Ignite Creation Date:
2025-12-24 @ 5:09 PM
Ignite Modification Date:
2026-02-01 @ 7:39 PM
Study NCT ID:
NCT02172950
Status:
COMPLETED
Last Update Posted:
2021-10-27
First Post:
2014-06-23
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Turkey (Türkiye)']}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@cslbehring.com', 'phone': 'use email contact', 'title': 'Clinical Trial Disclosure Manager', 'organization': 'CSL Behring'}, 'certainAgreement': {'otherDetails': 'CSL agreements and restrictions on publishing may vary with individual investigators; however, CSL will not prohibit any investigator from publishing. CSL supports the publication of results from all centers of a multi-center trial and generally requires that reports based on single-site data not precede the primary publication of the entire clinical trial.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Up to 5 years', 'eventGroups': [{'id': 'EG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII", 'otherNumAtRisk': 222, 'deathsNumAtRisk': 222, 'otherNumAffected': 130, 'seriousNumAtRisk': 222, 'deathsNumAffected': 1, 'seriousNumAffected': 23}, {'id': 'EG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII", 'otherNumAtRisk': 24, 'deathsNumAtRisk': 24, 'otherNumAffected': 23, 'seriousNumAtRisk': 24, 'deathsNumAffected': 0, 'seriousNumAffected': 14}], 'otherEvents': [{'term': 'Haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Allergy to arthropod sting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 5, 'numAffected': 2}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 18, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 44, 'numAffected': 15}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 12, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Head injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 11, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 13, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Coronavirus test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Inhibiting antibodies positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 19, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 13, 'numAffected': 6}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Bronchospasm', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Iron deficiency anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 29, 'numAffected': 20}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Ear pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Dental caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 11, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 8, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 28, 'numAffected': 20}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Haemarthrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 73, 'numAffected': 40}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 15, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 37, 'numAffected': 25}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 18, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 16, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 17, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 10, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 8, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 9, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 7, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Varicella', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 5, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Viral rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Scarlet fever', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Tinea capitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}], 'seriousEvents': [{'term': 'Shock', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Vasospasm', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Road traffic accident', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Abdominal injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Periprosthetic fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Skin laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Wound haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Anti factor VIII antibody positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Inhibiting antibodies positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Factor VIII inhibition', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Pregnancy of partner', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Social circumstances', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Generalised tonic-clonic seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Lennox-Gastaut syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Blindness transient', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Duodenal ulcer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Gastritis erosive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Gastrointestinal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Haemorrhoids', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Mouth haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Nephritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Haemophilic arthropathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Muscle haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Musculoskeletal stiffness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Tendinous contracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Lower respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Wound infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Device related infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Acinetobacter infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 222, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 24, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Incidence of Inhibitor Formation to FVIII in Previously Treated Patients (PTPs) With 100 Exposure Days (EDs) to CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '198', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At the closest visit after 100 EDs (up to 5 years).', 'unitOfMeasure': 'percentage of paticipants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population (SP) - comprised all the Enrolled Population who received at least 1 dose of rVIII-SingleChain (CSL627) during the study for any reason.'}, {'type': 'PRIMARY', 'title': 'Number of Previously Untreated Patients (PUPs) With High-titer Inhibitor Formation to FVIII With at Least 50 EDs to CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At the closest visit after 50 EDs (up to 5 years).', 'description': 'High-titer inhibitor is defined as an inhibitor titer of ≥ 5 Bethesda units/mL.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP'}, {'type': 'PRIMARY', 'title': 'Percent Treatment Success for Major Bleeding Episodes in PUPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}]}, {'units': 'Major bleeds', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to 5 years', 'description': 'Percentage of major bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator\'s clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Major bleeding episodes are defined as bleeding episodes for which a subject is required to seek treatment at the hemophilia center or that threatens the subject\'s life or loss of limb.', 'unitOfMeasure': 'percentage of treatment success', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Major bleeds', 'denomUnitsSelected': 'Major bleeds', 'populationDescription': 'Efficacy population (EP) - comprised all subjects in the Safety Population who received at least 1 dose of rVIII-SingleChain (CSL627) for either routine prophylaxis treatment or on-demand treatment during the study. This endpoint was analyzed for PUPs only.'}, {'type': 'PRIMARY', 'title': 'Annualized Spontaneous Bleeding Rate in PUPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'On-demand', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.9', 'spread': '2.252', 'groupId': 'OG000'}]}]}, {'title': 'Prophylaxis', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.04', 'spread': '6.374', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to 5 years', 'description': 'The annualized spontaneous bleeding rate for PUPs taking prophylaxis and on-demand treatment regimens.', 'unitOfMeasure': 'Number of Spontaneous bleeds per year', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EP. This endpoint was analyzed for PUPs only.'}, {'type': 'SECONDARY', 'title': 'Percentage of Bleeding Episodes Treated Successfully in PTPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'OG000'}]}, {'units': 'Number of treated bleeding events', 'counts': [{'value': '2413', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '87.1', 'groupId': 'OG000', 'lowerLimit': '75.3', 'upperLimit': '93.7'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to 5 years', 'description': 'Percentage of bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator\'s clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response".', 'unitOfMeasure': 'Percentage of treated bleeding events', 'dispersionType': '99% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Number of treated bleeding events', 'denomUnitsSelected': 'Number of treated bleeding events', 'populationDescription': 'EP'}, {'type': 'SECONDARY', 'title': 'Annualized Bleeding Rate in PTPs and PUPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'OG000'}, {'value': '23', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'OG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'On-demand', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '28.32', 'spread': '29.014', 'groupId': 'OG000'}, {'value': '5.12', 'spread': '5.331', 'groupId': 'OG001'}]}]}, {'title': 'Prophylaxis', 'denoms': [{'units': 'Participants', 'counts': [{'value': '209', 'groupId': 'OG000'}, {'value': '23', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2.84', 'spread': '4.981', 'groupId': 'OG000'}, {'value': '5.94', 'spread': '7.705', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to 5 years', 'description': 'The annualized bleeding rate for PTPs and PUPs taking prophylaxis and on-demand treatment regimens', 'unitOfMeasure': 'Number of bleeds per year', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EP'}, {'type': 'SECONDARY', 'title': 'Percentage of Bleeding Episodes Requiring 1, 2, 3, or > 3 Injections of CSL627 to Achieve Hemostasis in PTPs and PUPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'OG000'}, {'value': '24', 'groupId': 'OG001'}]}, {'units': 'Treated bleeds', 'counts': [{'value': '2413', 'groupId': 'OG000'}, {'value': '315', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'OG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': '1 infusion', 'categories': [{'measurements': [{'value': '71.5', 'groupId': 'OG000'}, {'value': '77.5', 'groupId': 'OG001'}]}]}, {'title': '2 infusions', 'categories': [{'measurements': [{'value': '14.8', 'groupId': 'OG000'}, {'value': '11.4', 'groupId': 'OG001'}]}]}, {'title': '3 infusions', 'categories': [{'measurements': [{'value': '6.9', 'groupId': 'OG000'}, {'value': '5.4', 'groupId': 'OG001'}]}]}, {'title': '>3 infusions', 'categories': [{'measurements': [{'value': '6.3', 'groupId': 'OG000'}, {'value': '3.5', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to 5 years', 'unitOfMeasure': 'Percentage of treated bleeds', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Treated bleeds', 'denomUnitsSelected': 'Treated bleeds', 'populationDescription': 'EP'}, {'type': 'SECONDARY', 'title': 'Mean Number of On-demand Infusions of CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'OG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'per participant per month', 'categories': [{'measurements': [{'value': '6.26', 'spread': '4.778', 'groupId': 'OG000'}, {'value': '1.23', 'spread': '1.296', 'groupId': 'OG001'}]}]}, {'title': 'per participant per year', 'categories': [{'measurements': [{'value': '75.18', 'spread': '57.335', 'groupId': 'OG000'}, {'value': '14.75', 'spread': '15.547', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to 5 years', 'unitOfMeasure': 'Number of infusions', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EP. This endpoint analyzes PTPs and PUPs that received on-demand treatment.'}, {'type': 'SECONDARY', 'title': 'Mean On-demand Dose Administered of CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'OG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'per participant per month', 'categories': [{'measurements': [{'value': '210.39', 'spread': '188.106', 'groupId': 'OG000'}, {'value': '41.93', 'spread': '44.643', 'groupId': 'OG001'}]}]}, {'title': 'per participant per year', 'categories': [{'measurements': [{'value': '2524.69', 'spread': '2257.278', 'groupId': 'OG000'}, {'value': '503.16', 'spread': '535.712', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to 5 years', 'unitOfMeasure': 'IU/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EP. This endpoint analyzes PTPs and PUPs that received on-demand treatment.'}, {'type': 'SECONDARY', 'title': 'Mean Prophylaxis Dose Administered of CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '211', 'groupId': 'OG000'}, {'value': '23', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'OG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'per participant per month', 'categories': [{'measurements': [{'value': '380.95', 'spread': '130.079', 'groupId': 'OG000'}, {'value': '389.30', 'spread': '243.191', 'groupId': 'OG001'}]}]}, {'title': 'per participant per year', 'categories': [{'measurements': [{'value': '4571.35', 'spread': '1560.944', 'groupId': 'OG000'}, {'value': '4671.54', 'spread': '2918.288', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to 5 years', 'unitOfMeasure': 'IU/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'EP. This endpoint analyzes PTPs and PUPs that received prophylaxis treatment.'}, {'type': 'SECONDARY', 'title': 'Mean Total Amount of CSL627 Administered During Surgery Period in PTPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '51663.0', 'spread': '62033.25', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day of surgery up to 336 hours post-surgery', 'unitOfMeasure': 'IU', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Surgery population'}, {'type': 'SECONDARY', 'title': 'Total Amount of CSL627 Administered During Surgery Period in PUPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'Participant 1', 'categories': [{'measurements': [{'value': '15693', 'groupId': 'OG000'}]}]}, {'title': 'Participant 2', 'categories': [{'measurements': [{'value': '5631', 'groupId': 'OG000'}]}]}, {'title': 'Participant 3', 'categories': [{'measurements': [{'value': '7330', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day of surgery up to 336 hours post-surgery', 'unitOfMeasure': 'IU', 'reportingStatus': 'POSTED', 'populationDescription': 'Surgery population'}, {'type': 'SECONDARY', 'title': 'Hemostatic Efficacy of rVIII-SingleChain for PTPs and PUPs Who Undergo Surgery', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'units': 'Surgeries', 'counts': [{'value': '32', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'OG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'Excellent', 'categories': [{'measurements': [{'value': '28', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'Good', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Poor/No response', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From the start of surgery through the post-operative recovery (generally up to 14 days after surgery)', 'description': 'The investigator will rate the efficacy of the rVIII-SingleChain treatment during surgery based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response".', 'unitOfMeasure': 'Number of surgeries', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Surgeries', 'denomUnitsSelected': 'Surgeries', 'populationDescription': 'Surgery population - comprised all subjects in the Safety Population who received at least 1 dose of rVIII-SingleChain for surgical prophylaxis'}, {'type': 'SECONDARY', 'title': 'Incidence of Inhibitor Formation to FVIII in PTPs After 10 EDs and After 50 EDs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '198', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'after 10 EDs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'after 50 EDs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to 5 years', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP'}, {'type': 'SECONDARY', 'title': 'Percentage of PTPs and PUPs Developing Antibodies Against CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'OG000'}, {'value': '24', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'OG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '15.3', 'groupId': 'OG000'}, {'value': '70.8', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP'}, {'type': 'SECONDARY', 'title': 'Percentage of PTPs and PUPs Developing Antibodies to Chinese Hamster Ovary (CHO) Proteins', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'OG000'}, {'value': '24', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'OG001', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP'}, {'type': 'SECONDARY', 'title': 'Number of PUPs With High-titer Inhibitor Formation to FVIII After 10 EDs With CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At the closest visit after 10 EDs (up to 5 years)', 'description': 'High-titer inhibitor is defined as an inhibitor titer of ≥ 5 Bethesda units/mL.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP'}, {'type': 'SECONDARY', 'title': 'Number of PUPs With Low-titer Inhibitor Formation to FVIII After 10 EDs and After 50 EDs With CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'title': 'after 10 EDs', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'after 50 EDs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At the closest visit after 10 and after 50 EDs (up to 5 years)', 'description': 'Low-titer inhibitor is defined as an inhibitor titer of less than 5 Bethesda units/mL.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP'}, {'type': 'SECONDARY', 'title': 'Incidence of Total Inhibitor Formation to FVIII in PUPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '50.0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to 5 years', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP'}, {'type': 'SECONDARY', 'title': 'Percent Treatment Success for Non-major Bleeding Episodes in PUPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}, {'units': 'Treated bleeding events', 'counts': [{'value': '315', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '92.1', 'groupId': 'OG000', 'lowerLimit': '87.0', 'upperLimit': '95.3'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to 5 years', 'description': 'Percentage of bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator\'s clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Non-major bleeding episodes are those not requiring treatment at the hemophilia center or not threatening subject\'s life or loss of limb.', 'unitOfMeasure': 'percentage of treated bleeding events', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Treated bleeding events', 'denomUnitsSelected': 'Treated bleeding events', 'populationDescription': 'EP. This endpoint was analyzed for PUPs only.'}, {'type': 'SECONDARY', 'title': 'Percentage of PUPs With Clinically Significant Abnormal Vital Signs Values After First Infusion of CSL627', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to 6 hours after first infusion', 'description': 'Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP. This endpoint was analyzed for PUPs only.'}, {'type': 'SECONDARY', 'title': 'Percentage of PUPs With Treatment-emergent Clinically Significant Abnormal Vital Signs Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CSL627: Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to 5 years', 'description': 'Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'SP. This endpoint was analyzed for PUPs only.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'FG001', 'title': 'Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '222'}, {'groupId': 'FG001', 'numSubjects': '24'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '197'}, {'groupId': 'FG001', 'numSubjects': '19'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '25'}, {'groupId': 'FG001', 'numSubjects': '5'}]}], 'dropWithdraws': [{'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'Lack of Efficacy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '10'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Patient traveling', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Patient went to other country', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Patient relocated overseas', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Patient moving', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Previously Treated Patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'BG001', 'title': 'Previously Untreated Patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.\n\nrVIII-SingleChain: Recombinant single-chain coagulation factor VIII"}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'title': '<=18 years', 'measurements': [{'value': '95', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '119', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '126', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '126', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'title': 'PTPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '222', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '22.5', 'spread': '14.55', 'groupId': 'BG000'}, {'value': '22.5', 'spread': '14.55', 'groupId': 'BG002'}]}]}, {'title': 'PUPs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '24', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '1.4', 'spread': '1.18', 'groupId': 'BG001'}, {'value': '1.4', 'spread': '1.18', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Age was analyzed separately as PTPs and PUPs'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '212', 'groupId': 'BG000'}, {'value': '23', 'groupId': 'BG001'}, {'value': '235', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '50', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '52', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '19', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '158', 'groupId': 'BG000'}, {'value': '15', 'groupId': 'BG001'}, {'value': '173', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Czechia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'United States', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '21', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '22', 'groupId': 'BG002'}]}]}, {'title': 'Malaysia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}]}, {'title': 'Thailand', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}, {'title': 'Portugal', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}, {'title': 'Austria', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}, {'title': 'Netherlands', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}, {'title': 'Ireland', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}, {'title': 'Poland', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '36', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '36', 'groupId': 'BG002'}]}]}, {'title': 'France', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}]}, {'title': 'Romania', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}, {'title': 'Hungary', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'Japan', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}]}, {'title': 'Philippines', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '18', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}]}, {'title': 'Ukraine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '17', 'groupId': 'BG002'}]}]}, {'title': 'United Kingdom', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Switzerland', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Spain', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}, {'title': 'Lebanon', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}, {'title': 'Canada', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Italy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}]}]}, {'title': 'South Africa', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '22', 'groupId': 'BG002'}]}]}, {'title': 'Georgia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}, {'title': 'Australia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}, {'title': 'Germany', 'denoms': [{'units': 'Participants', 'counts': [{'value': '222', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '246', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Age was analyzed separately as PTPs and PUPs'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-05-26', 'size': 5445030, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2021-08-30T11:05', 'hasProtocol': True}, {'date': '2020-10-02', 'size': 2066685, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2021-08-30T11:05', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 246}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-10-13'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-09', 'completionDateStruct': {'date': '2021-01-19', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-09-29', 'studyFirstSubmitDate': '2014-06-23', 'resultsFirstSubmitDate': '2021-08-30', 'studyFirstSubmitQcDate': '2014-06-23', 'lastUpdatePostDateStruct': {'date': '2021-10-27', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-09-29', 'studyFirstPostDateStruct': {'date': '2014-06-24', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-10-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-01-19', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Inhibitor Formation to FVIII in Previously Treated Patients (PTPs) With 100 Exposure Days (EDs) to CSL627', 'timeFrame': 'At the closest visit after 100 EDs (up to 5 years).'}, {'measure': 'Number of Previously Untreated Patients (PUPs) With High-titer Inhibitor Formation to FVIII With at Least 50 EDs to CSL627', 'timeFrame': 'At the closest visit after 50 EDs (up to 5 years).', 'description': 'High-titer inhibitor is defined as an inhibitor titer of ≥ 5 Bethesda units/mL.'}, {'measure': 'Percent Treatment Success for Major Bleeding Episodes in PUPs', 'timeFrame': 'Up to 5 years', 'description': 'Percentage of major bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator\'s clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Major bleeding episodes are defined as bleeding episodes for which a subject is required to seek treatment at the hemophilia center or that threatens the subject\'s life or loss of limb.'}, {'measure': 'Annualized Spontaneous Bleeding Rate in PUPs', 'timeFrame': 'Up to 5 years', 'description': 'The annualized spontaneous bleeding rate for PUPs taking prophylaxis and on-demand treatment regimens.'}], 'secondaryOutcomes': [{'measure': 'Percentage of Bleeding Episodes Treated Successfully in PTPs', 'timeFrame': 'Up to 5 years', 'description': 'Percentage of bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator\'s clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response".'}, {'measure': 'Annualized Bleeding Rate in PTPs and PUPs', 'timeFrame': 'Up to 5 years', 'description': 'The annualized bleeding rate for PTPs and PUPs taking prophylaxis and on-demand treatment regimens'}, {'measure': 'Percentage of Bleeding Episodes Requiring 1, 2, 3, or > 3 Injections of CSL627 to Achieve Hemostasis in PTPs and PUPs', 'timeFrame': 'Up to 5 years'}, {'measure': 'Mean Number of On-demand Infusions of CSL627', 'timeFrame': 'Up to 5 years'}, {'measure': 'Mean On-demand Dose Administered of CSL627', 'timeFrame': 'Up to 5 years'}, {'measure': 'Mean Prophylaxis Dose Administered of CSL627', 'timeFrame': 'Up to 5 years'}, {'measure': 'Mean Total Amount of CSL627 Administered During Surgery Period in PTPs', 'timeFrame': 'Day of surgery up to 336 hours post-surgery'}, {'measure': 'Total Amount of CSL627 Administered During Surgery Period in PUPs', 'timeFrame': 'Day of surgery up to 336 hours post-surgery'}, {'measure': 'Hemostatic Efficacy of rVIII-SingleChain for PTPs and PUPs Who Undergo Surgery', 'timeFrame': 'From the start of surgery through the post-operative recovery (generally up to 14 days after surgery)', 'description': 'The investigator will rate the efficacy of the rVIII-SingleChain treatment during surgery based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response".'}, {'measure': 'Incidence of Inhibitor Formation to FVIII in PTPs After 10 EDs and After 50 EDs', 'timeFrame': 'Up to 5 years'}, {'measure': 'Percentage of PTPs and PUPs Developing Antibodies Against CSL627', 'timeFrame': 'PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).'}, {'measure': 'Percentage of PTPs and PUPs Developing Antibodies to Chinese Hamster Ovary (CHO) Proteins', 'timeFrame': 'PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).'}, {'measure': 'Number of PUPs With High-titer Inhibitor Formation to FVIII After 10 EDs With CSL627', 'timeFrame': 'At the closest visit after 10 EDs (up to 5 years)', 'description': 'High-titer inhibitor is defined as an inhibitor titer of ≥ 5 Bethesda units/mL.'}, {'measure': 'Number of PUPs With Low-titer Inhibitor Formation to FVIII After 10 EDs and After 50 EDs With CSL627', 'timeFrame': 'At the closest visit after 10 and after 50 EDs (up to 5 years)', 'description': 'Low-titer inhibitor is defined as an inhibitor titer of less than 5 Bethesda units/mL.'}, {'measure': 'Incidence of Total Inhibitor Formation to FVIII in PUPs', 'timeFrame': 'Up to 5 years'}, {'measure': 'Percent Treatment Success for Non-major Bleeding Episodes in PUPs', 'timeFrame': 'Up to 5 years', 'description': 'Percentage of bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator\'s clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Non-major bleeding episodes are those not requiring treatment at the hemophilia center or not threatening subject\'s life or loss of limb.'}, {'measure': 'Percentage of PUPs With Clinically Significant Abnormal Vital Signs Values After First Infusion of CSL627', 'timeFrame': 'Up to 6 hours after first infusion', 'description': 'Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator.'}, {'measure': 'Percentage of PUPs With Treatment-emergent Clinically Significant Abnormal Vital Signs Values', 'timeFrame': 'Up to 5 years', 'description': 'Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Hemophilia A', 'Severe Hemophilia A']}, 'descriptionModule': {'briefSummary': 'This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII-SingleChain for prophylaxis and on-demand treatment of bleeding episodes in at least 200 previously treated patients (PTPs) with severe congenital hemophilia A and previous exposure to FVIII products who achieve at least 100 exposure days (EDs) to rVIII-SingleChain in this study, as well as in previously untreated patients (PUPs) with no previous exposure to any FVIII product who achieve at least 50 EDs to rVIII-SingleChain in this study. A substudy (open to both PTPs and PUPs) will investigate the use of rVIII-SingleChain in surgery. A substudy (open to PUPs who develop an inhibitor to rVIII-SingleChain) will investigate the use of rVIII-SingleChain in immune tolerance induction (ITI) therapy.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nPTPs:\n\n* Males of any age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels \\< 1%) and who participated in a previous CSL-sponsored clinical study with rVIII-SingleChain.\n* Males 0 to \\<65 years age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels \\< 1%), who have at least 50 EDs to any FVIII product, and who are not currently enrolled in a CSL-sponsored clinical study with rVIII-SingleChain.\n\nPUPs:\n\n* Males 0 to \\<18 years of who have been diagnosed with severe congenital hemophilia A (FVIII activity levels \\< 1%)\n* No prior exposure to any Factor VIII product (with the exception of short-term use of blood products).\n\nITI substudy:\n\n* PUPs who have developed a confirmed inhibitor to rVIII-SingleChain in the main study.\n\nExclusion Criteria:\n\n* Known or suspected hypersensitivity to rVIII-SingleChain or to any excipients of rVIII-SingleChain or Chinese hamster ovary (CHO) proteins.\n* Currently receiving a therapy not permitted during the study.\n* Serum creatinine \\> 2 x upper limit of normal, alanine aminotransferase or aspartate aminotransferase \\> 5 x upper limit of normal at Screening (if specified)\n* Any first-order family (eg, siblings) history of FVIII inhibitors\n* For PTPs not rolling over directly from a CSL-sponsored clinical study with rVIII-SingleChain: any history of or current FVIII inhibitors'}, 'identificationModule': {'nctId': 'NCT02172950', 'briefTitle': 'An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A', 'organization': {'class': 'INDUSTRY', 'fullName': 'CSL Behring'}, 'officialTitle': 'A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects With Severe Hemophilia A', 'orgStudyIdInfo': {'id': 'CSL627_3001'}, 'secondaryIdInfos': [{'id': '2013-003262-13', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Previously treated patients (PTPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.", 'interventionNames': ['Biological: rVIII-SingleChain']}, {'type': 'EXPERIMENTAL', 'label': 'Previously untreated patients (PUPs)', 'description': "The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.", 'interventionNames': ['Biological: rVIII-SingleChain']}], 'interventions': [{'name': 'rVIII-SingleChain', 'type': 'BIOLOGICAL', 'otherNames': ['CSL627'], 'description': 'Recombinant single-chain coagulation factor VIII', 'armGroupLabels': ['Previously treated patients (PTPs)', 'Previously untreated patients (PUPs)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92103-8651', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': 'Study Site 8400213', 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'Study Site 8400241', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '06106', 'city': 'Hartford', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Study Site 8400118', 'geoPoint': {'lat': 41.76371, 'lon': -72.68509}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'Study Site 8400116', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Study Site 8400184', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '70112', 'city': 'New Orleans', 'state': 'Louisiana', 'country': 'United States', 'facility': 'Study Site 8400204', 'geoPoint': {'lat': 29.95465, 'lon': -90.07507}}, {'zip': '75390-9016', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Study Site 8400240', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Study Site 8400041', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Study Site 8400154', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '3052', 'city': 'Melbourne', 'country': 'Australia', 'facility': 'Study Site 0360014', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}, {'zip': 'WA 6009', 'city': 'Nedlands', 'country': 'Australia', 'facility': 'Study Site 0360028', 'geoPoint': {'lat': -31.98184, 'lon': 115.8073}}, {'zip': 'WA 6000', 'city': 'Perth', 'country': 'Australia', 'facility': 'Study Site 0360031', 'geoPoint': {'lat': -31.95224, 'lon': 115.8614}}, {'zip': '8036', 'city': 'Graz', 'country': 'Austria', 'facility': 'Study Site 0400012', 'geoPoint': {'lat': 47.06733, 'lon': 15.44197}}, {'zip': '4020', 'city': 'Linz', 'country': 'Austria', 'facility': 'Study Site 0400003', 'geoPoint': {'lat': 48.30639, 'lon': 14.28611}}, {'zip': '1090', 'city': 'Vienna', 'country': 'Austria', 'facility': 'Study Site 0400001', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'zip': '1090', 'city': 'Vienna', 'country': 'Austria', 'facility': 'Study Site 0400002', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'zip': 'E2L 4L2', 'city': 'Saint John', 'country': 'Canada', 'facility': 'Study Site 1240022', 'geoPoint': {'lat': 45.27076, 'lon': -66.05616}}, {'zip': '500 05', 'city': 'Hradec Králové', 'country': 'Czechia', 'facility': 'Study Site 2030017', 'geoPoint': {'lat': 50.20923, 'lon': 15.83277}}, {'zip': '29609', 'city': 'Brest', 'country': 'France', 'facility': 'Study Site 2500015', 'geoPoint': {'lat': 48.39029, 'lon': -4.48628}}, {'zip': '94270', 'city': 'Le Kremlin-Bicêtre', 'country': 'France', 'facility': 'Study Site 2500017', 'geoPoint': {'lat': 48.81471, 'lon': 2.36073}}, {'zip': '59037', 'city': 'Lille', 'country': 'France', 'facility': 'Study Site 2500028', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'zip': '44093', 'city': 'Nantes', 'country': 'France', 'facility': 'Study Site 2500018', 'geoPoint': {'lat': 47.21725, 'lon': -1.55336}}, {'zip': '75015', 'city': 'Paris', 'country': 'France', 'facility': 'Study Site 2500002', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '0179', 'city': 'Tbilisi', 'country': 'Georgia', 'facility': 'Study Site 2680001', 'geoPoint': {'lat': 41.69143, 'lon': 44.83412}}, {'zip': '53127', 'city': 'Bonn', 'country': 'Germany', 'facility': 'Study Site 2760034', 'geoPoint': {'lat': 50.73438, 'lon': 7.09549}}, {'zip': '60596', 'city': 'Frankfurt', 'country': 'Germany', 'facility': 'Study Site 2760091', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}, {'zip': '35385', 'city': 'Giessen', 'country': 'Germany', 'facility': 'Study Site 2760087', 'geoPoint': {'lat': 50.58727, 'lon': 8.67554}}, {'zip': '30625', 'city': 'Hanover', 'country': 'Germany', 'facility': 'Study Site 2760066', 'geoPoint': {'lat': 52.37052, 'lon': 9.73322}}, {'zip': '4032', 'city': 'Debrecen', 'country': 'Hungary', 'facility': 'Study Site 3480007', 'geoPoint': {'lat': 47.53167, 'lon': 21.62444}}, {'zip': '12', 'city': 'Dublin', 'country': 'Ireland', 'facility': 'Study Site 3720002', 'geoPoint': {'lat': 53.33306, 'lon': -6.24889}}, {'zip': '20122', 'city': 'Milan', 'country': 'Italy', 'facility': 'Study Site 3800023', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '663-8501', 'city': 'Hyōgo', 'country': 'Japan', 'facility': 'Study Site 3920031', 'geoPoint': {'lat': 43.36667, 'lon': 144.43333}}, {'zip': '466-8550', 'city': 'Nagoya', 'country': 'Japan', 'facility': 'Study Site 3920029', 'geoPoint': {'lat': 35.18147, 'lon': 136.90641}}, {'zip': '710-8602', 'city': 'Okayama', 'country': 'Japan', 'facility': 'Study Site 3920064', 'geoPoint': {'lat': 34.65, 'lon': 133.93333}}, {'zip': '339-8551', 'city': 'Saitama', 'country': 'Japan', 'facility': 'Study Site 3920033', 'geoPoint': {'lat': 35.90807, 'lon': 139.65657}}, {'zip': '160-0023', 'city': 'Tokyo', 'country': 'Japan', 'facility': 'Study Site 3920025', 'geoPoint': {'lat': 35.6895, 'lon': 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