Ignite Creation Date:
2025-12-25 @ 1:06 AM
Ignite Modification Date:
2025-12-25 @ 11:18 PM
Study NCT ID:
NCT02629393
Status:
COMPLETED
Last Update Posted:
2023-10-17
First Post:
2015-12-03
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['France', 'Germany', 'Italy', 'Malaysia', 'Taiwan']}, 'conditionBrowseModule': {'meshes': [{'id': 'C565372', 'term': 'Molybdenum Cofactor Deficiency, Complementation Group A'}, {'id': 'C535811', 'term': 'Molybdenum cofactor deficiency'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'info@bridgebio.com', 'phone': '650-391-9740', 'title': 'Business Development and Operations', 'organization': 'Origin Biosciences (affiliate of BridgeBio)'}, 'certainAgreement': {'otherDetails': "Institution/Investigator shall have the right to publish or present the results of Institution's and Investigator's activities including that Study Data which was obtained or derived at Institution. Institution/Investigator agree to submit any proposed publication/presentation to Sponsor for review at least 60 days prior to submitting any such proposed publication. Within 30 days of its receipt, Sponsor shall advise if any changes are needed to protect confidentiality.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Due to the small number of patients and no control, there was no SAP for this study. Results were reported by individual patient and were only descriptive. Results in tabular format are unavailable except for binary measures, such as survival and feeding ability. For more information and summary data, please refer to the referenced FDA approval package.'}}, 'adverseEventsModule': {'timeFrame': 'Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.', 'description': 'All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A.\n\nAll SAE data presented.', 'eventGroups': [{'id': 'EG000', 'title': 'Patients Treated With ORGN001 (Formerly ALXN1101)', 'description': 'All patients who received at least one dose of ORGN001 (formerly ALXN1101)', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 4, 'seriousNumAtRisk': 5, 'deathsNumAffected': 0, 'seriousNumAffected': 4}], 'otherEvents': [{'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Hypoglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Hyperbilirubinaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Anal fissure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Conjunctival haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Pathogen resistance', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Catheter site infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Rhinorrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Chiari network', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Dermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Staphylococcus test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Catheter site rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Otitis media acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Catheter site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Catheter site haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Catheter site swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Skin laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Cardiac failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Ventricular septal defect', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Eye discharge', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Oral herpes', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}], 'seriousEvents': [{'term': 'Apneoa', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Complication associated with device', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Bacteraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Catheter site swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Viral tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Gastrointestinal viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Central venous catheterisation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Device leakage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Product Issues', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Cardiac failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Seizures', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Respiratory syncytial virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Device related sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}, {'term': 'Device related infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (25.0)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Overall Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ORGN001 (Formerly ALXN1101)', 'description': 'ORGN001 (formerly ALXN1101)'}], 'classes': [{'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Through last observation (average of 24 months)', 'description': 'Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Feeding Pattern', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ORGN001 (Formerly ALXN1101)', 'description': 'Patients with a confirmed diagnosis of MOCD Type A and treated with at least one dose of ORGN001 (formerly ALXN1101)'}], 'classes': [{'title': 'Oral feeding at Baseline', 'categories': [{'title': 'Able to feed orally', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': 'Not able to feed orally', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Oral feeding at Month 12', 'categories': [{'title': 'Able to feed orally', 'measurements': [{'value': '3', 'groupId': 'OG000'}]}, {'title': 'Not able to feed orally', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At Month 12 visit', 'description': 'Number of patients who can feed orally', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Patients Treated With ORGN001 (Formerly ALXN1101)', 'description': 'All patients who received at least one dose of ORGN001 (formerly ALXN1101)'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}], 'dropWithdraws': [{'type': 'MOCD Type A not genetically confirmed', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Patients Treated With ORGN001 (Formerly ALXN1101)', 'description': 'All patients who received at least one dose of ORGN001 (formerly ALXN1101)'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000', 'lowerLimit': '1', 'upperLimit': '1004'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'days old', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Norway', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Israel', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Only patients with genetically confirmed MOCD Type A are included'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-01-29', 'size': 1202664, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2023-08-04T14:41', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-09', 'completionDateStruct': {'date': '2022-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-09-22', 'studyFirstSubmitDate': '2015-12-03', 'resultsFirstSubmitDate': '2023-08-10', 'studyFirstSubmitQcDate': '2015-12-10', 'lastUpdatePostDateStruct': {'date': '2023-10-17', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-09-22', 'studyFirstPostDateStruct': {'date': '2015-12-14', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2023-10-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Overall Survival', 'timeFrame': 'Through last observation (average of 24 months)', 'description': 'Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation.'}], 'secondaryOutcomes': [{'measure': 'Feeding Pattern', 'timeFrame': 'At Month 12 visit', 'description': 'Number of patients who can feed orally'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Molybdenum Cofactor Deficiency (MoCD)', 'Molybdenum Cofactor (MoCo) biosynthesis', 'sulfite oxidase (SO)', 'S sulfocysteine (SSC)', 'xanthine oxidoreductase', 'aldehyde oxidase'], 'conditions': ['Molybdenum Cofactor Deficiency, Type A']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214018Orig1s000TOC.cfm', 'label': 'FDA Drug Approval Package'}]}, 'descriptionModule': {'briefSummary': 'To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '5 Years', 'minimumAge': '1 Day', 'healthyVolunteers': False, 'eligibilityCriteria': "Patients must meet all of the following inclusion criteria to be considered for enrollment in this study:\n\n1. Male or female neonatal patient (1 to 28 days of age \\[inclusive\\] at the time of ORGN001 administration, with day 1 of age corresponding to the day of birth) or infant (29 days to \\<2 years of age) or child (2 to 5 years of age \\[inclusive\\]) with MoCD Type A, previously untreated with ORGN001 or treated with ORGN001 through Compassionate Use/Individual Named Patient access\n2. In neonates, diagnosis of MoCD Type A, based on:\n\n Prenatal genetic diagnosis, or Onset of clinical and/or laboratory signs and symptoms consistent with MoCD Type A (eg, seizures, exaggerated startle response, high-pitched cry, axial hypotonia, limb hypertonia, feeding difficulties, elevated urinary sulfite and/or SSC, elevated xanthine in urine or blood, or low or absent uric acid in the urine or blood) within the first 28 days after birth\n3. In infants or children, diagnosis of MoCD Type A, based on:\n\n Confirmed genetic diagnosis (genetic confirmation of the diagnosis of MoCD Type A may be obtained after initiation of ORGN001 therapy in certain cases), biochemical profile, and clinical presentation consistent with MoCD Type A\n4. Parent or legal guardian must have signed the informed consent form (ICF) prior to any study procedures being performed\n\nPatients will be excluded from participating in the study if they meet any of the following criteria:\n\n1. Diagnosis other than MoCD Type A (may be determined after the initiation of study drug)\n2. Condition that is considered by the treating physician to be a contraindication to therapy, including evidence of abnormalities on brain imaging not attributable to MoCD Type A, or that might otherwise interfere with the patient's participation in the study, pose any additional risk for the patient, or confound patient assessments\n3. Antenatal and/or postnatal brain imaging prior to initiation of treatment with ORGN001 that indicates cortical or subcortical cystic encephalomalacia, clinically significant intracranial hemorrhage, or other abnormalities on brain imaging determined by the treating physician to be clinically significant\n4. Modified Glasgow Coma Scale (mGCS) for Infants and Children score of less than 7 for more than 24 hours (does not apply to children less than 1 day in age)."}, 'identificationModule': {'nctId': 'NCT02629393', 'briefTitle': 'Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A', 'organization': {'class': 'INDUSTRY', 'fullName': 'Origin Biosciences'}, 'officialTitle': 'A Phase 2/3, Multicenter, Multinational, Open Label Study to Evaluate the Efficacy and Safety of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A', 'orgStudyIdInfo': {'id': 'ALXN1101-MCD-202'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ORGN001 (formerly ALXN1101)', 'interventionNames': ['Drug: ORGN001 (formerly ALXN1101)']}], 'interventions': [{'name': 'ORGN001 (formerly ALXN1101)', 'type': 'DRUG', 'armGroupLabels': ['ORGN001 (formerly ALXN1101)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '48201', 'city': 'Detroit', 'state': 'Michigan', 'country': 'United States', 'facility': "Children's Hosptial of Michigan", 'geoPoint': {'lat': 42.33143, 'lon': -83.04575}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '53226-4874', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': "Children's Hospital of Wisconsin", 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '18341', 'city': 'Afula', 'country': 'Israel', 'facility': 'HaEmek Medical Center', 'geoPoint': {'lat': 32.60907, 'lon': 35.2892}}, {'city': 'Stavanger', 'country': 'Norway', 'facility': 'Stavanger Universitetssjukehus', 'geoPoint': {'lat': 58.97005, 'lon': 5.73332}}, {'zip': '08950', 'city': 'Esplugues de Llobregat', 'state': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Deu', 'geoPoint': {'lat': 41.37732, 'lon': 2.08809}}, {'zip': '06100', 'city': 'Ankara', 'country': 'Turkey (Türkiye)', 'facility': 'Hacettepe University of Medicine', 'geoPoint': {'lat': 39.91987, 'lon': 32.85427}}, {'city': 'Ankara', 'country': 'Turkey (Türkiye)', 'facility': 'Gazi University', 'geoPoint': {'lat': 39.91987, 'lon': 32.85427}}, {'zip': '07058', 'city': 'Antalya', 'country': 'Turkey (Türkiye)', 'facility': 'Akdeniz University Medical Faculty', 'geoPoint': {'lat': 36.90812, 'lon': 30.69556}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'state': 'Greater Manchester', 'country': 'United Kingdom', 'facility': 'Willink Biochemical Genetics Unit', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hosptial', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Liza Squires, M.D.', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Origin Biosciences'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Origin Biosciences', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}