Ignite Creation Date:
2025-12-24 @ 3:05 PM
Ignite Modification Date:
2026-01-02 @ 7:05 AM
Study NCT ID:
NCT00914459
Status:
COMPLETED
Last Update Posted:
2017-02-10
First Post:
2009-06-04
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Bulgaria', 'Greece', 'Poland']}, '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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C427184', 'term': 'recombinant factor VIII SQ'}, {'id': 'D005169', 'term': 'Factor VIII'}, {'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}], 'ancestors': [{'id': 'D001779', 'term': 'Blood Coagulation Factors'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011498', 'term': 'Protein Precursors'}, {'id': 'D001685', 'term': 'Biological Factors'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTrials.gov_Inquiries@pfizer.com', 'phone': '1--800--718--1021', 'title': 'Pfizer ClinicalTrials.gov Call Center', 'organization': 'Pfizer, Inc.'}, 'certainAgreement': {'otherDetails': 'Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to 30 days after last study visit (Month 25)', 'description': 'Same event may appear as both an AE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and nonserious in another participant, or one participant may have experienced both serious and nonserious event during study. Adverse events data was planned to be reported for overall population.', 'eventGroups': [{'id': 'EG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.', 'otherNumAtRisk': 37, 'otherNumAffected': 28, 'seriousNumAtRisk': 37, 'seriousNumAffected': 6}], 'otherEvents': [{'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 12}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Adenoiditis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Otitis media acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Cestode infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Infectious mononucleosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Laryngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pyoderma streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Varicella', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Head injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Joint injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Limb injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Tooth fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Arthropod sting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Craniocerebral injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Lip injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Mouth injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 7}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Haemarthrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Groin pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Joint range of motion decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Muscle haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Dental caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Lip haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Mouth haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Oral cavity fistula', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Tooth pulp haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Obstructive airways disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Underweight', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Skin mass', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Solar urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Tooth extraction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}], 'seriousEvents': [{'term': 'Factor VIII inhibition', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 4}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Laryngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Head injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Scrotal disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Acute respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 37, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants With Clinically Significant Factor VIII Inhibitor Development', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: Less Than 6 Years', 'description': 'Participants below 6 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}, {'id': 'OG001', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.00', 'upperLimit': '18.53'}, {'value': '0.00', 'groupId': 'OG001', 'lowerLimit': '0.00', 'upperLimit': '17.65'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Month 24', 'description': 'Clinically significant factor VIII (FVIII) inhibitors were defined as a central laboratory confirmed positive inhibitor of greater than or equal to (\\>=) 0.6 Bethesda units (BU) using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 6-week interval and one of the following within 4 weeks before the initial or within 4 weeks following the second positive FVIII inhibitor sample collection: 1) the need for the participant to administer alternative hemostatic products in order to achieve sufficient efficacy, 2) \\>=2 events indicating a decrease in the efficacy of the study treatment. Percentage of participants who developed clinically significant Factor VIII inhibitor after study drug administration were reported.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety analysis population included all enrolled participants who received at least 1 dose of ReFacto AF.'}, {'type': 'PRIMARY', 'title': 'Incremental Recovery', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: Less Than 6 Years', 'description': 'Participants below 6 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}, {'id': 'OG001', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'title': 'Day 1 (n= 17, 18)', 'categories': [{'measurements': [{'value': '1.67', 'spread': '0.361', 'groupId': 'OG000'}, {'value': '1.97', 'spread': '0.437', 'groupId': 'OG001'}]}]}, {'title': 'Day 15 (n= 18, 17)', 'categories': [{'measurements': [{'value': '1.23', 'spread': '0.650', 'groupId': 'OG000'}, {'value': '1.91', 'spread': '0.423', 'groupId': 'OG001'}]}]}, {'title': 'Day 50 (n= 17, 18)', 'categories': [{'measurements': [{'value': '1.66', 'spread': '0.626', 'groupId': 'OG000'}, {'value': '1.96', 'spread': '0.586', 'groupId': 'OG001'}]}]}, {'title': 'Month 6 (n= 2, 5)', 'categories': [{'measurements': [{'value': '1.69', 'spread': '0.210', 'groupId': 'OG000'}, {'value': '2.17', 'spread': '0.379', 'groupId': 'OG001'}]}]}, {'title': 'Month 18 (n= 4, 5)', 'categories': [{'measurements': [{'value': '1.81', 'spread': '0.405', 'groupId': 'OG000'}, {'value': '1.80', 'spread': '0.493', 'groupId': 'OG001'}]}]}, {'title': 'Final Visit (n= 17, 17)', 'categories': [{'measurements': [{'value': '1.98', 'spread': '1.454', 'groupId': 'OG000'}, {'value': '1.89', 'spread': '0.503', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Days 1, 15, 50, Months 6, 18 and Final visit (up to Month 24)', 'description': 'Incremental recovery was the increase in circulating FVIII activity for every international unit (IU) of ReFacto AF administered per kilogram of body weight. It was measured in international units per deciliter (IU/dL) per international units per kilogram (IU/kg).', 'unitOfMeasure': '(IU/dL)/(IU/kg)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The pharmacokinetic (PK) parameter analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "n" signifies participants who were evaluable at the specified time point for each arm respectively.'}, {'type': 'PRIMARY', 'title': 'Terminal Elimination Half Life of ReFacto AF (t1/2)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '9.12', 'spread': '1.9429', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'T1/2 was the time for the plasma concentration of drug to decrease by one-half of its original concentration.', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK parameter analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure. Data was not planned to be collected and analyzed for reporting arm "ReFacto AF: Less Than 6 Years", as pre-specified in protocol.'}, {'type': 'PRIMARY', 'title': 'Clearance (CL)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '4.406', 'spread': '30', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.', 'unitOfMeasure': 'milliliter per hour per kilogram', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK parameter analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure. Data was not planned to be collected and analyzed for reporting arm "ReFacto AF: Less Than 6 Years", as pre-specified in protocol.'}, {'type': 'SECONDARY', 'title': 'Mean Annualized Bleeding Rates (ABRs): All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged less than or equal to \\[\\<=\\] 12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'title': 'On-demand regimen (n=14)', 'categories': [{'measurements': [{'value': '27.51', 'spread': '20.387', 'groupId': 'OG000'}]}]}, {'title': 'Preventive regimen (n=0)', 'categories': [{'measurements': [{'value': 'NA', 'spread': 'NA', 'comment': 'Data was not calculated and reported because none of the participants were reported at baseline as following a preventive regimen.', 'groupId': 'OG000'}]}]}, {'title': 'Primary or secondary prophylaxis regimen (n=22)', 'categories': [{'measurements': [{'value': '4.18', 'spread': '3.849', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Month 24', 'description': 'ABR for each participant was calculated as the number of bleeds requiring administration of FVIII replacement product (taken from the Infusion Log Diary case report form), divided by the total therapy duration (in days), then multiplied by 365.25. ABR for the participants who reported following a primary or secondary prophylaxis, on-demand regimen or preventive regimen at baseline were reported.', 'unitOfMeasure': 'bleeds per year', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure and "n" signifies participants who were evaluable at the specified time points.'}, {'type': 'SECONDARY', 'title': 'Response to First On-Demand Treatment for New Bleeds: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}, {'units': 'bleeds', 'counts': [{'value': '804', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'title': 'Excellent', 'categories': [{'measurements': [{'value': '713', 'spread': '20.387', 'groupId': 'OG000'}]}]}, {'title': 'Good', 'categories': [{'measurements': [{'value': '73', 'spread': 'NA', 'groupId': 'OG000'}]}]}, {'title': 'Moderate', 'categories': [{'measurements': [{'value': '16', 'spread': '3.849', 'groupId': 'OG000'}]}]}, {'title': 'Data Not Recorded', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Month 24', 'description': "A 4-point scale of assessment of 'on-demand' treatment (administration of an unscheduled bolus infusion of Refacto-AF to stop bleeding) is defined as: 1. Excellent: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered. 2. Good: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least one additional infusion administered for complete resolution of the bleeding episode;or, Definite pain relief and/or improvement in signs of bleeding starting after 8 hours following infusion, with no additional infusion administered. 3. Moderate: Probable or slight improvement starting after 8 hours following the infusion, with at least one additional infusion administered for complete resolution of the bleeding episode. 4. No Response: No improvement at all between infusions or during the 24-hour interval following an infusion, or condition worsens.", 'unitOfMeasure': 'responses', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'bleeds', 'denomUnitsSelected': 'bleeds', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure and received at least 1 dose of ReFacto AF for at least one bleeding episode.'}, {'type': 'SECONDARY', 'title': 'Number of On-Demand ReFacto AF Infusions to Treat a New Bleed: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}, {'units': 'bleeds', 'counts': [{'value': '804', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.1', 'spread': '0.55', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Month 24', 'description': 'The infusion log diary case report form (CRF) was used to determine the number of on-demand (administration of an unscheduled bolus infusion of Refacto-AF to stop bleeding) ReFacto AF infusions administered to treat a new bleed. This was calculated by adding the initial for a new bleed (on-demand) infusion to any subsequent (on-demand) infusions for the same "previously treated bleed" (same bleed with same start date/time).', 'unitOfMeasure': 'infusions', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'bleeds', 'denomUnitsSelected': 'bleeds', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Number of Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of ReFacto AF: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.0', 'spread': '1.15', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Month 24', 'description': 'The number of breakthrough bleeds within 48 hours following a prophylaxis dose of ReFacto AF was summarized. The infusion log diary CRF was used to determine the number of infusions administered to treat a new bleed counting only those infusions which were administered \\<=48 hours after an infusion marked as "prophylaxis" (which had no associated bleed).', 'unitOfMeasure': 'breakthrough bleeds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF.'}, {'type': 'SECONDARY', 'title': 'Average Infusion Dose of ReFacto AF: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'title': 'With prophylaxis regimen at baseline (n= 22)', 'categories': [{'measurements': [{'value': '37.0', 'spread': '8.70', 'groupId': 'OG000'}]}]}, {'title': 'With non-prophylaxis regimen at baseline (n= 15)', 'categories': [{'measurements': [{'value': '29.5', 'spread': '7.61', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Month 24', 'description': 'The average infusion dose (by weight) for each participant was calculated as his total factor FVIII consumption (in IU) divided by weight (in kg) divided by the number of infusions administered in total study duration. Data was reported separately for participants classified at baseline as following non-prophylaxis regimen (for example: on-demand regimen, preventive, or not specified), and participants classified at baseline following a primary or secondary prophylaxis regimen.', 'unitOfMeasure': 'IU/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "n" signifies participants who were evaluable for each specified baseline category.'}, {'type': 'SECONDARY', 'title': 'Total Factor VIII Consumption: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'title': 'With Prophylaxis regimen at baseline (n= 22)', 'categories': [{'measurements': [{'value': '97959.4', 'spread': '48474.09', 'groupId': 'OG000'}]}]}, {'title': 'With non-prophylaxis regimen at baseline (n= 15)', 'categories': [{'measurements': [{'value': '84051.7', 'spread': '47362.60', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Month 24', 'description': 'Total factor VIII consumption for each participant was calculated by sum of the total amount of ReFacto AF (in IU) infused for each ReFacto AF infusion (recorded in the infusion log diary CRF). Data was reported separately for participants classified at baseline as following non-prophylaxis regimen (for example: on-demand regimen, preventive, or not specified), and participants classified at baseline following a primary or secondary prophylaxis regimen.', 'unitOfMeasure': 'IU', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "n" signifies participants who were evaluable for each specified baseline category.'}, {'type': 'SECONDARY', 'title': 'Number of Less-Than-Expected-Therapeutic Effect (LETE) Bleeds in the On-Demand Setting: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}, {'units': 'bleeding episodes', 'counts': [{'value': '804', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Month 24', 'description': 'LETE in on-demand setting was based on response to treatment of a bleeding episode. LETE in the on-demand setting occurred if participant recorded 2 successive "no response" ratings after 2 successive ReFacto AF infusions. Both infusions were to be administered at an interval of 24 hours for treatment of same bleeding event in absence of confounding factor which included: known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than 4 hours between onset of bleed to infusion, delay of greater than 24 hours before administration of a follow-up infusion, known compromised ReFacto AF, faulty administration of ReFacto AF, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator (e.g., kidney stones or use of medications known to impair platelet function, such as aspirin or NSAIDs),or ongoing trauma responsible for continued bleeding.', 'unitOfMeasure': 'LETE bleeds', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'bleeding episodes', 'denomUnitsSelected': 'bleeding episodes', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure and received treatment for at least one bleed.'}, {'type': 'SECONDARY', 'title': 'Number of Less-Than-Expected-Therapeutic Effect (LETE) Bleeds in the Prophylaxis Setting: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}, {'units': 'prophylaxis infusions', 'counts': [{'value': '2457', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Month 24', 'description': 'LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (\\<=48 hours) after a regularly scheduled prophylactic dose of ReFacto AF (which was not used to treat a bleed) in the absence of confounding factors. Therefore, LETE in the prophylaxis setting is the occurrence of a bleed. Confounding factors include: Known presence or subsequent identification of a FVIII inhibitor, known inadequate prophylactic dose, known lack of adherence to the prescribed prophylaxis regimen, bleed occurs in a target joint identified at the start of the study, known compromised ReFacto AF, faulty administration of ReFacto AF, an underlying, predisposing condition responsible for the bleed in the opinion of the investigator (e.g., kidney stones or use of medications known to impair platelet function, such as aspirin or NSAIDs) or traumatic injury responsible for bleeding.', 'unitOfMeasure': 'LETE bleeds', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'prophylaxis infusions', 'denomUnitsSelected': 'prophylaxis infusions', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Participants who received at least 1 prophylaxis dose of ReFacto AF were reported.'}, {'type': 'SECONDARY', 'title': 'Number of Occurrences of Less-Than-Expected-Therapeutic Effect (LETE) in the Low Recovery Setting: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Month 24', 'description': 'LETE in the low recovery setting was defined as lower than expected recovery of FVIII (in the opinion of investigator), following the infusion of ReFacto AF in the absence of confounding factors for the low recovery. The only confounding factors for low recovery are as follows: known presence or subsequent identification of a FVIII inhibitor, known compromised ReFacto AF, faulty administration of ReFacto AF, including inadequate dosing.', 'unitOfMeasure': 'LETE bleeds', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF.'}, {'type': 'SECONDARY', 'title': 'Number of Participants Requiring Escalated Dose of Prescribed Regimen During the Treatment Period: All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Month 24', 'description': "Participants who met the dose escalation criteria were prescribed a higher dose and/or more frequent doses as per the investigator's discretion.", 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Participants who used a prophylaxis regimen were analyzed for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Plasma Concentration of Factor VIII at 0.5 Hour Post-dose (C0.5)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: Less Than 6 Years', 'description': 'Participants below 6 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}, {'id': 'OG001', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.752', 'spread': '18', 'groupId': 'OG000'}, {'value': '0.903', 'spread': '45', 'groupId': 'OG001'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': '0.5 hour post-dose on Day 1', 'unitOfMeasure': 'IU/mL', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK parameter analysis population included all enrolled participants who received at least 1 dose of ReFacto AF.'}, {'type': 'SECONDARY', 'title': 'Area Under the Plasma Time Curve From Time 0 Extrapolated to Infinite Time (AUCinf)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '9.89', 'spread': '41', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'AUCinf is the area under the plasma concentration-time profile from time 0 extrapolated to infinite time. It was calculated as International units\\*hour per milliliter (IU\\*hr/mL).', 'unitOfMeasure': 'IU*hr/mL', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK parameter analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure. Data was not planned to be collected and analyzed for reporting arm "ReFacto AF: Less Than 6 Years", as pre-specified in protocol.'}, {'type': 'SECONDARY', 'title': 'Area Under the Plasma Time Curve From Time Zero to Time of Last Measurable Concentration (AUClast)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '9.49', 'spread': '41', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'AUClast is the area under the plasma versus time curve from time zero to time of last measurable concentration (AUClast)', 'unitOfMeasure': 'IU*hr/mL', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK parameter analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure. Data was not planned to be collected and analyzed for reporting arm "ReFacto AF: Less Than 6 Years", as pre-specified in protocol.'}, {'type': 'SECONDARY', 'title': 'Volume of Distribution at Steady State (Vss)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '56.42', 'spread': '15', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'Volume of distribution was defined as the theoretical volume in which the total amount of drug was uniformly distributed to produce the desired blood concentration of a drug. Steady state volume of distribution (Vss) was the apparent volume of distribution at steady-state.', 'unitOfMeasure': 'mL/kg', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK parameter analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure. Data was not planned to be collected and analyzed for reporting arm "ReFacto AF: Less Than 6 Years", as pre-specified in protocol.'}, {'type': 'SECONDARY', 'title': 'Mean Residence Time (MRT) of ReFacto AF', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'categories': [{'measurements': [{'value': '13.91', 'groupId': 'OG000', 'lowerLimit': '8.51', 'upperLimit': '18.3'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'MRT was calculated as AUMCinf / AUCinf-TI/2, where AUMCinf is the area under the first moment curve from time zero to infinity and TI was the duration of infusion.', 'unitOfMeasure': 'hour', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'PK parameter analysis population included all enrolled participants who received at least 1 dose of ReFacto AF. Here, "number of participants analyzed" signifies participants who were evaluable for this outcome measure. Data was not planned to be collected and analyzed for reporting arm "ReFacto AF: Less Than 6 Years", as pre-specified in protocol.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs): All Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ReFacto AF: All Participants', 'description': 'All participants (aged \\<=12 years of age) were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'classes': [{'title': 'AEs', 'categories': [{'measurements': [{'value': '28', 'groupId': 'OG000'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to 30 days after last study visit (Month 25)', 'description': 'An adverse event (AE) was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), persistent or significant disability or incapacity, congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety analysis population included all enrolled participants who received at least 1 dose of ReFacto AF.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'ReFacto AF: Less Than 6 Years', 'description': 'Participants below 6 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 international units per kilogram \\[IU/kg\\] up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the Summary of Product Characteristics (SmPC).'}, {'id': 'FG001', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}, {'groupId': 'FG001', 'numSubjects': '19'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '17'}, {'groupId': 'FG001', 'numSubjects': '18'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Parent/Legal guardian request', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'BG000'}, {'value': '19', 'groupId': 'BG001'}, {'value': '37', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'ReFacto AF: Less Than 6 Years', 'description': 'Participants below 6 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}, {'id': 'BG001', 'title': 'ReFacto AF: 6 to Less Than 12 Years', 'description': 'Participants of 6 to 12 years of age were treated with IV injections of ReFacto AF at a dose and frequency prescribed by the investigator (minimum dose of 17 IU/kg up to maximum dose of 51 IU/kg) as per local standard of care in accordance with the SmPC.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '3.6', 'spread': '1.42', 'groupId': 'BG000'}, {'value': '9.2', 'spread': '1.47', 'groupId': 'BG001'}, {'value': '6.5', 'spread': '3.20', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Gender', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '18', 'groupId': 'BG000'}, {'value': '19', 'groupId': 'BG001'}, {'value': '37', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'All treated analysis set included all enrolled participants who received at least 1 dose of ReFacto AF.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 37}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-12', 'completionDateStruct': {'date': '2016-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-12-21', 'studyFirstSubmitDate': '2009-06-04', 'resultsFirstSubmitDate': '2016-08-08', 'studyFirstSubmitQcDate': '2009-06-04', 'lastUpdatePostDateStruct': {'date': '2017-02-10', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2016-10-28', 'studyFirstPostDateStruct': {'date': '2009-06-05', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2016-12-21', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants With Clinically Significant Factor VIII Inhibitor Development', 'timeFrame': 'Baseline up to Month 24', 'description': 'Clinically significant factor VIII (FVIII) inhibitors were defined as a central laboratory confirmed positive inhibitor of greater than or equal to (\\>=) 0.6 Bethesda units (BU) using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 6-week interval and one of the following within 4 weeks before the initial or within 4 weeks following the second positive FVIII inhibitor sample collection: 1) the need for the participant to administer alternative hemostatic products in order to achieve sufficient efficacy, 2) \\>=2 events indicating a decrease in the efficacy of the study treatment. Percentage of participants who developed clinically significant Factor VIII inhibitor after study drug administration were reported.'}, {'measure': 'Incremental Recovery', 'timeFrame': 'Days 1, 15, 50, Months 6, 18 and Final visit (up to Month 24)', 'description': 'Incremental recovery was the increase in circulating FVIII activity for every international unit (IU) of ReFacto AF administered per kilogram of body weight. It was measured in international units per deciliter (IU/dL) per international units per kilogram (IU/kg).'}, {'measure': 'Terminal Elimination Half Life of ReFacto AF (t1/2)', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'T1/2 was the time for the plasma concentration of drug to decrease by one-half of its original concentration.'}, {'measure': 'Clearance (CL)', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.'}], 'secondaryOutcomes': [{'measure': 'Mean Annualized Bleeding Rates (ABRs): All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': 'ABR for each participant was calculated as the number of bleeds requiring administration of FVIII replacement product (taken from the Infusion Log Diary case report form), divided by the total therapy duration (in days), then multiplied by 365.25. ABR for the participants who reported following a primary or secondary prophylaxis, on-demand regimen or preventive regimen at baseline were reported.'}, {'measure': 'Response to First On-Demand Treatment for New Bleeds: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': "A 4-point scale of assessment of 'on-demand' treatment (administration of an unscheduled bolus infusion of Refacto-AF to stop bleeding) is defined as: 1. Excellent: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered. 2. Good: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least one additional infusion administered for complete resolution of the bleeding episode;or, Definite pain relief and/or improvement in signs of bleeding starting after 8 hours following infusion, with no additional infusion administered. 3. Moderate: Probable or slight improvement starting after 8 hours following the infusion, with at least one additional infusion administered for complete resolution of the bleeding episode. 4. No Response: No improvement at all between infusions or during the 24-hour interval following an infusion, or condition worsens."}, {'measure': 'Number of On-Demand ReFacto AF Infusions to Treat a New Bleed: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': 'The infusion log diary case report form (CRF) was used to determine the number of on-demand (administration of an unscheduled bolus infusion of Refacto-AF to stop bleeding) ReFacto AF infusions administered to treat a new bleed. This was calculated by adding the initial for a new bleed (on-demand) infusion to any subsequent (on-demand) infusions for the same "previously treated bleed" (same bleed with same start date/time).'}, {'measure': 'Number of Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of ReFacto AF: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': 'The number of breakthrough bleeds within 48 hours following a prophylaxis dose of ReFacto AF was summarized. The infusion log diary CRF was used to determine the number of infusions administered to treat a new bleed counting only those infusions which were administered \\<=48 hours after an infusion marked as "prophylaxis" (which had no associated bleed).'}, {'measure': 'Average Infusion Dose of ReFacto AF: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': 'The average infusion dose (by weight) for each participant was calculated as his total factor FVIII consumption (in IU) divided by weight (in kg) divided by the number of infusions administered in total study duration. Data was reported separately for participants classified at baseline as following non-prophylaxis regimen (for example: on-demand regimen, preventive, or not specified), and participants classified at baseline following a primary or secondary prophylaxis regimen.'}, {'measure': 'Total Factor VIII Consumption: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': 'Total factor VIII consumption for each participant was calculated by sum of the total amount of ReFacto AF (in IU) infused for each ReFacto AF infusion (recorded in the infusion log diary CRF). Data was reported separately for participants classified at baseline as following non-prophylaxis regimen (for example: on-demand regimen, preventive, or not specified), and participants classified at baseline following a primary or secondary prophylaxis regimen.'}, {'measure': 'Number of Less-Than-Expected-Therapeutic Effect (LETE) Bleeds in the On-Demand Setting: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': 'LETE in on-demand setting was based on response to treatment of a bleeding episode. LETE in the on-demand setting occurred if participant recorded 2 successive "no response" ratings after 2 successive ReFacto AF infusions. Both infusions were to be administered at an interval of 24 hours for treatment of same bleeding event in absence of confounding factor which included: known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than 4 hours between onset of bleed to infusion, delay of greater than 24 hours before administration of a follow-up infusion, known compromised ReFacto AF, faulty administration of ReFacto AF, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator (e.g., kidney stones or use of medications known to impair platelet function, such as aspirin or NSAIDs),or ongoing trauma responsible for continued bleeding.'}, {'measure': 'Number of Less-Than-Expected-Therapeutic Effect (LETE) Bleeds in the Prophylaxis Setting: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': 'LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (\\<=48 hours) after a regularly scheduled prophylactic dose of ReFacto AF (which was not used to treat a bleed) in the absence of confounding factors. Therefore, LETE in the prophylaxis setting is the occurrence of a bleed. Confounding factors include: Known presence or subsequent identification of a FVIII inhibitor, known inadequate prophylactic dose, known lack of adherence to the prescribed prophylaxis regimen, bleed occurs in a target joint identified at the start of the study, known compromised ReFacto AF, faulty administration of ReFacto AF, an underlying, predisposing condition responsible for the bleed in the opinion of the investigator (e.g., kidney stones or use of medications known to impair platelet function, such as aspirin or NSAIDs) or traumatic injury responsible for bleeding.'}, {'measure': 'Number of Occurrences of Less-Than-Expected-Therapeutic Effect (LETE) in the Low Recovery Setting: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': 'LETE in the low recovery setting was defined as lower than expected recovery of FVIII (in the opinion of investigator), following the infusion of ReFacto AF in the absence of confounding factors for the low recovery. The only confounding factors for low recovery are as follows: known presence or subsequent identification of a FVIII inhibitor, known compromised ReFacto AF, faulty administration of ReFacto AF, including inadequate dosing.'}, {'measure': 'Number of Participants Requiring Escalated Dose of Prescribed Regimen During the Treatment Period: All Participants', 'timeFrame': 'Baseline up to Month 24', 'description': "Participants who met the dose escalation criteria were prescribed a higher dose and/or more frequent doses as per the investigator's discretion."}, {'measure': 'Plasma Concentration of Factor VIII at 0.5 Hour Post-dose (C0.5)', 'timeFrame': '0.5 hour post-dose on Day 1'}, {'measure': 'Area Under the Plasma Time Curve From Time 0 Extrapolated to Infinite Time (AUCinf)', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'AUCinf is the area under the plasma concentration-time profile from time 0 extrapolated to infinite time. It was calculated as International units\\*hour per milliliter (IU\\*hr/mL).'}, {'measure': 'Area Under the Plasma Time Curve From Time Zero to Time of Last Measurable Concentration (AUClast)', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'AUClast is the area under the plasma versus time curve from time zero to time of last measurable concentration (AUClast)'}, {'measure': 'Volume of Distribution at Steady State (Vss)', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'Volume of distribution was defined as the theoretical volume in which the total amount of drug was uniformly distributed to produce the desired blood concentration of a drug. Steady state volume of distribution (Vss) was the apparent volume of distribution at steady-state.'}, {'measure': 'Mean Residence Time (MRT) of ReFacto AF', 'timeFrame': 'Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1', 'description': 'MRT was calculated as AUMCinf / AUCinf-TI/2, where AUMCinf is the area under the first moment curve from time zero to infinity and TI was the duration of infusion.'}, {'measure': 'Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs): All Participants', 'timeFrame': 'Baseline up to 30 days after last study visit (Month 25)', 'description': 'An adverse event (AE) was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), persistent or significant disability or incapacity, congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 30 days after last dose that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['hemophilia A'], 'conditions': ['Hemophilia A']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=3082B2-4433&StudyName=Study%20Evaluating%20Safety%20And%20Efficacy%20Of%20Moroctocog%20Alfa%20%28AF-CC%29%20In%20Previously%20Treated%20Hemophilia%20A%20Patients', 'label': 'To obtain contact information for a study center near you, click here.'}]}, 'descriptionModule': {'briefSummary': 'The study will be investigating pharmacokinetics, safety and efficacy in patients less than 12 years of age with severe hemophilia A that have been previously treated with Factor VIII products ( including blood products).'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male subjects less than 12 years of age with a documented history of severe hemophilia A (FVIII:C less than 1%).\n* Subjects who are less than 6 years of age must have had at least 50 Exposure Days (EDs) to prior FVIII products (including blood products).\n* Subjects who are equal to or greater than 6 years of age must have had greater than 150 EDs to prior FVIII products (including blood products).\n\nExclusion Criteria:\n\n* For laboratory assessments, any measured Bethesda inhibitor titer equal to or greater than 0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer greater than ULN for the testing laboratory at the time of screening.\n* Any other bleeding disorder in addition to hemophilia A.\n* Treatment with any investigational drug or device within 30 days before the time of signing the parental informed consent/assent form.\n* Major surgery planned to occur during the course of the study.\n* Regular (e.g., daily; every other day) use of agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDS).\n* Regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin \\[IVIG\\], routine systemic corticosteroids), or currently receiving immune tolerance induction (ITI) for inhibitor treatment.\n* The subject is receiving treatment for HIV or hepatitis infection (unless the subject is on a stable antiviral regimen \\[i.e., consistent treatment regimen for at least 3 months before the parental informed consent/assent form is signed\\]).\n* Platelet count less than 100,000/µL.\n* Prothrombin time (PT) equal to or greater than 1.25 x ULN, or international normalized ratio (INR) equal to or greater than 1.5.\n* Known hypersensitivity to hamster protein.'}, 'identificationModule': {'nctId': 'NCT00914459', 'briefTitle': 'Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pfizer'}, 'officialTitle': 'A Non-randomized, Open-label Study To Evaluate The Pharmacokinetics, Safety And Efficacy Of Refacto Af In Previously Treated Pediatric Subjects Less Than Twelve Years Of Age With Severe Hemophilia A (Fviii:c <1%).', 'orgStudyIdInfo': {'id': '3082B2-4433'}, 'secondaryIdInfos': [{'id': 'B1831005', 'type': 'OTHER', 'domain': 'Alias Study Number'}, {'id': '2008-008435-29', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Moroctocog alfa (AF-CC)', 'description': 'Open Label', 'interventionNames': ['Biological: Moroctocog alfa ( AF-CC)', 'Procedure: Laboratory tests']}], 'interventions': [{'name': 'Moroctocog alfa ( AF-CC)', 'type': 'BIOLOGICAL', 'otherNames': ['ReFacto AF'], 'description': 'Dosing is at the discretion of the Investigator', 'armGroupLabels': ['Moroctocog alfa (AF-CC)']}, {'name': 'Laboratory tests', 'type': 'PROCEDURE', 'description': 'Factor VIII PK samples, Hematology, Chemistry and Coagulation testing, FactorVIII Inhibitor and Anti Factor VIII antibody', 'armGroupLabels': ['Moroctocog alfa (AF-CC)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '70211', 'city': 'Kuopio', 'country': 'Finland', 'facility': 'Kuopio University Hospital', 'geoPoint': {'lat': 62.89238, 'lon': 27.67703}}, {'zip': '0186', 'city': 'Tbilisi', 'country': 'Georgia', 'facility': 'LTD Medinvesti- Institute of hematology and transfusiology', 'geoPoint': {'lat': 41.69143, 'lon': 44.83412}}, {'zip': '43100', 'city': 'Parma', 'country': 'Italy', 'facility': "Centro di Riferimento Regionale per la cura dell'Emofilia e delle Malattie Emorragiche Congenite", 'geoPoint': {'lat': 44.79935, 'lon': 10.32618}}, {'zip': '200642', 'city': 'Craiova', 'state': 'Dolj', 'country': 'Romania', 'facility': 'Spitalul Clinic Judetean de Urgenta Craiova', 'geoPoint': {'lat': 44.31667, 'lon': 23.8}}, {'zip': '011026', 'city': 'Bucharest', 'country': 'Romania', 'facility': 'Sanador', 'geoPoint': {'lat': 44.43225, 'lon': 26.10626}}, {'zip': '11000', 'city': 'Belgrade', 'country': 'Serbia', 'facility': "University Children's Hospital", 'geoPoint': {'lat': 44.80401, 'lon': 20.46513}}, {'zip': '11070', 'city': 'Belgrade', 'country': 'Serbia', 'facility': 'Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"', 'geoPoint': {'lat': 44.80401, 'lon': 20.46513}}, {'zip': '11407', 'city': 'Jerez de la Frontera', 'state': 'Cádiz', 'country': 'Spain', 'facility': 'Hospital Jerez de la Frontera', 'geoPoint': {'lat': 36.68645, 'lon': -6.13606}}, {'zip': '11009', 'city': 'Cadiz', 'state': 'Spain', 'country': 'Spain', 'facility': 'Hospital Universitario Puerta del Mar', 'geoPoint': {'lat': 36.52672, 'lon': -6.2891}}, {'zip': '50009', 'city': 'Zaragoza', 'state': 'Zaragoza', 'country': 'Spain', 'facility': 'Hospital Universitario Miguel Servet', 'geoPoint': {'lat': 41.65606, 'lon': -0.87734}}, {'zip': '28046', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital La Paz', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '171 76', 'city': 'Stockholm', 'country': 'Sweden', 'facility': 'Karolinska Universitetssjukhuset-Solna', 'geoPoint': {'lat': 59.32938, 'lon': 18.06871}}, {'zip': '01330', 'city': 'Adana', 'state': 'Balcali/adana', 'country': 'Turkey (Türkiye)', 'facility': 'Cukurova University Department of Pediatrics, Pediatric Hematology Division', 'geoPoint': {'lat': 36.98615, 'lon': 35.32531}}, {'zip': '35100', 'city': 'Izmir', 'state': 'Bornova /izmir', 'country': 'Turkey (Türkiye)', 'facility': 'Ege University Department of Pediatrics, Pediatric Hematology Division', 'geoPoint': {'lat': 38.41273, 'lon': 27.13838}}, {'zip': '07059', 'city': 'Antalya', 'state': 'Kampus', 'country': 'Turkey (Türkiye)', 'facility': 'Akdeniz Universitesi Tip Fakultesi', 'geoPoint': {'lat': 36.90812, 'lon': 30.69556}}, {'zip': '69063', 'city': 'Zaporizhzhia', 'state': 'Ukraine', 'country': 'Ukraine', 'facility': 'Komunalna ustanova "Zaporizka oblasna klinichna dytiacha likarnia"', 'geoPoint': {'lat': 47.15214, 'lon': 35.74246}}, {'zip': '79044', 'city': 'Lviv', 'country': 'Ukraine', 'facility': 'Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii medych', 'geoPoint': {'lat': 49.83826, 'lon': 24.02324}}], 'overallOfficials': [{'name': 'Pfizer CT.gov Call Center', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Pfizer'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pfizer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}