers greater than or equal to 1:100 as determined by ELISA binding immunoassay\n* Participants with a positive response for the ELISPOT for T-cell responses to AAV9\n* Serology consistent with exposure to HIV, or serology consistent with active hepatitis B or C infection\n* Bleeding disorder or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated according to local institutional policy\n* Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing\n* Any item (braces, etc.) which would exclude the participant from being able to undergo MRI according to local institutional policy\n* Any other situation that precludes the participant from undergoing procedures required in this study\n* Participants with cardiomyopathy or significant congenital heart abnormalities\n* The presence of significant non-MPS IlIA related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study\n* Abnormal laboratory values Grade 2 or higher as defined in CTCAE v4.03 for GGT, total bilirubin (except in subjects diagnosed with Gilbert's syndrome), creatinine, hemoglobin, WBC count, platelet count, PT and aPTT\n* Female participant who is pregnant or demonstrates a positive urine or beta-hCG result at screening assessment (if applicable)\n* Any vaccination with viral attenuated vaccines less than 30 days prior to the scheduled date of treatment (and use of prednisolone)\n* Previous treatment by Haematopoietic Stem Cell transplantation\n* Previous participation in a gene/cell therapy or ERT clinical trial\n* Participants who are anticipated to undergo a procedure involving anesthesia within 6 months post- drug administration\n* Dysphagia present at Grade 3 or higher, as defined in CTCAE v4.03"}, 'identificationModule': {'nctId': 'NCT04088734', 'briefTitle': 'Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ultragenyx Pharmaceutical Inc'}, 'officialTitle': 'A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease', 'orgStudyIdInfo': {'id': 'ABT-003'}, 'secondaryIdInfos': [{'id': 'UX111-CL201', 'type': 'OTHER', 'domain': 'Ultragenyx Pharmaceutical Inc'}, {'id': '2018-000504-42', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ABO-102', 'description': 'Dose of 3x10\\^13 vg/kg', 'interventionNames': ['Drug: ABO-102']}], 'interventions': [{'name': 'ABO-102', 'type': 'DRUG', 'otherNames': ['scAAV9.U1a.hSGSH', 'UX111'], 'description': 'Single dose of ABO-102 (scAAV9.U1a.hSGSH) administered by intravenous injection through a peripheral limb vein at a dose of 3 X 10\\^13 vg/kg', 'armGroupLabels': ['ABO-102']}]}, 'contactsLocationsModule': {'locations': [{'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Pittsburgh", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '5006', 'city': 'North Adelaide', 'state': 'South Australia', 'country': 'Australia', 'facility': "Adelaide Women's and Children's Hospital", 'geoPoint': {'lat': -34.90733, 'lon': 138.59141}}, {'zip': '15706', 'city': 'Santiago de Compostela', 'country': 'Spain', 'facility': 'Hospital Clínico Universitario de Santiago', 'geoPoint': {'lat': 42.88052, 'lon': -8.54569}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Ultragenyx Pharmaceutical Inc'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ultragenyx Pharmaceutical Inc', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Abeona Therapeutics, Inc', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}