Viewing Study NCT01818492

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Ignite Creation Date: 2025-12-24 @ 3:35 PM
Ignite Modification Date: 2025-12-29 @ 6:46 AM
Study NCT ID: NCT01818492
Status: COMPLETED
Last Update Posted: 2023-02-21
First Post: 2013-03-21
Is NOT Gene Therapy: True
Has Adverse Events: True
Brief Title: Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis
Sponsor:
Organization:
Overview Dates Organization Conditions Design Enrollment Locations Outcomes Modules Raw JSON

Raw JSON

{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Austria', 'Czechia', 'Turkey (Türkiye)']}, 'conditionBrowseModule': {'meshes': [{'id': 'D051359', 'term': 'Lymphohistiocytosis, Hemophagocytic'}], 'ancestors': [{'id': 'D015616', 'term': 'Histiocytosis, Non-Langerhans-Cell'}, {'id': 'D015614', 'term': 'Histiocytosis'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000644327', 'term': 'Emapalumab'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'CarlJohan.Treutiger@sobi.com', 'phone': '46 76 00 11 815', 'title': 'Carl Johan Treutiger', 'organization': 'Sobi AB'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation).', 'description': 'Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .', 'eventGroups': [{'id': 'EG000', 'title': 'Pre-conditioning', 'description': 'All patients before start of conditioning. Conditioning is the treatments used to prepare a patient for stem cell transplantation.', 'otherNumAtRisk': 45, 'deathsNumAtRisk': 45, 'otherNumAffected': 41, 'seriousNumAtRisk': 45, 'deathsNumAffected': 10, 'seriousNumAffected': 28}, {'id': 'EG001', 'title': 'Post-conditioning', 'description': 'All patients after start of conditioning. Conditioning is the treatments used to prepare a patient for stem cell transplantation.', 'otherNumAtRisk': 30, 'deathsNumAtRisk': 30, 'otherNumAffected': 28, 'seriousNumAtRisk': 30, 'deathsNumAffected': 5, 'seriousNumAffected': 20}], 'otherEvents': [{'term': 'Lymphadenopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Lymphocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Thrombotic microangiopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 4}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Bradycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pericardial effusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Stomatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Condition aggravated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 18}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Mucosal inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 8}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 15}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Graft versus host disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Graft versus host disease in gastrointestinal tract', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Graft versus host disease in liver', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Graft versus host disease in skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 5}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'BK virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Clostridium difficile infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Cytomegalovirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Otitis externa', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Staphylococcal bacteraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Adenovirus test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Blood immunoglobulin G decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Human rhinovirus test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Polyomavirus test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Transaminases increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Fluid overload', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 4}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hyperglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypoalbuminaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 4}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypomagnesaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hyponatraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypophosphataemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Agitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Renal failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Atelectasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypoxia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pulmonary haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Tachypnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Drug eruption', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 4}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 6}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Rash erythematous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Rash macular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Rash maculo-papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Skin disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 19}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 9}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}], 'seriousEvents': [{'term': 'Haemolytic anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Lymphocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Thrombotic microangiopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Cardiac arrest', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Cardiac tamponade', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Cardiomyopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Cardiopulmonary failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 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'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Condition aggravated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Multiple organ dysfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 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'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Histoplasmosis disseminated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Necrotizing fasciitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pneumocystis jirovecii pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Respiratory syncytial virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Staphylococcal bacteraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Staphylococcal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Clostridium difficile colitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Clostridium difficile infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Klebsiella sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Bronchiolitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Enterococcal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Epstein-Barr virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Gastroenteritis rotavirus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pneumonia cytomegaloviral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pneumonia pseudomonal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Rhinovirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'BSCT failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Engraft failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Spinal compression fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Blood creatinine increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Failure to thrive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Cerebral disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Haemorrhage intracranial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Neurological decompensation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Subdural hygroma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'ARDS', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Acute respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Aspiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Haemothorax', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pulmonary alveolar haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pulmonary artery thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pulmonary haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Pulmonary hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Respiratory distress', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Circulatory collapse', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 45, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Overall Response Rate (ORR) Second Line', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '63.0', 'groupId': 'OG000', 'lowerLimit': '42.4', 'upperLimit': '80.6'}]}]}], 'analyses': [{'pValue': '0.0134', 'groupIds': ['OG000'], 'groupDescription': 'Pre-specified null hypothesis that ORR is at most 40%.', 'statisticalMethod': 'Exact binomial test', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'This test was undertaken at the one-sided 0.025 significance level.'}], 'paramType': 'NUMBER', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.\n\nCR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \\<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \\>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.\n\nPR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.\n\nHI: improvement (\\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Primary analysis set: Second Line (27 patients who had previously received conventional HLH therapy before enrollment, data collected by regulatory cut-off: 20 July 2017)'}, {'type': 'PRIMARY', 'title': 'Overall Response Rate (ORR) All Treated', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '64.7', 'groupId': 'OG000', 'lowerLimit': '46.5', 'upperLimit': '80.3'}]}]}], 'analyses': [{'pValue': '0.0031', 'groupIds': ['OG000'], 'groupDescription': 'Pre-specified null hypothesis that ORR is at most 40%', 'statisticalMethod': 'Exact binomial test', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'This test was undertaken at the one-sided 0.025 significance level.'}], 'paramType': 'NUMBER', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.\n\nCR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \\<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \\>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.\n\nPR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.\n\nHI: improvement (\\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Primary analysis set: All Treated (all patients who received any part of an emapalumab infusion, data collected by regulatory cut-off: 20 July 2017)'}, {'type': 'PRIMARY', 'title': 'Overall Response Rate (ORR) Follow-on Analysis Set:', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '58.8', 'groupId': 'OG000', 'lowerLimit': '40.7', 'upperLimit': '75.4'}]}]}], 'analyses': [{'pValue': '0.0205', 'groupIds': ['OG000'], 'groupDescription': 'Pre-specified null hypothesis that ORR is at most 40%', 'statisticalMethod': 'Exact binomial test', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'This test was undertaken at the one-sided 0.025 significance level.'}], 'paramType': 'NUMBER', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.\n\nCR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \\<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \\>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.\n\nPR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.\n\nHI: improvement (\\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Follow-on analysis set: Second Line (34 patients who had previously received conventional HLH therapy before enrollment, totality of the data collected in the NI-0501-04 study)'}, {'type': 'PRIMARY', 'title': 'Overall Response Rate (ORR) at End of Treatment in Study NI-0501-04 (EOT 04) Follow-on Analysis Set: All Treated', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '60.0', 'groupId': 'OG000', 'lowerLimit': '44.3', 'upperLimit': '74.3'}]}]}], 'analyses': [{'pValue': '0.0053', 'groupIds': ['OG000'], 'groupDescription': 'Pre-specified null hypothesis that ORR is at most 40%.', 'statisticalMethod': 'Exact binomial test', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'This test was undertaken at the one-sided 0.025 significance level.'}], 'paramType': 'NUMBER', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.\n\nCR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \\<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \\>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.\n\nPR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.\n\nHI: improvement (\\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Follow-on analysis set: All Treated (45 patients who received any part of an emapalumab infusion, totality of the data collected in the NI-0501-04)'}, {'type': 'SECONDARY', 'title': 'Time to Response', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '7.0', 'groupId': 'OG000', 'lowerLimit': '6.0', 'upperLimit': '9.0'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Time from the date of the first dose of emapalumab to first achievement of response (at least HLH improvement)', 'unitOfMeasure': 'Count of days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'All Treated (all patients who received any part of an emapalumab infusion)'}, {'type': 'SECONDARY', 'title': 'Durability of First Response', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '58.0', 'comment': 'Upper limit of 95% CI is not estimable due to too few events.', 'groupId': 'OG000', 'lowerLimit': '39.0', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Maintenance of response achieved any time during the study', 'unitOfMeasure': 'Count of days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'All Treated (all patients who received any part of an emapalumab infusion)'}, {'type': 'SECONDARY', 'title': 'Overall Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '42', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Time from the date of first dose to last dose, or 8 weeks after first dose.', 'description': 'Number of patients being alive at end of treatment or at week 8, pending on which comes first.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All Treated (all patients who received any part of an emapalumab infusion)'}, {'type': 'SECONDARY', 'title': 'Number of Patients Able to Reduce Glucocorticoids', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'title': 'Reduction ≥50%', 'categories': [{'measurements': [{'value': '46.7', 'groupId': 'OG000'}]}]}, {'title': 'Reduction ≥30%-<50%', 'categories': [{'measurements': [{'value': '11.1', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks.', 'description': 'Number of patients able to reduce glucocorticoids by 50% or more and between ≥30%-\\<50%, of baseline dose at EOT 04.', 'unitOfMeasure': 'Percentage of patients', 'reportingStatus': 'POSTED', 'populationDescription': 'All Treated (all patients who received any part of an emapalumab infusion)'}, {'type': 'SECONDARY', 'title': 'Cumulative Duration of Response', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '78.6', 'groupId': 'OG000', 'lowerLimit': '33.3', 'upperLimit': '91.9'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'up to start of HSCT conditioning, whenever HSCT conditioning is scheduled (at least 4 weeks after treatment start), or End of Treatment 04/05 (if the patient did not have HSCT performed)', 'description': 'Percent of treatment time in response from the first achievement of an Overall Response until HSCT conditioning, or End of Treatment 04/05 (if the patient did not have HSCT performed) Where applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05.', 'unitOfMeasure': 'Percentage of treatment time', 'dispersionType': 'Inter-Quartile Range', 'reportingStatus': 'POSTED', 'populationDescription': 'All Treated (all patients who received any part of an emapalumab infusion)'}, {'type': 'SECONDARY', 'title': 'Survival Pre-HSCT', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'title': 'Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.83', 'groupId': 'OG000', 'lowerLimit': '0.669', 'upperLimit': '0.915'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.73', 'groupId': 'OG000', 'lowerLimit': '0.524', 'upperLimit': '0.859'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Assessed up to HSCT, whenever HSCT occurred (up to approximately 6 months after treatment initiation)', 'description': 'Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients who receive HSCT will be censored at that date; patients who did not receive HSCT will be censored at last date of contact.\n\nWhere applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05.', 'unitOfMeasure': 'Kaplan-Meier survival probability', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'All Treated (all patients who received any part of an emapalumab infusion)'}, {'type': 'SECONDARY', 'title': 'Survival Post-HSCT', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'classes': [{'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.82', 'groupId': 'OG000', 'lowerLimit': '0.621', 'upperLimit': '0.921'}]}]}, {'title': 'Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.82', 'groupId': 'OG000', 'lowerLimit': '0.621', 'upperLimit': '0.921'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Assessed up to Last Observation (up to 1 year after transplant, or up to approximately 18 months after treatment initiation)', 'description': 'Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients without an event will be censored at last assessment date in either the NI-0501-04 or NI-0501-05 study. Patients who do not proceed to HSCT will be excluded from this analysis.', 'unitOfMeasure': 'Kaplan-Meier survival probability', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'All Treated (all patients who received any part of an emapalumab infusion)'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '45'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '35'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Additional therapy when not allowed PP', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}]}]}], 'preAssignmentDetails': 'Patients were screened within 1 week prior to the first administration of emapalumab (NI-0501). 66 patients were screened, and 45 were enrolled and treated.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'NI-0501', 'description': 'All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000', 'lowerLimit': '0.1', 'upperLimit': '13'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Age, Customized', 'classes': [{'title': '<2 years', 'categories': [{'measurements': [{'value': '32', 'groupId': 'BG000'}]}]}, {'title': '≥ 2 - < 12 years', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}, {'title': '≥ 12 - <18 years', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '23', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '22', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '30', 'groupId': 'BG000'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}, {'title': 'African Descent', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}, {'title': 'Mixed/multi-racial', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'LOther', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '23', 'groupId': 'BG000'}]}]}, {'title': 'Europe', 'categories': [{'measurements': [{'value': '22', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'HLH disease chracteristics at baseline', 'classes': [{'title': 'Fever', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}, {'title': 'Splenomegaly', 'categories': [{'measurements': [{'value': '27', 'groupId': 'BG000'}]}]}, {'title': 'Platelet counts < 100 x10^9/L', 'categories': [{'measurements': [{'value': '31', 'groupId': 'BG000'}]}]}, {'title': 'Neutrophil counts < 1 x10^9/L', 'categories': [{'measurements': [{'value': '25', 'groupId': 'BG000'}]}]}, {'title': 'Ferritin > 500 μg/L', 'categories': [{'measurements': [{'value': '38', 'groupId': 'BG000'}]}]}, {'title': 'Ferritin > 2000 μg/L', 'categories': [{'measurements': [{'value': '30', 'groupId': 'BG000'}]}]}, {'title': 'Fibrinogen < 1.5 g/L', 'categories': [{'measurements': [{'value': '20', 'groupId': 'BG000'}]}]}, {'title': 'Triglycerides > 3 mmol/L', 'categories': [{'measurements': [{'value': '29', 'groupId': 'BG000'}]}]}, {'title': 'D-Dimers > 500 μg/L', 'categories': [{'measurements': [{'value': '33', 'groupId': 'BG000'}]}]}, {'title': 'ALT > 125 IU/L', 'categories': [{'measurements': [{'value': '21', 'groupId': 'BG000'}]}]}, {'title': 'Total bilirubin > 25 μmol/L', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}, {'title': 'LDH > 1000 IU/L', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}, {'title': 'CNS involvement', 'categories': [{'measurements': [{'value': '15', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2016-03-24', 'size': 1611229, 'label': 'Study Protocol: US protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-06-17T17:09', 'hasProtocol': True}, {'date': '2016-02-26', 'size': 1697857, 'label': 'Study Protocol: EU protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-06-17T17:10', 'hasProtocol': True}, {'date': '2019-09-20', 'size': 26997737, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_002.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-06-19T15:27', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 45}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-04', 'completionDateStruct': {'date': '2019-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-01-23', 'studyFirstSubmitDate': '2013-03-21', 'resultsFirstSubmitDate': '2020-06-26', 'studyFirstSubmitQcDate': '2013-03-25', 'lastUpdatePostDateStruct': {'date': '2023-02-21', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-08-17', 'studyFirstPostDateStruct': {'date': '2013-03-26', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-09-04', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Overall Response Rate (ORR) Second Line', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.\n\nCR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \\<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \\>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.\n\nPR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.\n\nHI: improvement (\\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).'}, {'measure': 'Overall Response Rate (ORR) All Treated', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.\n\nCR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \\<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \\>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.\n\nPR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.\n\nHI: improvement (\\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).'}, {'measure': 'Overall Response Rate (ORR) Follow-on Analysis Set:', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.\n\nCR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \\<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \\>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.\n\nPR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.\n\nHI: improvement (\\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).'}, {'measure': 'Overall Response Rate (ORR) at End of Treatment in Study NI-0501-04 (EOT 04) Follow-on Analysis Set: All Treated', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.\n\nCR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \\<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \\>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.\n\nPR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.\n\nHI: improvement (\\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).'}], 'secondaryOutcomes': [{'measure': 'Time to Response', 'timeFrame': 'Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Time from the date of the first dose of emapalumab to first achievement of response (at least HLH improvement)'}, {'measure': 'Durability of First Response', 'timeFrame': 'Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)', 'description': 'Maintenance of response achieved any time during the study'}, {'measure': 'Overall Survival', 'timeFrame': 'Time from the date of first dose to last dose, or 8 weeks after first dose.', 'description': 'Number of patients being alive at end of treatment or at week 8, pending on which comes first.'}, {'measure': 'Number of Patients Able to Reduce Glucocorticoids', 'timeFrame': 'End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks.', 'description': 'Number of patients able to reduce glucocorticoids by 50% or more and between ≥30%-\\<50%, of baseline dose at EOT 04.'}, {'measure': 'Cumulative Duration of Response', 'timeFrame': 'up to start of HSCT conditioning, whenever HSCT conditioning is scheduled (at least 4 weeks after treatment start), or End of Treatment 04/05 (if the patient did not have HSCT performed)', 'description': 'Percent of treatment time in response from the first achievement of an Overall Response until HSCT conditioning, or End of Treatment 04/05 (if the patient did not have HSCT performed) Where applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05.'}, {'measure': 'Survival Pre-HSCT', 'timeFrame': 'Assessed up to HSCT, whenever HSCT occurred (up to approximately 6 months after treatment initiation)', 'description': 'Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients who receive HSCT will be censored at that date; patients who did not receive HSCT will be censored at last date of contact.\n\nWhere applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05.'}, {'measure': 'Survival Post-HSCT', 'timeFrame': 'Assessed up to Last Observation (up to 1 year after transplant, or up to approximately 18 months after treatment initiation)', 'description': 'Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients without an event will be censored at last assessment date in either the NI-0501-04 or NI-0501-05 study. Patients who do not proceed to HSCT will be excluded from this analysis.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Emapalumab'], 'conditions': ['Primary Haemophagocytic Lymphohistiocytosis']}, 'referencesModule': {'references': [{'pmid': '38662147', 'type': 'DERIVED', 'citation': 'Brossard P, Laveille C. Population Pharmacokinetics of the Anti-Interferon-Gamma Monoclonal Antibody Emapalumab: An Updated Analysis. Rheumatol Ther. 2024 Jun;11(3):869-880. doi: 10.1007/s40744-024-00669-y. Epub 2024 Apr 25.'}, {'pmid': '32374962', 'type': 'DERIVED', 'citation': 'Locatelli F, Jordan MB, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington TP, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, De Benedetti F, Grom A, Lapeyre G, Jacqmin P, Ballabio M, de Min C. Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis. N Engl J Med. 2020 May 7;382(19):1811-1822. doi: 10.1056/NEJMoa1911326.'}], 'seeAlsoLinks': [{'url': 'https://www.clinicaltrials.gov/ct2/show/NCT02069899', 'label': 'NI-0501-05, long-term follow-up study description on clinicaltrials.gov'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment.', 'detailedDescription': 'The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment.\n\nAll participants in the NI-0501-04 study (NCT01818492) were invited to participate in the long-term follow-up study NI-0501-05 (NCT02069899). For the primary completion date, mentioned here, we refer to the NI-0501-04 study, even though in accordance with the NI-0501-04 study objectives, namely the assessment of long-term efficacy and safety endpoints, the study analyses also included data collected in the long-term follow-up study NI-0501-05. Hence these data are reported together. Study NI-0501-05 accepts patients from NI-0501-04 and NI-0501-06. Data collection for the patients from NI-0501-04 is completed.\n\nThe primary efficacy and safety analyses are based on the regulatory cut-off date of 20 July 2017. Refer to the publication in N Engl J Med 2020 May 7; 382 (19):1811-1822. Follow-on analyses have been conducted on all patients enrolled in the study, i.e. including the patients enrolled after the cut-off date of 20 July 2017. The results reported here refer to the totality of the 45 patients enrolled.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Gender: male and female\n* Age: up to and including 18 years at diagnosis of Haemophagocytic Lymphohistiocytosis\n* Primary HLH patients\n* Patient (if ≥ 18 years old), or patient's legal representative(s) must have signed informed consent\n\nExclusion Criteria:\n\n* Diagnosis of secondary Haemophagocytic Lymphohistiocytosis consequent to a proven rheumatic or neoplastic disease.\n* Body weight \\< 3 kg.\n* Patients treated with biologics within a specific timeframe\n* Active Mycobacteria, Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter and Leishmania infections.\n* Presence of malignancy.\n* Concomitant disease or malformation severely affecting the cardiovascular, pulmonary, liver or renal function"}, 'identificationModule': {'nctId': 'NCT01818492', 'briefTitle': 'Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Swedish Orphan Biovitrum'}, 'officialTitle': 'A Phase 2/3 Open-label Single Arm Multicentre Study to Assess Safety Tolerability Pharmacokinetics and Efficacy of i.v. Administrations of NI-0501 an Anti-IFNγ mAb in Paediatric Patients With Primary Haemophagocytic Lymphohistiocytosis', 'orgStudyIdInfo': {'id': 'NI-0501-04'}, 'secondaryIdInfos': [{'id': '2012-003632-23', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'NI-0501', 'description': 'NI-0501 administered by IV infusion at a starting dose of 1 mg/kg.', 'interventionNames': ['Biological: NI-0501']}], 'interventions': [{'name': 'NI-0501', 'type': 'BIOLOGICAL', 'armGroupLabels': ['NI-0501']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045-7106', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '19803', 'city': 'Wilmington', 'state': 'Delaware', 'country': 'United States', 'facility': 'Alfred I. duPont Hospital for Children - Nemours Center for Cancer and Blood Disorders - Division of Pediatric Hematology Oncology', 'geoPoint': {'lat': 39.74595, 'lon': -75.54659}}, {'zip': '30329', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Children's Healthcare of Atlanta", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Dana-Farber Cancer Institute (DFCI)', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '27599', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina at Chapel Hill', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '45229-3039', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital - Division of Immunobiology - Department of Pediatrics", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': "Texas Children's Cancer Center", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84113', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': "Primary Children's Hospital", 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '48149', 'city': 'Münster', 'country': 'Germany', 'facility': "University Children's Hospital", 'geoPoint': {'lat': 51.96236, 'lon': 7.62571}}, {'zip': '50139', 'city': 'Florence', 'country': 'Italy', 'facility': 'Azienda Ospedaliero Universitaria Meyer', 'geoPoint': {'lat': 43.77925, 'lon': 11.24626}}, {'zip': '16147', 'city': 'Genoa', 'country': 'Italy', 'facility': 'Istituto Giannina Gaslini', 'geoPoint': {'lat': 44.40478, 'lon': 8.94439}}, {'zip': '20900', 'city': 'Monza', 'country': 'Italy', 'facility': 'Azienda Ospedaliera San Gerardo', 'geoPoint': {'lat': 45.58005, 'lon': 9.27246}}, {'zip': '35128', 'city': 'Padua', 'country': 'Italy', 'facility': 'Azienda Ospedaliera Padova - Clinica di Oncoematologia Pediatrica', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'zip': '00165', 'city': 'Roma', 'country': 'Italy', 'facility': "Ospedale Pediatrico Bambino Gesu'", 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '37126', 'city': 'Verona', 'country': 'Italy', 'facility': 'Ospedale Donna Bambino - U.O.C. Oncoematologia Pediatrica', 'geoPoint': {'lat': 45.43854, 'lon': 10.9938}}, {'zip': '08950', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Déu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '119-129', 'city': 'Barcelona', 'country': 'Spain', 'facility': "Hospital Universitario Vall d'Hebron", 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '28009', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario Niño Jesús', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '17176', 'city': 'Stockholm', 'country': 'Sweden', 'facility': 'Karolinska University Hospital', 'geoPoint': {'lat': 59.32938, 'lon': 18.06871}}, {'zip': 'WC1N 3JH', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital - Department of Haematology', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Radmila Kanceva, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Swedish Orphan Biovitrum'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Swedish Orphan Biovitrum', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Seventh Framework Programme', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}