Ignite Creation Date:
2025-12-25 @ 3:24 AM
Ignite Modification Date:
2025-12-26 @ 2:03 AM
Study NCT ID:
NCT01757405
Status:
COMPLETED
Last Update Posted:
2021-05-11
First Post:
2012-12-21
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Belgium', 'India']}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}, {'id': 'D002836', 'term': 'Hemophilia B'}], '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'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C103587', 'term': 'recombinant FVIIa'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTransparency@shire.com', 'phone': '+1 866 842 5335', 'title': 'Study Director', 'organization': 'Shire'}, 'certainAgreement': {'otherDetails': "Baxalta's agreements with PIs may vary per requirements of individual PI, but contain common elements. For this study, results may not be published without prior written approval of Sponsor.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': '6 months (throughout study period)', 'eventGroups': [{'id': 'EG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) every 3 hours as on-demand intravenous bolus infusions.', 'otherNumAtRisk': 18, 'otherNumAffected': 4, 'seriousNumAtRisk': 18, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.', 'otherNumAtRisk': 20, 'otherNumAffected': 2, 'seriousNumAtRisk': 20, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Hepatic Enzyme Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Nasal Congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Oropharyngeal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Drug Hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Sinus Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Tinea Versicolour', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}], 'seriousEvents': [{'term': 'Craniocerebral injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Joint injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Head Injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Limb Injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Drug Ineffective', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Muscle Haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Bleeding Episode With "Treatment Success"', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}, {'units': 'Bleeding Episodes', 'counts': [{'value': '289', 'groupId': 'OG000'}, {'value': '256', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIaBI) every 3 hours as on-demand intravenous bolus infusions.'}, {'id': 'OG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.'}], 'classes': [{'categories': [{'measurements': [{'value': '96.19', 'groupId': 'OG000', 'lowerLimit': '93.31', 'upperLimit': '97.86'}, {'value': '79.30', 'groupId': 'OG001', 'lowerLimit': '73.92', 'upperLimit': '83.81'}]}]}], 'analyses': [{'groupIds': ['OG000', 'OG001'], 'paramType': 'Ratio of success proportion', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '90', 'paramValue': '1.21', 'ciLowerLimit': '1.15', 'ciUpperLimit': '1.28', 'groupDescription': 'Equivalence test of successfully treated bleeding episodes (BEs) between or within treatment arms. Denoting the success rates in the two treatment groups by p1 and p2 , the null hypotheses of H01 : p1/p2 \\< 0.83 and H02 : p1/p2 \\> 1.20 was implicitly tested against the one-sided alternatives Ha1: 0.83 ≤ p1/p2 and Ha2 : p1/p2 ≤ 1.20, by comparing the 90% two-sided confidence interval (CI) of the ratio of success proportions to the equivalence region defined as \\[0.83, 1.20\\].', 'statisticalMethod': 'Ratio of success proportion', 'nonInferiorityType': 'NON_INFERIORITY_OR_EQUIVALENCE_LEGACY', 'nonInferiorityComment': 'Equivalence of treatment success proportions in all bleeding episodes for the two treatment groups was determined by comparing the 90% two-sided CI of the ratio of success proportions to the equivalence region defined as \\[0.83, 1.20\\].'}], 'paramType': 'NUMBER', 'timeFrame': 'within 12 hours of first dose', 'description': 'No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.', 'unitOfMeasure': 'percent of bleeding episodes', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding Episodes', 'denomUnitsSelected': 'Bleeding Episodes', 'populationDescription': 'Full Analysis Dataset'}, {'type': 'SECONDARY', 'title': 'Treatment Response for Each Bleeding Episode', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}, {'units': 'Bleeding Episodes', 'counts': [{'value': '289', 'groupId': 'OG000'}, {'value': '256', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIaBI) every 3 hours as on-demand intravenous bolus infusions.'}, {'id': 'OG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.'}], 'classes': [{'title': 'Successful', 'categories': [{'measurements': [{'value': '87.89', 'groupId': 'OG000', 'lowerLimit': '83.62', 'upperLimit': '91.16'}, {'value': '79.30', 'groupId': 'OG001', 'lowerLimit': '73.92', 'upperLimit': '83.81'}]}]}, {'title': 'Excellent', 'categories': [{'measurements': [{'value': '34.60', 'groupId': 'OG000', 'lowerLimit': '29.35', 'upperLimit': '40.26'}, {'value': '36.72', 'groupId': 'OG001', 'lowerLimit': '31.05', 'upperLimit': '42.78'}]}]}, {'title': 'Good', 'categories': [{'measurements': [{'value': '53.29', 'groupId': 'OG000', 'lowerLimit': '47.53', 'upperLimit': '58.96'}, {'value': '42.58', 'groupId': 'OG001', 'lowerLimit': '36.67', 'upperLimit': '48.70'}]}]}, {'title': 'Moderate', 'categories': [{'measurements': [{'value': '9.69', 'groupId': 'OG000', 'lowerLimit': '6.79', 'upperLimit': '13.65'}, {'value': '18.36', 'groupId': 'OG001', 'lowerLimit': '14.10', 'upperLimit': '23.56'}]}]}, {'title': 'No Assessment Available', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.39', 'groupId': 'OG001', 'lowerLimit': '0.07', 'upperLimit': '2.18'}]}]}, {'title': 'None', 'categories': [{'measurements': [{'value': '2.42', 'groupId': 'OG000', 'lowerLimit': '1.18', 'upperLimit': '4.91'}, {'value': '1.95', 'groupId': 'OG001', 'lowerLimit': '0.84', 'upperLimit': '4.49'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'within 24 hours of infusion', 'description': 'Participants rated the treatment of each bleeding episode. If treatment occurred under direct supervision of treating physician, the physician rated the response. Ratings based on a 4 point scale; EXCELLENT - full relief of pain and cessation of objective signs of bleeding (swelling, tenderness, decrease in range of motion \\[for muscle bleeds\\]) within 9 hours of treatment initiation. No additional infusion required to control bleeding, other than prescribed dosing regimen. GOOD - Substantial relief of pain and/or cessation of objective signs of bleeding within 9 hours of treatment initiation. No additional infusion required to control bleeding, other than prescribed dosing regimen. MODERATE - slight relief of pain and slight improvement of signs of bleeding within 9 hours of treatment initiation. Requires additional infusion beyond treatment regimen. NONE - No improvement or condition worsens. SUCCESSFUL = EXCELLENT or GOOD.', 'unitOfMeasure': 'percent of bleeding episodes', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding Episodes', 'denomUnitsSelected': 'Bleeding Episodes', 'populationDescription': 'Full Analysis Dataset'}, {'type': 'SECONDARY', 'title': 'Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}, {'units': 'Bleeding Episodes', 'counts': [{'value': '289', 'groupId': 'OG000'}, {'value': '256', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIaBI) every 3 hours as on-demand intravenous bolus infusions.'}, {'id': 'OG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.'}], 'classes': [{'categories': [{'measurements': [{'value': '93.43', 'groupId': 'OG000', 'lowerLimit': '89.96', 'upperLimit': '95.75'}, {'value': '76.17', 'groupId': 'OG001', 'lowerLimit': '70.59', 'upperLimit': '80.98'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '24 hours post infusion', 'description': 'Clinical responders defined as sustained bleeding control, (no additional hemostatic medication including rFVIIa BI required between 12 and 24 hours after first infusion of the successfully treated bleeding episode).', 'unitOfMeasure': 'percent of bleeding episodes', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding Episodes', 'denomUnitsSelected': 'Bleeding Episodes', 'populationDescription': 'Full Analysis Dataset'}, {'type': 'SECONDARY', 'title': 'Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIaBI) every 3 hours as on-demand intravenous bolus infusions.'}, {'id': 'OG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.'}], 'classes': [{'title': 'SAE-Moderate-Unrelated', 'categories': [{'measurements': [{'value': '5.6', 'groupId': 'OG000'}, {'value': '5.0', 'groupId': 'OG001'}]}]}, {'title': 'SAE-Severe-Unrelated', 'categories': [{'measurements': [{'value': '11.1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'SAE-Severe-Related', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5.0', 'groupId': 'OG001'}]}]}, {'title': 'nsAE-Mild-Unrelated', 'categories': [{'measurements': [{'value': '22.2', 'groupId': 'OG000'}, {'value': '30.0', 'groupId': 'OG001'}]}]}, {'title': 'nsAE-Moderate-Unrelated', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '15.0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '6 months (throughout study period)', 'description': 'Safety was determined by the number of AEs (both serious AEs \\[SAEs\\] and non-serious AEs \\[nsAE\\]). Tolerability was determined by the number of AEs related to rFVIIa BI (both SAEs and nsAEs) as determined by causality assessment of the AEs by the investigator. An AE was deemed Related if the investigator judged the AE to be "possibly related" or "probably related" to rFVIIa BI. The percentage of participants with AEs were presented by seriousness (SAE, nsAE), severity (Mild, Moderate or Severe) and causality (Related or Not Related to rFVIIa BI).', 'unitOfMeasure': 'percent of participants with AEs', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Analysis Dataset'}, {'type': 'SECONDARY', 'title': 'Safety and Tolerability of Treatment Regimens by Clinical Assessment of Adverse Events (AEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}, {'units': 'adverse events', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIaBI) every 3 hours as on-demand intravenous bolus infusions.'}, {'id': 'OG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.'}], 'classes': [{'title': 'SAE-Moderate-Unrelated', 'categories': [{'measurements': [{'value': '6.7', 'groupId': 'OG000'}, {'value': '6.3', 'groupId': 'OG001'}]}]}, {'title': 'SAE-Severe-Unrelated', 'categories': [{'measurements': [{'value': '20.0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'SAE-Severe-Related', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '12.5', 'groupId': 'OG001'}]}]}, {'title': 'nsAE-Mild-Unrelated', 'categories': [{'measurements': [{'value': '73.3', 'groupId': 'OG000'}, {'value': '62.5', 'groupId': 'OG001'}]}]}, {'title': 'nsAE-Moderate-Unrelated', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '18.8', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '6 months (throughout study period)', 'description': 'Safety was determined by the number of AEs (both serious AEs \\[SAEs\\] and non-serious AEs \\[nsAE\\]). Tolerability was determined by the number of AEs related to rFVIIa BI (both SAEs and nsAEs) as determined by causality assessment of the AEs by the investigator. An AE was deemed Related if the investigator judges the AE to be "possibly related" or "probably related" to rFVIIa BI. The percentage of AEs were presented by seriousness (SAE, nsAE), severity (Mild, Moderate or Severe) and causality (Related or Not Related \\[to rFVIIa BI\\]).', 'unitOfMeasure': 'percent of AEs', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'adverse events', 'denomUnitsSelected': 'adverse events', 'populationDescription': 'Safety Analysis Dataset'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Inhibitor Development to FVII', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIaBI) every 3 hours as on-demand intravenous bolus infusions.'}, {'id': 'OG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '6 months (throughout study period)', 'description': 'Development of rFVII inhibitors or FVIIa binding antibodies during the study.', 'unitOfMeasure': 'percent of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Analysis Dataset'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIaBI) every 3 hours as on-demand intravenous bolus infusions.'}, {'id': 'FG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}, {'groupId': 'FG001', 'numSubjects': '20'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '17'}, {'groupId': 'FG001', 'numSubjects': '18'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Enrollment was conduced at 16 clinical sites from the following countries: Japan, Taiwan, Poland, Romania, Russian Federation, Serbia, Spain, Ukraine and the United States.', 'preAssignmentDetails': '40 participants provided informed consent and were screened for study participation, of which there was 1 screen failure. 39 participants (in pre-assignment period) were randomized where 1 participant withdrew after randomization but prior to treatment, therefore 38 participants were treated with recombinant activated factor VII BI (rFVIIa).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with up to 3 doses of 90 micrograms/kg of recombinant activated factor VII BI (rFVIIaBI) every 3 hours as on-demand intravenous bolus infusions.'}, {'id': 'BG001', 'title': 'Arm 2: 1 x 270 Micrograms/kg rFVIIa BI', 'description': 'Bleeding episodes treated with 1 dose of 270 micrograms/kg of recombinant activated factor VII BI (rFVIIa BI) as on-demand intravenous bolus infusion.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '28', 'groupId': 'BG000', 'lowerLimit': '12', 'upperLimit': '53'}, {'value': '28', 'groupId': 'BG001', 'lowerLimit': '12', 'upperLimit': '54'}, {'value': '28', 'groupId': 'BG002', 'lowerLimit': '12', 'upperLimit': '54'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'Years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', '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': '20', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 40}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-02-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-04', 'dispFirstSubmitDate': '2015-11-18', 'completionDateStruct': {'date': '2014-11-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-04-14', 'studyFirstSubmitDate': '2012-12-21', 'dispFirstSubmitQcDate': '2015-11-18', 'resultsFirstSubmitDate': '2016-08-31', 'studyFirstSubmitQcDate': '2012-12-21', 'dispFirstPostDateStruct': {'date': '2015-12-18', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2021-05-11', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2016-08-31', 'studyFirstPostDateStruct': {'date': '2012-12-28', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2016-10-25', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-11-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Bleeding Episode With "Treatment Success"', 'timeFrame': 'within 12 hours of first dose', 'description': 'No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.'}], 'secondaryOutcomes': [{'measure': 'Treatment Response for Each Bleeding Episode', 'timeFrame': 'within 24 hours of infusion', 'description': 'Participants rated the treatment of each bleeding episode. If treatment occurred under direct supervision of treating physician, the physician rated the response. Ratings based on a 4 point scale; EXCELLENT - full relief of pain and cessation of objective signs of bleeding (swelling, tenderness, decrease in range of motion \\[for muscle bleeds\\]) within 9 hours of treatment initiation. No additional infusion required to control bleeding, other than prescribed dosing regimen. GOOD - Substantial relief of pain and/or cessation of objective signs of bleeding within 9 hours of treatment initiation. No additional infusion required to control bleeding, other than prescribed dosing regimen. MODERATE - slight relief of pain and slight improvement of signs of bleeding within 9 hours of treatment initiation. Requires additional infusion beyond treatment regimen. NONE - No improvement or condition worsens. SUCCESSFUL = EXCELLENT or GOOD.'}, {'measure': 'Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes', 'timeFrame': '24 hours post infusion', 'description': 'Clinical responders defined as sustained bleeding control, (no additional hemostatic medication including rFVIIa BI required between 12 and 24 hours after first infusion of the successfully treated bleeding episode).'}, {'measure': 'Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs)', 'timeFrame': '6 months (throughout study period)', 'description': 'Safety was determined by the number of AEs (both serious AEs \\[SAEs\\] and non-serious AEs \\[nsAE\\]). Tolerability was determined by the number of AEs related to rFVIIa BI (both SAEs and nsAEs) as determined by causality assessment of the AEs by the investigator. An AE was deemed Related if the investigator judged the AE to be "possibly related" or "probably related" to rFVIIa BI. The percentage of participants with AEs were presented by seriousness (SAE, nsAE), severity (Mild, Moderate or Severe) and causality (Related or Not Related to rFVIIa BI).'}, {'measure': 'Safety and Tolerability of Treatment Regimens by Clinical Assessment of Adverse Events (AEs)', 'timeFrame': '6 months (throughout study period)', 'description': 'Safety was determined by the number of AEs (both serious AEs \\[SAEs\\] and non-serious AEs \\[nsAE\\]). Tolerability was determined by the number of AEs related to rFVIIa BI (both SAEs and nsAEs) as determined by causality assessment of the AEs by the investigator. An AE was deemed Related if the investigator judges the AE to be "possibly related" or "probably related" to rFVIIa BI. The percentage of AEs were presented by seriousness (SAE, nsAE), severity (Mild, Moderate or Severe) and causality (Related or Not Related \\[to rFVIIa BI\\]).'}, {'measure': 'Percentage of Participants With Inhibitor Development to FVII', 'timeFrame': '6 months (throughout study period)', 'description': 'Development of rFVII inhibitors or FVIIa binding antibodies during the study.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['with Factor VIII (FVIII) or Factor IX (FIX) inhibitors'], 'conditions': ['Hemophilia A', 'Hemophilia B']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Main Inclusion Criteria:\n\n* Participant is male with hemophilia A or B with inhibitors, with a high titer (≥5 Bethesda unit (BU)) or a historical high anamnestic response.\n* Participant is 12 to 65 years old at the time of screening.\n* Participant is currently using or has used bypassing agents for treatment of bleeding episodes.\n* Participant has an annualized bleed rate of 5 or more bleeding episodes per year on average over the 2 years prior to the Screening visit.\n* Participant has a Karnofsky Performance Score ≥60.\n* Participant is hepatitis C virus negative (HCV-) either by antibody testing or polymerase chain reaction (PCR); or hepatitis C virus positive (HCV+) with stable hepatic disease.\n* Participant is human immunodeficiency virus negative (HIV-) or HIV+ with stable disease, CD4+ count ≥200 cells/mm\\^3 at screening.\n* Participant is willing and able to comply with the requirements of the protocol.\n\nMain Exclusion Criteria:\n\n* Participant is not willing to go on an on-demand treatment scheme.\n* Participant is positive for a FVII inhibitor at screening.\n* Participant has clinically symptomatic liver disease.\n* Participant has a platelet count \\<100,000/µL.\n* The use of α-interferon with or without ribavirin is planned for an HCV-infected participant or the use of a protease inhibitor is planned for an HIV-infected participant.\n\n * Participants currently taking any of these medications for ≥30 days are eligible.\n* Participant has a known hypersensitivity to rFVIIa, hamster or murine proteins, or Tween 80.\n* Participant has a known history of being non-responsive to rFVIIa treatment of bleeding episodes.\n* Participant has a prior history of thromboembolic event or diagnosis of other diseases that may increase the participant's risk of thromboembolic complications.\n* Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.\n* Participant is a family member or employee of the investigator.\n* Participant is scheduled for surgery during the study period."}, 'identificationModule': {'nctId': 'NCT01757405', 'briefTitle': 'Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'A PHASE 3, PROSPECTIVE, OPEN-LABEL, RANDOMIZED STUDY TO EVALUATE SAFETY AND EFFICACY OF RECOMBINANT ACTIVATED FVII BI (rFVIIa BI) IN THE TREATMENT OF ACUTE BLEEDING EPISODES PER AN ON-DEMAND REGIMEN IN PATIENTS WITH HEMOPHILIA A OR B WITH INHIBITORS', 'orgStudyIdInfo': {'id': '021101'}, 'secondaryIdInfos': [{'id': '2011-006294-26', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '≤ 3 doses of 90 µg/kg rFVIIa BI', 'interventionNames': ['Biological: Recombinant Factor VIIa BI (rFVIIa BI)']}, {'type': 'EXPERIMENTAL', 'label': 'One dose of 270 µg/kg rFVIIa BI', 'interventionNames': ['Biological: Recombinant Factor VIIa BI (rFVIIa BI)']}], 'interventions': [{'name': 'Recombinant Factor VIIa BI (rFVIIa BI)', 'type': 'BIOLOGICAL', 'description': 'Administered approximately every 3 hours as an intravenous bolus injection on-demand', 'armGroupLabels': ['≤ 3 doses of 90 µg/kg rFVIIa BI']}, {'name': 'Recombinant Factor VIIa BI (rFVIIa BI)', 'type': 'BIOLOGICAL', 'description': 'Administered as a single intravenous bolus injection on-demand', 'armGroupLabels': ['One dose of 270 µg/kg rFVIIa BI']}]}, 'contactsLocationsModule': {'locations': [{'zip': '33607', 'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'Health Point Medical Group "St Joseph\'s Children\'s Hospital"', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '6348522', 'city': 'Nara', 'country': 'Japan', 'facility': 'Nara Medical University Hospital', 'geoPoint': {'lat': 34.68505, 'lon': 135.80485}}, {'zip': '1600023', 'city': 'Tokyo', 'country': 'Japan', 'facility': 'Tokyo Medical University Hospital', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': '31-501', 'city': 'Krakow', 'country': 'Poland', 'facility': 'Kracow Medical Center, LLC', 'geoPoint': {'lat': 50.06143, 'lon': 19.93658}}, {'zip': '02-776', 'city': 'Warsaw', 'country': 'Poland', 'facility': 'Institute of Haematology and Transfusion Medicine, Clinic of Haemostatic Disorders and Internal Diseases', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'zip': '300011', 'city': 'Timișoara', 'country': 'Romania', 'facility': 'Louis Turcanu Emergency Clinical Children´s Hospital', 'geoPoint': {'lat': 45.75372, 'lon': 21.22571}}, {'zip': '610027', 'city': 'Kirov', 'country': 'Russia', 'facility': 'Kirov Hematology and Blood Transfusion Research Institute under the Federal Medical and Biological Agency of Russia', 'geoPoint': {'lat': 58.59809, 'lon': 49.65783}}, {'zip': '125167', 'city': 'Moscow', 'country': 'Russia', 'facility': 'Hematology Research Center under RAMS (State Institution), Department of Reconstructive Orthopedic Surgery for Hemophilia Patients', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'zip': '195213', 'city': 'Saint Petersburg', 'country': 'Russia', 'facility': 'St. Petersburg City Healthcare Institution Municipal Policlinic # 37', 'geoPoint': {'lat': 59.93863, 'lon': 30.31413}}, {'zip': '11000', 'city': 'Belgrade', 'country': 'Serbia', 'facility': 'Clinic for Hematology of the Clinical Center of Serbia', 'geoPoint': {'lat': 44.80401, 'lon': 20.46513}}, {'zip': '15006', 'city': 'A Coruña', 'country': 'Spain', 'facility': 'Hospital Teresa Herrera Materno Infantil del C.H.U.Carretera del Pasajes/nlaboratorio de hematología', 'geoPoint': {'lat': 43.37135, 'lon': -8.396}}, {'zip': '41013', 'city': 'Seville', 'country': 'Spain', 'facility': 'University Hospital Virgen del Rocio', 'geoPoint': {'lat': 37.38283, 'lon': -5.97317}}, {'zip': '11490', 'city': 'Taipei', 'country': 'Taiwan', 'facility': 'Tri-Service General Hospital (TSGH)', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'zip': '83045', 'city': 'Donetsk', 'country': 'Ukraine', 'facility': 'V.K. Gusak Institute of Urgent and Reconstructive Surgery within the Ukrainian National Academy of Medical Sciences, Hematology Department', 'geoPoint': {'lat': 48.023, 'lon': 37.80224}}, {'zip': '79044', 'city': 'Kiev', 'country': 'Ukraine', 'facility': 'Kyiv City Clinical Hospital #9, City Scientific-Practical Center for Diagnostics and Treatment of Patients with Hemostatic Pathlogies', 'geoPoint': {'lat': 50.45466, 'lon': 30.5238}}, {'city': 'Lviv', 'country': 'Ukraine', 'facility': 'State Institution "Institute of Blood Pathology and Transfusion Medicine within the Ukrainian National Academy of Medical Sciences", Hematology Department', 'geoPoint': {'lat': 49.83826, 'lon': 24.02324}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Shire'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Baxalta now part of Shire', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}