Ignite Creation Date:
2025-12-25 @ 1:43 AM
Ignite Modification Date:
2025-12-27 @ 8:15 AM
Study NCT ID:
NCT01178294
Status:
COMPLETED
Last Update Posted:
2021-05-13
First Post:
2010-08-06
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['France', 'Germany', 'Hungary', 'Italy', 'Sweden']}, 'conditionBrowseModule': {'meshes': [{'id': 'C536392', 'term': 'Factor 8 deficiency, acquired'}, {'id': 'D006467', 'term': 'Hemophilia A'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTransparency@shire.com', 'phone': '+1 866 842 5335', 'title': 'Study Director', 'organization': 'Shire'}, 'certainAgreement': {'otherDetails': 'For this study, PIs are restricted from independently publishing results before completion of a single multicenter publication or one year after study completion, whichever occurs first. Baxter requires a review of results communications (eg, for confidential information) ≥30 days prior to submission or communication. Baxter may request an additional delay of ≤60 days (eg, if a patentable invention is disclosed).', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Due to the low number of subjects available for evaluation, statistical tests could only be performed for the primary outcome measure. The results of all other outcome measures are descriptive.'}}, 'adverseEventsModule': {'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose', 'description': 'Subjects were monitored for adverse events from the time the subject presented with the initial bleeding episode until the end of the follow-up period.', 'eventGroups': [{'id': 'EG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)', 'otherNumAtRisk': 29, 'otherNumAffected': 27, 'seriousNumAtRisk': 29, 'seriousNumAffected': 13}], 'otherEvents': [{'term': 'Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Cardiac failure congestive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 12, 'numAffected': 12}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Mouth ulceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Peripheral edema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Bacteremia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Bacteriuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Antibody test positive', 'notes': 'Positive OBI-1 (anti-porcine FVIII) inhibitor test result', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Troponin I increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Hypoglycemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Hypokalemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 11, 'numAffected': 7}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Hypomagnesemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Hypophosphatemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Muscle hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Ecchymosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}], 'seriousEvents': [{'term': 'Asthenia', 'notes': 'Generalized weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Hematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Atrial fibrillation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Brain edema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Intracranial hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Transient ischemic attack', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Esophagitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Renal failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Systemic mycosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Gastrointestinal hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Cholangitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Anaphylactic reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Hemarthrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Intestinal hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Tracheostomy malfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Vascular pseudoaneurysm', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Grand mal convulsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 29, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Serious Bleeding Episodes Responsive to OBI-1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}, {'units': 'Initial Serious Bleeding Episodes', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000', 'lowerLimit': '88.1', 'upperLimit': '100'}]}]}], 'analyses': [{'pValue': '<0.001', 'groupIds': ['OG000'], 'ciNumSides': 'TWO_SIDED', 'groupDescription': 'Summary statistics for the percentage of participants with serious bleeding episodes responsive at 24 hours after the initiation of treatment are presented, along with the 95% confidence interval (two-sided 95% Clopper-Pearson confidence interval).', 'statisticalMethod': 'one-sided binomial exact test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'NUMBER', 'timeFrame': '24 hours after initiation of treatment', 'description': 'The initial serious ("qualifying") bleeding episode for each subject was analyzed for the primary efficacy outcome measure. A \'positive response\' is defined as \'effective\' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or \'partially effective\' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). \'Poorly effective\' is defined as \'bleeding slightly reduced or unchanged and FVIII levels of less than 50%\'. \'Not effective\' is defined as \'bleeding worsening and FVIII levels of less than 50%\'.', 'unitOfMeasure': 'percentage of serious bleeding episodes', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Initial Serious Bleeding Episodes', 'denomUnitsSelected': 'Initial Serious Bleeding Episodes', 'populationDescription': 'Intent to Treat (ITT) population = 29 subjects with initial serious bleeding episodes'}, {'type': 'SECONDARY', 'title': 'Overall Percentage of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}, {'units': 'Initial Serious Bleeding Episodes', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '86.2', 'groupId': 'OG000', 'lowerLimit': '68.3', 'upperLimit': '96.1'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At the time of final treatment dosing (varied from participant to participant depending on bleeding episodes)', 'description': "Treatment success was defined as control of qualifying bleeding episode at the time of final treatment dosing. A serious bleeding episode was considered 'successfully controlled' if the investigator had checked 'completed OBI-1 therapy as treatment success' on the eCRF.", 'unitOfMeasure': 'percentage of serious bleeding episodes', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Initial Serious Bleeding Episodes', 'denomUnitsSelected': 'Initial Serious Bleeding Episodes', 'populationDescription': 'ITT population = 29 subjects with initial serious bleeding episodes (BEs)'}, {'type': 'SECONDARY', 'title': 'Percentage of Serious Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}, {'units': 'Responses Available at 8 hrs', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '95.2', 'groupId': 'OG000', 'lowerLimit': '76.2', 'upperLimit': '99.9'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '8 hours', 'description': "A 'positive response' is defined as 'effective' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' is defined as 'bleeding slightly reduced or unchanged and FVIII levels of less than 50%'. 'Not effective' is defined as 'bleeding worsening and FVIII levels of less than 50%'.", 'unitOfMeasure': 'percentage of serious bleeding episodes', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Responses Available at 8 hrs', 'denomUnitsSelected': 'Responses Available at 8 hrs', 'populationDescription': '21 subjects of the ITT population (n=29) had responses available at 8 hours after initial infusion of OBI-1.'}, {'type': 'SECONDARY', 'title': 'Percentage of Serious Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}, {'units': 'Responses Available at 16 hrs', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000', 'lowerLimit': '82.4', 'upperLimit': '100'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '16 hours', 'description': "A 'positive response' is defined as 'effective' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' is defined as 'bleeding slightly reduced or unchanged and FVIII levels of less than 50%'. 'Not effective' is defined as 'bleeding worsening and FVIII levels of less than 50%'.", 'unitOfMeasure': 'percentage of serious bleeding episodes', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Responses Available at 16 hrs', 'denomUnitsSelected': 'Responses Available at 16 hrs', 'populationDescription': '19 subjects of the ITT population (n=29) had responses available at 16 hours after initial infusion of OBI-1.'}, {'type': 'SECONDARY', 'title': 'Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '2.10', 'spread': '1.109', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Time of successful control of qualifying bleeding episode (varied from participant to participant)', 'description': "'Frequency of infusions' was calculated as the 'average number of infusions per day'. 'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'.", 'unitOfMeasure': 'average number of infusions per day', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "The analysis was performed in 25 of 29 participants in the ITT population whose 'qualifying' bleeding episode was controlled successfully."}, {'type': 'SECONDARY', 'title': "Total Dose of OBI-1 Required to Successfully Control 'Qualifying' Bleeding Episodes", 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '2683.2', 'spread': '2928.61', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Time of successful control of qualifying bleeding episode (varied from participant to participant)', 'description': "'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'.", 'unitOfMeasure': 'dose in U/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "The analysis was performed in 25 of 29 participants in the ITT population whose 'qualifying' bleeding episode was controlled successfully."}, {'type': 'SECONDARY', 'title': "Total Number of Infusions of OBI-1 Required to Successfully Control 'Qualifying' Bleeding Episodes", 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '15.4', 'spread': '12.64', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Time of successful control of qualifying bleeding episode (varied from participant to participant)', 'description': "'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'. A serious bleeding episode was considered 'successfully controlled' if the investigator had checked 'completed OBI-1 therapy as treatment success' on the eCRF.", 'unitOfMeasure': 'infusions per participant', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "The analysis was performed in 25 of 29 participants in the ITT population whose 'qualifying' bleeding episode was controlled successfully."}, {'type': 'SECONDARY', 'title': 'Correlation Between Positive Response to OBI-1 Therapy at 8 Hours and Eventual Control of Serious Bleeding Episodes at 24 Hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '24 hours', 'unitOfMeasure': 'subjects with eventual bleed control', 'reportingStatus': 'POSTED', 'populationDescription': 'Of 21 subjects in the ITT population (n=29) with responses available at 8 hours after initial infusion of OBI-1, 20 had a positive response.'}, {'type': 'SECONDARY', 'title': 'Correlation Between Response to OBI-1 Therapy at 16 Hours and Eventual Control of Serious Bleeding Episodes at 24 Hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '24 hours', 'unitOfMeasure': 'subjects with eventual bleed control', 'reportingStatus': 'POSTED', 'populationDescription': 'All 19 subjects in the ITT population (n=29) who had responses available at 16 hours after initial infusion of OBI-1 had a positive response.'}, {'type': 'SECONDARY', 'title': 'Correlation Between Response to OBI-1 Therapy at Specified Time Points and Eventual Control of Serious Bleeding Episodes at 24 Hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '25', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '24 hours', 'unitOfMeasure': 'participants with bleeds controlled', 'reportingStatus': 'POSTED', 'populationDescription': '29 subjects (ITT population) had responses available at 24 hours after initial infusion of OBI-1.'}, {'type': 'SECONDARY', 'title': 'Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers, the Total Dose of OBI-1, the Outcome at 24 Hours and the Eventual Control of the Bleeding Episode', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose', 'reportingStatus': 'POSTED', 'populationDescription': 'Because of expected sparseness of positive anti-OBI-1 antibody titers, formal statistical analyses of correlation were not performed.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK) Analysis- Plasma Clearance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'title': 'Chromogenic FVIII activity assay', 'categories': [{'measurements': [{'value': '11.80', 'spread': '13.44', 'groupId': 'OG000'}]}]}, {'title': 'One-stage FVIII activity assay', 'categories': [{'measurements': [{'value': '18.07', 'spread': '21.78', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours', 'description': 'Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics.', 'unitOfMeasure': 'U/(percent activity*hours)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK population (= all subjects in the ITT population who consent to PK draws and have factor VIII levels measured at the central reference laboratory)'}, {'type': 'SECONDARY', 'title': 'PK Analysis- Volume of Distribution (Vd) at Steady State', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'title': 'Chromogenic FVIII activity assay', 'categories': [{'measurements': [{'value': '53.8', 'spread': '52.9', 'groupId': 'OG000'}]}]}, {'title': 'One-stage FVIII activity assay', 'categories': [{'measurements': [{'value': '65.1', 'spread': '45.1', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours', 'description': 'Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics.', 'unitOfMeasure': 'U/percent activity', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK population'}, {'type': 'SECONDARY', 'title': 'PK Analysis- Area Under the Concentration-time Curve (AUC) From Time 0 to the Last Measurable Concentration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'title': 'Chromogenic FVIII activity assay', 'categories': [{'measurements': [{'value': '599', 'spread': '459', 'groupId': 'OG000'}]}]}, {'title': 'One-stage FVIII activity assay', 'categories': [{'measurements': [{'value': '423', 'spread': '340', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours', 'description': 'Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics. AUC was calculated as area under the percent activity-time curve.', 'unitOfMeasure': 'percent activity*hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK population'}, {'type': 'SECONDARY', 'title': 'PK Analysis- Terminal Half-life', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'title': 'Chromogenic FVIII activity assay', 'categories': [{'measurements': [{'value': '3.3', 'spread': '0.4', 'groupId': 'OG000'}]}]}, {'title': 'One-stage FVIII activity assay', 'categories': [{'measurements': [{'value': '3.5', 'spread': '1.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours', 'description': 'Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics. Half-life was calculated as the time it took to reduce percent activity by half.', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK population'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Developed de Novo Anti-OBI-1 Antibody Titers', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': '28 eligible subjects with acquired hemophilia A in the ITT population (n=29), of whom 18 had no detectable anti-porcine FVIII inhibitor titers at baseline (\\<0.6 BU) and 10 had detectable anti-porcine FVIII antibody titers at baseline (\\>=0.6 BU)'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Developed an Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': '21 subjects in the ITT population (n=29) with available baseline and follow-up test results'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Anti-human Factor VIII Antibody Titer', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose', 'reportingStatus': 'POSTED', 'populationDescription': 'Anti-human factor VIII antibody titer data were presented in subject data listings. No statistical test was planned for anti-human factor VIII antibody titer.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': '25 subjects enrolled under OBI-1-301 + 4 subjects enrolled under expanded access OBI-1-301a.', 'groupId': 'FG000', 'numSubjects': '29'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Lack of Efficacy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Development of inhibitors to OBI-1', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Subject status is terminal', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Completion status could not be verified', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Enrollment was conducted at 12 clinical sites in 4 countries (USA, Canada, UK, India). Eight sites enrolled subjects under Protocol OBI-1-301 only. Two US sites enrolled subjects under the expanded access protocol OBI-1-301a and subsequently under protocol OBI-1-301. Another 2 US sites enrolled subjects under OBI-1-301a only.', 'preAssignmentDetails': '29 subjects were enrolled and all were treated with OBI-1. Data from the expanded access subjects (Protocol 301a, n= 4) are included with the data from subjects enrolled under Protocol OBI-1-301 (n=25). 10 subjects discontinued prematurely and 18 completed the study. For one subject, the completion status could not be verified.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '69.8', 'spread': '13.28', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '10', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '19', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}, {'title': 'Canada', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}, {'title': 'India', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Of 16 subjects enrolled in the US, 12 were enrolled under Protocol 301 and 4 were enrolled under the expanded access Protocol 301a.', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'The baseline analysis population comprises all 29 treated subjects (=safety analysis set).'}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 29}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-11-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-04', 'dispFirstSubmitDate': '2014-09-09', 'completionDateStruct': {'date': '2013-10-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-04-17', 'studyFirstSubmitDate': '2010-08-06', 'dispFirstSubmitQcDate': '2014-09-17', 'resultsFirstSubmitDate': '2015-04-28', 'studyFirstSubmitQcDate': '2010-08-09', 'dispFirstPostDateStruct': {'date': '2014-09-29', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2021-05-13', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2015-11-17', 'studyFirstPostDateStruct': {'date': '2010-08-10', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-12-21', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-07-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Anti-human Factor VIII Antibody Titer', 'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose'}], 'primaryOutcomes': [{'measure': 'Percentage of Serious Bleeding Episodes Responsive to OBI-1', 'timeFrame': '24 hours after initiation of treatment', 'description': 'The initial serious ("qualifying") bleeding episode for each subject was analyzed for the primary efficacy outcome measure. A \'positive response\' is defined as \'effective\' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or \'partially effective\' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). \'Poorly effective\' is defined as \'bleeding slightly reduced or unchanged and FVIII levels of less than 50%\'. \'Not effective\' is defined as \'bleeding worsening and FVIII levels of less than 50%\'.'}], 'secondaryOutcomes': [{'measure': 'Overall Percentage of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator', 'timeFrame': 'At the time of final treatment dosing (varied from participant to participant depending on bleeding episodes)', 'description': "Treatment success was defined as control of qualifying bleeding episode at the time of final treatment dosing. A serious bleeding episode was considered 'successfully controlled' if the investigator had checked 'completed OBI-1 therapy as treatment success' on the eCRF."}, {'measure': 'Percentage of Serious Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator', 'timeFrame': '8 hours', 'description': "A 'positive response' is defined as 'effective' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' is defined as 'bleeding slightly reduced or unchanged and FVIII levels of less than 50%'. 'Not effective' is defined as 'bleeding worsening and FVIII levels of less than 50%'."}, {'measure': 'Percentage of Serious Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator', 'timeFrame': '16 hours', 'description': "A 'positive response' is defined as 'effective' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' is defined as 'bleeding slightly reduced or unchanged and FVIII levels of less than 50%'. 'Not effective' is defined as 'bleeding worsening and FVIII levels of less than 50%'."}, {'measure': 'Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes', 'timeFrame': 'Time of successful control of qualifying bleeding episode (varied from participant to participant)', 'description': "'Frequency of infusions' was calculated as the 'average number of infusions per day'. 'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'."}, {'measure': "Total Dose of OBI-1 Required to Successfully Control 'Qualifying' Bleeding Episodes", 'timeFrame': 'Time of successful control of qualifying bleeding episode (varied from participant to participant)', 'description': "'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'."}, {'measure': "Total Number of Infusions of OBI-1 Required to Successfully Control 'Qualifying' Bleeding Episodes", 'timeFrame': 'Time of successful control of qualifying bleeding episode (varied from participant to participant)', 'description': "'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'. A serious bleeding episode was considered 'successfully controlled' if the investigator had checked 'completed OBI-1 therapy as treatment success' on the eCRF."}, {'measure': 'Correlation Between Positive Response to OBI-1 Therapy at 8 Hours and Eventual Control of Serious Bleeding Episodes at 24 Hours', 'timeFrame': '24 hours'}, {'measure': 'Correlation Between Response to OBI-1 Therapy at 16 Hours and Eventual Control of Serious Bleeding Episodes at 24 Hours', 'timeFrame': '24 hours'}, {'measure': 'Correlation Between Response to OBI-1 Therapy at Specified Time Points and Eventual Control of Serious Bleeding Episodes at 24 Hours', 'timeFrame': '24 hours'}, {'measure': 'Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers, the Total Dose of OBI-1, the Outcome at 24 Hours and the Eventual Control of the Bleeding Episode', 'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose'}, {'measure': 'Pharmacokinetics (PK) Analysis- Plasma Clearance', 'timeFrame': 'Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours', 'description': 'Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics.'}, {'measure': 'PK Analysis- Volume of Distribution (Vd) at Steady State', 'timeFrame': 'Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours', 'description': 'Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics.'}, {'measure': 'PK Analysis- Area Under the Concentration-time Curve (AUC) From Time 0 to the Last Measurable Concentration', 'timeFrame': 'Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours', 'description': 'Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics. AUC was calculated as area under the percent activity-time curve.'}, {'measure': 'PK Analysis- Terminal Half-life', 'timeFrame': 'Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours', 'description': 'Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics. Half-life was calculated as the time it took to reduce percent activity by half.'}, {'measure': 'Number of Participants Who Developed de Novo Anti-OBI-1 Antibody Titers', 'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose'}, {'measure': 'Number of Participants Who Developed an Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer', 'timeFrame': 'Through 90 days ± 7 days following final OBI-1 dose'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['haemophilia A', 'blood coagulation disorders', 'coagulation protein disorder', 'hemophilia A', 'Acquired Hemophilia A', 'hematologic diseases', 'hemorrhagic disorders'], 'conditions': ['Acquired Hemophilia A']}, 'referencesModule': {'references': [{'pmid': '25623166', 'type': 'RESULT', 'citation': 'Kruse-Jarres R, St-Louis J, Greist A, Shapiro A, Smith H, Chowdary P, Drebes A, Gomperts E, Bourgeois C, Mo M, Novack A, Farin H, Ewenstein B. Efficacy and safety of OBI-1, an antihaemophilic factor VIII (recombinant), porcine sequence, in subjects with acquired haemophilia A. Haemophilia. 2015 Mar;21(2):162-170. doi: 10.1111/hae.12627. Epub 2015 Jan 27.'}, {'pmid': '39228858', 'type': 'DERIVED', 'citation': 'Miesbach W, Curry N, Knobl P, Percy C, Santoro R, Schmaier AH, Trautmann-Grill K, Badejo K, Chen J, Nouri M, Oberai P, Klamroth R. Real-world use of recombinant porcine sequence factor VIII in the treatment of acquired hemophilia A: EU PASS. Ther Adv Hematol. 2024 Sep 2;15:20406207241260332. doi: 10.1177/20406207241260332. eCollection 2024.'}]}, 'descriptionModule': {'briefSummary': 'This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with acquired hemophilia A.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Written informed consent from subject, trusted person or person who is legally authorized to sign on behalf of the participant (Legal Representative in U.S.), depending on local regulations\n* Participants with acquired hemophilia with autoimmune inhibitory antibodies to human factor VIII with a clinical diagnosis established by the following criteria: a) Prolonged activated partial thromboplastin time (aPTT), b) Prothrombin time (PT) ≤ upper limit of normal (ULN) + 2 seconds and platelet count within normal range, c) Abnormal aPTT mixing study (patient-normal control 1:1) consistent with a factor VIII inhibitors reduced factor VIII activity level (below 10%)\n* Has a serious bleeding episode, as documented by the investigator\n* Be willing and able to follow all instructions and attend all study visits\n* Participants taking anti-thrombotics (such as clopidogrel, heparin or heparin analogue) may be included provided three half-lives of the agent have elapsed since the last dose of the agent\n* Life expectancy, prior to onset of the hemorrhagic episode, of at least 90 days\n* Participants of reproductive age must use acceptable methods of contraception and if female, undergo pregnancy testing as part of the screening process\n\nExclusion Criteria:\n\n* Hemodynamically unstable after blood transfusion, fluid resuscitation and pharmacologic or volume replacement pressor therapy. This hemodynamic instability is characterized by symptomatic hypotension resulting in vital organ dysfunction, such as cardiac ischemia, oliguria (urine volume \\<0.5 mL/kg in the previous six hours), central nervous system hypoperfusion manifested by mental status change such as confusion (unless head injury or intracranial hemorrhage is present), pulmonary compromise, and/or acidosis (manifested by pH and lactate levels)\n* Has an established reason for bleeding that is not correctable\n* Bleeding episode assessed likely to resolve on its own if left untreated\n* Anti-OBI-1 inhibitor that exceeds 20 Bethesda Units (BU) (prospectively or retrospectively)\n* Subsequent bleeding episode at the site of the initial qualifying bleeding episode within two weeks following the final OBI-1 dose for the initial qualifying bleeding episode, or subsequent bleeding episode at a different site than the initial qualifying bleeding episode within 1 week following the final OBI-1 dose for the initial qualifying bleeding episode will not be considered "new" qualifying bleeding episodes\n* Prior history of bleeding disorder other than acquired hemophilia.\n* Known major sensitivity to therapeutic products of pig or hamster origin; examples include therapeutics of porcine origin (e.g. previously marketed porcine factor VIII, Hyate-C®) and recombinant therapeutics prepared from hamster cells (e.g. Humira®, Advate® and Enbrel®)\n* Use of hemophilia medication: rFVIIa within 3 hours prior to OBI-1 administration or aPCC treatment within 6 hours prior to OBI-1 administration\n* Participation in any other clinical study within 30 days of the first OBI-1 treatment\n* Anticipated need for treatment or device during the study that may interfere with the evaluation of the safety or efficacy of OBI-1, or whose safety or efficacy may be affected by OBI-1\n* Is currently pregnant, breastfeeding, or planning to become pregnant or father a child during the study\n* Abnormal baseline findings, any other medical condition(s) or laboratory findings that, in the opinion of the investigator, might jeopardize the subject\'s safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study\n* Inability or unwillingness to comply with the study design, protocol requirements, or the follow-up procedures\n* Participant of majority age under legal protection'}, 'identificationModule': {'nctId': 'NCT01178294', 'briefTitle': 'Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Acquired Hemophilia A Due to Factor VIII Inhibitory Auto-antibodies', 'orgStudyIdInfo': {'id': 'OBI-1-301'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'OBI-1', 'description': 'Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)', 'interventionNames': ['Biological: OBI-1']}], 'interventions': [{'name': 'OBI-1', 'type': 'BIOLOGICAL', 'description': 'Intravenous infusion', 'armGroupLabels': ['OBI-1']}]}, 'contactsLocationsModule': {'locations': [{'zip': '46260', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana Hemophilia and Thrombosis Center', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '70112-2699', 'city': 'New Orleans', 'state': 'Louisiana', 'country': 'United States', 'facility': 'Louisiana Center for Bleeding & Clotting Disorders', 'geoPoint': {'lat': 29.95465, 'lon': -90.07507}}, {'zip': '20892-1508', 'city': 'Bethesda', 'state': 'Maryland', 'country': 'United States', 'facility': 'National Institutes of Health - Warren G. Magnuson Clinical Center', 'geoPoint': {'lat': 38.98067, 'lon': -77.10026}}, {'zip': '02111', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Tufts Medical Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Brigham and Women's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '27599-7305', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina at Chapel Hill Hospital', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '17033', 'city': 'Hershey', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'The Pennsylvania State University and Milton S. Hershey Medical Center', 'geoPoint': {'lat': 40.28592, 'lon': -76.65025}}, {'zip': '37232-9830', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Vanderbilt University Medical Center, Hemostasis/Hemophilia Clinic', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': 'H1T 2M4', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Maisonneuve-Rosemont Hospital', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': '600006', 'city': 'Chennai', 'state': 'Tamil Nadu', 'country': 'India', 'facility': 'Apollo Hospitals', 'geoPoint': {'lat': 13.08784, 'lon': 80.27847}}, {'zip': 'NW3 2QG', 'city': 'London', 'state': 'England', 'country': 'United Kingdom', 'facility': 'Royal Free Hospital-Katharine Dormandy Haemophilia Centre and Thrombosis Unit', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'RG249NA', 'city': 'Basingstoke', 'state': 'Hampshire/England', 'country': 'United Kingdom', 'facility': 'Basingstoke and North Hampshire NHS Foundation Trust', 'geoPoint': {'lat': 51.26249, 'lon': -1.08708}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Takeda'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Baxalta now part of Shire', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}