Ignite Creation Date:
2025-12-25 @ 12:31 AM
Ignite Modification Date:
2026-01-21 @ 5:03 PM
Study NCT ID:
NCT02733367
Status:
COMPLETED
Last Update Posted:
2019-11-05
First Post:
2016-03-16
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Extension Study for Patients Entered Into Study Infacort 003
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000309', 'term': 'Adrenal Insufficiency'}], 'ancestors': [{'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'info@diurnal.co.uk', 'phone': '+44 (0) 2920 682069', 'title': 'Information Line', 'organization': 'Diurnal'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'This study was designed as a safety study to evaluate the long-term use of Infacort in routine clinical practice. Efficacy results should be viewed as exploratory and interpreted with care.'}}, 'adverseEventsModule': {'timeFrame': 'Adverse events were recorded from the time of the first intake of Infacort in this study until the final study visit (i.e. 29 months).', 'description': 'Details of any adverse events were collected, including date of onset, end date, frequency, severity, seriousness, relationship to Infacort, action taken, and outcome. Any adverse event was followed, whenever possible, until it returned to the baseline condition or became stable with no further change expected.', 'eventGroups': [{'id': 'EG000', 'title': 'Infacort', 'description': 'Infacort® granules\n\nInfacort®: Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.', 'otherNumAtRisk': 18, 'deathsNumAtRisk': 18, 'otherNumAffected': 14, 'seriousNumAtRisk': 18, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Secondary hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Thermal burn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Tooth injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Venomous sting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Scar', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Genitourinary operation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Circumcision', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Body temperature increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 6, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pharyngeal erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 45, 'numAffected': 10}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 7, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 4, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Dental caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 6, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 12, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Vulval disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Dermatitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Synovitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Scoliosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 6, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Enterovirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 11, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hand-foot-and-mouth disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Laryngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Molluscum contagiosum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Otitis media viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pharyngotonsilitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Roseola', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Tonsilitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 21, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Scarlet fever', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}], 'seriousEvents': [{'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Erysipelas', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Urinary Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pyelonephritis acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Infacort', 'description': 'Infacort® granules\n\nInfacort®: Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.'}], 'classes': [{'title': 'Treatment-Emergent Adverse Events (TEAE) - Overall', 'categories': [{'measurements': [{'value': '193', 'groupId': 'OG000'}]}]}, {'title': 'Serious TEAEs', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Severe TEAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate TEAEs', 'categories': [{'measurements': [{'value': '42', 'groupId': 'OG000'}]}]}, {'title': 'Mild TEAEs', 'categories': [{'measurements': [{'value': '151', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '29 months', 'description': 'The primary endpoint was the nature and occurrence of serious adverse events (SAEs) and adverse events (AEs) observed throughout the study. AEs were recorded from the time of the first intake of Infacort until the final visit.', 'unitOfMeasure': 'adverse events', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Growth Velocity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Infacort', 'description': 'Infacort® granules\n\nInfacort®: Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.'}], 'classes': [{'title': 'visit 6 - 11th Month', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6343', 'spread': '2.51996', 'groupId': 'OG000'}]}]}, {'title': 'Visit 10 - 23rd Month', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.9214', 'spread': '1.88757', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '29 months', 'description': 'Growth velocity standard deviation score (SDS). Body height/length (cm) was obtained at each visit by specially trained paediatric endocrine nurses or physicians using standard calibrated auxological methods.', 'unitOfMeasure': 'Standard Deviation Score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Reference data for growth velocity is only available for patients \\<=6 years old at time of assessment. Therefore n=2 \\& not 4 for C1 at Month 11. Subject 001 was re-enrolled so no Visit 10 data available and, therefore, n=1 for C1 at Month 23.'}, {'type': 'SECONDARY', 'title': 'Cortisol Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Infacort', 'description': 'Infacort® granules\n\nInfacort®: Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.'}], 'classes': [{'title': 'Visit 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '43.329', 'spread': '48.3217', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '22.022', 'spread': '27.1323', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '29 months', 'description': 'Cortisol levels measured from dried blood spots. The dried blood spots were analysed for multi-steroids, including cortisol (all subjects). Blood spot absolute laboratory values for the safety population are presented. A dried blood spot sample was collected at the initial and final visits, every month for the first 2 months of the study and thereafter every 6 months (unless required after 3 months).', 'unitOfMeasure': 'nmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The reason why n=14 at Visit 1 is because of no result for Subjects 005, 010, 017 \\& 018. N=12 at Final Visit due to 12 completers.'}, {'type': 'SECONDARY', 'title': 'Number of Participants Exhibiting a Change in Tanner Development Stage', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Infacort', 'description': 'Infacort® granules\n\nInfacort®: Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg).'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '29 months', 'description': 'The Tanner Development Stage was assessed as an additional analysis in this study. All assessments (breast, genitalia, and pubic hair) were Grade 1 (pre-pubertal) at baseline, with only 1 subject (in Cohort 2) showing a change during the study. Subject 018 showed progression to Grade 2 in the pubic hair category (sparse, pigmented hair mainly on labia).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': "'Count of Participants' data below represents the number of subjects that exhibited a change from baseline in their Tanner Development Stage."}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Infacort', 'description': 'Infacort® granules\n\nInfacort®: Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Infacort', 'description': 'Infacort® granules\n\nInfacort®: Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '18', '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': '1021.6', 'spread': '650.84', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'days', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '10', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '18', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', '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': '18', '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': 'Germany', 'categories': [{'measurements': [{'value': '18', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Body Mass Index', 'classes': [{'categories': [{'measurements': [{'value': '17.02', 'spread': '2.083', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kg/m^2', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Body Surface Area', 'classes': [{'categories': [{'measurements': [{'value': '0.582', 'spread': '0.1803', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'm^2', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Subjects recruited were those who had satisfactorily completed study Infacort 003 and agreed to participate in study Infacort 004. The investigator ensured that all subjects who were treated in study Infacort 003 were invited to participate.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-07-20', 'size': 8538966, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2019-08-09T05:38', 'hasProtocol': True}, {'date': '2017-02-01', 'size': 7069360, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2019-08-09T06:16', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 18}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-03-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-10', 'completionDateStruct': {'date': '2018-08-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-10-22', 'studyFirstSubmitDate': '2016-03-16', 'resultsFirstSubmitDate': '2019-08-09', 'studyFirstSubmitQcDate': '2016-04-05', 'lastUpdatePostDateStruct': {'date': '2019-11-05', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-08-09', 'studyFirstPostDateStruct': {'date': '2016-04-11', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-09-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-08-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)', 'timeFrame': '29 months', 'description': 'The primary endpoint was the nature and occurrence of serious adverse events (SAEs) and adverse events (AEs) observed throughout the study. AEs were recorded from the time of the first intake of Infacort until the final visit.'}], 'secondaryOutcomes': [{'measure': 'Growth Velocity', 'timeFrame': '29 months', 'description': 'Growth velocity standard deviation score (SDS). Body height/length (cm) was obtained at each visit by specially trained paediatric endocrine nurses or physicians using standard calibrated auxological methods.'}, {'measure': 'Cortisol Levels', 'timeFrame': '29 months', 'description': 'Cortisol levels measured from dried blood spots. The dried blood spots were analysed for multi-steroids, including cortisol (all subjects). Blood spot absolute laboratory values for the safety population are presented. A dried blood spot sample was collected at the initial and final visits, every month for the first 2 months of the study and thereafter every 6 months (unless required after 3 months).'}, {'measure': 'Number of Participants Exhibiting a Change in Tanner Development Stage', 'timeFrame': '29 months', 'description': 'The Tanner Development Stage was assessed as an additional analysis in this study. All assessments (breast, genitalia, and pubic hair) were Grade 1 (pre-pubertal) at baseline, with only 1 subject (in Cohort 2) showing a change during the study. Subject 018 showed progression to Grade 2 in the pubic hair category (sparse, pigmented hair mainly on labia).'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Adrenal Insufficiency']}, 'descriptionModule': {'briefSummary': 'A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia who had completed study Infacort 003. All subjects who had satisfactorily completed study Infacort 003 were offered the opportunity to take part in Infacort 004.', 'detailedDescription': 'A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with AI who had completed study Infacort 003 (EudraCT number 2014-002265-30). All subjects who had satisfactorily completed study Infacort 003 wiere offered the opportunity to participate in study Infacort 004 at or after their final visit of study Infacort 003. Subjects received the usual clinically-appropriate dose (since bioequivalence has been demonstrated with conventional hydrocortisone), as determined by the Investigator, which was administered according to usual clinical practice - generally 3 or 4 times a day. Subjects could continue to be treated in this study until they met the study withdrawal criteria, until Infacort® was commercially available locally (which has now been achieved), or until the Sponsor decided to discontinue the study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '6 Years', 'minimumAge': '1 Month', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nSubjects successfully completing study Infacort 003, whose inclusion criteria were:\n\n1. Male and female children less than 6 years of age.\n2. A diagnosis of adrenal insufficiency (AI) as confirmed by an inappropriately low cortisol usually with other supporting tests.\n3. Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).\n4. Adequately hydrated and nourished. In addition, the parents/carers must be able to understand and give written Informed Consent for this extension study.\n\nExclusion Criteria:\n\n1. Clinically evident acute AI (adrenal crisis) (Note: the subject can be re-evaluated for eligibility once the episode is over)\n2. Inability of the child to take oral therapy\n3. Subjects with clinical signs of acute infection or fever on inclusion (Note: the subject can be re-evaluated for eligibility once the episode is over)\n4. Any surgical or medical condition that in the opinion of the Investigator may place the subject at higher risk from his/her participation in the study\n5. Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons\n6. Subjects who are in a dependent relationship with the Investigator or the Sponsor'}, 'identificationModule': {'nctId': 'NCT02733367', 'briefTitle': 'Extension Study for Patients Entered Into Study Infacort 003', 'organization': {'class': 'INDUSTRY', 'fullName': 'Neurocrine UK Limited'}, 'officialTitle': 'Open-label, Long-term Follow-up of Safety and Biochemical Disease Control of Infacort® in Neonates, Infants and Children With Congenital Adrenal Hyperplasia and Adrenal Insufficiency Previously Enrolled in the Infacort 003 Study', 'orgStudyIdInfo': {'id': 'Infacort 004'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Infacort', 'description': 'Infacort® granules', 'interventionNames': ['Drug: Infacort®']}], 'interventions': [{'name': 'Infacort®', 'type': 'DRUG', 'description': 'Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg).', 'armGroupLabels': ['Infacort']}]}, 'contactsLocationsModule': {'locations': [{'zip': '13353', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Charité-Universitätsmedizin Berlin, CVK', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}], 'overallOfficials': [{'name': 'Wiegand', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Charite University, Berlin, Germany'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Neurocrine UK Limited', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}