Ignite Creation Date:
2025-12-24 @ 11:14 PM
Ignite Modification Date:
2026-02-27 @ 10:34 AM
Study NCT ID:
NCT02020369
Status:
COMPLETED
Last Update Posted:
2017-06-14
First Post:
2013-12-18
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Phase III Study of Coagulation FVIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'D005167', 'term': 'Factor VII'}], 'ancestors': [{'id': 'D004792', 'term': 'Enzyme Precursors'}, {'id': 'D045762', 'term': 'Enzymes and Coenzymes'}, {'id': 'D001779', 'term': 'Blood Coagulation Factors'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011498', 'term': 'Protein Precursors'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'jeffry.lawrence@lfb-usa.com', 'phone': '+1.508.370.5113', 'title': 'Jeffry Lawrence, MD, Vice President, Clinical Development', 'organization': 'LFB USA Inc.'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse event data was collected 2 years, 3 months, 2 days', 'eventGroups': [{'id': 'EG000', 'title': 'Coagulation Factor VIIa (Recombinant): 75 µg/kg', 'description': 'Coagulation Factor VIIa (Recombinant): 75 µg/kg for 3 months\n\nCoagulation Factor VIIa (Recombinant): A cross over design to assess the efficacy of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX', 'otherNumAtRisk': 25, 'otherNumAffected': 3, 'seriousNumAtRisk': 25, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Coagulation Factor VIIa (Recombinant): 225 µg/kg', 'description': 'Coagulation Factor VIIa (Recombinant) : 225 µg/kg for 3 months\n\nCoagulation Factor VIIa (Recombinant): A cross over design to assess the efficacy of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX', 'otherNumAtRisk': 25, 'otherNumAffected': 3, 'seriousNumAtRisk': 25, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.1'}], 'seriousEvents': [{'term': 'acute tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.1'}, {'term': 'subarachnoid hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 15.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Proportion of Successfully Treated Mild/Moderate Bleeding Episodes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}, {'units': 'Bleeding Episodes (BEs)', 'counts': [{'value': '252', 'groupId': 'OG000'}, {'value': '213', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'FVIIa 75 µg/kg', 'description': '75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}, {'id': 'OG001', 'title': 'FVIIa 225µg/kg', 'description': '225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}], 'classes': [{'categories': [{'measurements': [{'value': '.849', 'groupId': 'OG000', 'lowerLimit': '.740', 'upperLimit': '.957'}, {'value': '.932', 'groupId': 'OG001', 'lowerLimit': '.881', 'upperLimit': '.983'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '12 hours after first administration of study drug', 'description': 'For the primary efficacy endpoint, successful treatment of a bleeding episode was defined as a combination of the following:\n\n* "Good" or "Excellent" response noted by the patient\n* Study drug treatment: No further treatment with study drug beyond timepoint for this bleeding episode\n* No other hemostatic treatment needed for this bleeding episode\n* No administration of blood products that would indicate continuation of bleeding beyond timepoint\n* No increase of pain beyond timepoint that could not otherwise be explained', 'unitOfMeasure': 'Proportion of Success of BEs', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding Episodes (BEs)', 'denomUnitsSelected': 'Bleeding Episodes (BEs)', 'populationDescription': 'Treated Population'}, {'type': 'SECONDARY', 'title': 'Proportion of Mild/Moderate Bleeding Episodes With Patient (Pt)-Reported "Good" or "Excellent" Responses at 12 Hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}, {'units': 'Bleeding Episodes (BEs)', 'counts': [{'value': '252', 'groupId': 'OG000'}, {'value': '213', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'FVIIa: 75 µg/kg', 'description': '75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}, {'id': 'OG001', 'title': 'FVIIa: 225 µg/kg', 'description': '225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}], 'classes': [{'categories': [{'measurements': [{'value': '0.857', 'groupId': 'OG000', 'lowerLimit': '0.750', 'upperLimit': '0.964'}, {'value': '0.937', 'groupId': 'OG001', 'lowerLimit': '0.888', 'upperLimit': '0.986'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'at 12 hours', 'description': 'Based on Patient-Reported "Good" or "Excellent" responses as per the below descriptions:\n\nGood: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug.\n\nExcellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required.', 'unitOfMeasure': 'Pt-reported proportion success of BEs', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding Episodes (BEs)', 'denomUnitsSelected': 'Bleeding Episodes (BEs)', 'populationDescription': 'Treated Population'}, {'type': 'SECONDARY', 'title': 'Time to Assessment of a "Good" or "Excellent" Response of Mild/Moderate Bleeding Episodes by the Patient', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}, {'units': 'Bleeding Episodes with event', 'counts': [{'value': '240', 'groupId': 'OG000'}, {'value': '208', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'FVIIa: 75 µg/kg', 'description': '75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}, {'id': 'OG001', 'title': 'FVIIa: 225 µg/kg', 'description': '225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}], 'classes': [{'categories': [{'measurements': [{'value': '5.98', 'groupId': 'OG000', 'lowerLimit': '5.95', 'upperLimit': '6.00'}, {'value': '3.00', 'comment': 'The confidence interval (CI) for the median time is a pointwise CI. The median was 3 hours and approximately 30% of bleeding episodes had good/excellent response exactly at 3 hours which made the confidence limits non-calculable.', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Within 24 hours of Bleeding Episode', 'description': "Categories of Response to Treatment are Described as Follows:\n\nNone: No noticeable effect of the treatment on the bleed or worsening of patient's condition. Continuation of treatment with the study drug was needed.\n\nModerate: Some effect of the treatment on the bleed was noticed, e.g., pain decreased or bleeding signs improved, but bleed continued and required continued treatment with the study drug. Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug.\n\nExcellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required.", 'unitOfMeasure': 'Hours', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding Episodes with event', 'denomUnitsSelected': 'Bleeding Episodes with event', 'populationDescription': 'Treated Population with non-missing measurements'}, {'type': 'SECONDARY', 'title': 'Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}, {'units': 'Bleeding episodes', 'counts': [{'value': '252', 'groupId': 'OG000'}, {'value': '211', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Factor VIIa: 75 µg/kg', 'description': '75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}, {'id': 'OG001', 'title': 'FVIIa: 225 µg/kg', 'description': '225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}], 'classes': [{'categories': [{'measurements': [{'value': '2.5', 'spread': '1.75', 'groupId': 'OG000'}, {'value': '1.4', 'spread': '0.96', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 24 hours of Bleeding Episode', 'unitOfMeasure': 'Number of Administrations of Study Drug', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding episodes', 'denomUnitsSelected': 'Bleeding episodes', 'populationDescription': 'Treated Population with non-missing measurements'}, {'type': 'SECONDARY', 'title': 'Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}, {'units': 'Bleeding Episodes', 'counts': [{'value': '251', 'groupId': 'OG000'}, {'value': '211', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'FVIIa: 75 µg/kg', 'description': '75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}, {'id': 'OG001', 'title': 'FVIIa: 225 µg/kg', 'description': '225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode'}], 'classes': [{'categories': [{'measurements': [{'value': '187.868', 'spread': '131.7982', 'groupId': 'OG000'}, {'value': '252.963', 'spread': '78.9732', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Through study completion', 'unitOfMeasure': 'µg/kg per bleeding episode', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding Episodes', 'denomUnitsSelected': 'Bleeding Episodes', 'populationDescription': 'Treated Population with non-missing measurements'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'FVIIa: 225 µg/kg First, Then 75 µg/kg', 'description': 'Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study.'}, {'id': 'FG001', 'title': 'FVIIa: 75 µg/kg First, Then 225 µg/kg', 'description': 'Coagulation Factor VIIa (Recombinant): Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}, {'groupId': 'FG001', 'numSubjects': '13'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}, {'groupId': 'FG001', 'numSubjects': '11'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '2'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'FVIIa 75 µg/kg First, Then 225 µg/kg', 'description': 'Coagulation Factor VIIa (Recombinant): First Intervention (3 months), Second Intervention (3 months), continue cycle until end of study.'}, {'id': 'BG001', 'title': 'FVIIa 225 µg/kg First, Then 75 µg/kg', 'description': 'Coagulation Factor VIIa (Recombinant): First Intervention (3 months), Second Intervention (3 months), continue cycle until end of study.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '22', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '31.8', 'spread': '12.10', 'groupId': 'BG000'}, {'value': '30.1', 'spread': '12.98', 'groupId': 'BG001'}, {'value': '31.0', 'spread': '12.35', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex/Gender, Customized', 'classes': [{'title': 'Male (n)', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '26', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Russian Federation', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}, {'title': 'United States', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}, {'title': 'Ukraine', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}, {'title': 'Poland', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Georgia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'Bulgaria', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 27}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-05', 'completionDateStruct': {'date': '2015-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-05-15', 'studyFirstSubmitDate': '2013-12-18', 'resultsFirstSubmitDate': '2016-07-12', 'studyFirstSubmitQcDate': '2013-12-18', 'lastUpdatePostDateStruct': {'date': '2017-06-14', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-05-15', 'studyFirstPostDateStruct': {'date': '2013-12-24', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2017-06-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2015-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of Successfully Treated Mild/Moderate Bleeding Episodes', 'timeFrame': '12 hours after first administration of study drug', 'description': 'For the primary efficacy endpoint, successful treatment of a bleeding episode was defined as a combination of the following:\n\n* "Good" or "Excellent" response noted by the patient\n* Study drug treatment: No further treatment with study drug beyond timepoint for this bleeding episode\n* No other hemostatic treatment needed for this bleeding episode\n* No administration of blood products that would indicate continuation of bleeding beyond timepoint\n* No increase of pain beyond timepoint that could not otherwise be explained'}], 'secondaryOutcomes': [{'measure': 'Proportion of Mild/Moderate Bleeding Episodes With Patient (Pt)-Reported "Good" or "Excellent" Responses at 12 Hours', 'timeFrame': 'at 12 hours', 'description': 'Based on Patient-Reported "Good" or "Excellent" responses as per the below descriptions:\n\nGood: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug.\n\nExcellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required.'}, {'measure': 'Time to Assessment of a "Good" or "Excellent" Response of Mild/Moderate Bleeding Episodes by the Patient', 'timeFrame': 'Within 24 hours of Bleeding Episode', 'description': "Categories of Response to Treatment are Described as Follows:\n\nNone: No noticeable effect of the treatment on the bleed or worsening of patient's condition. Continuation of treatment with the study drug was needed.\n\nModerate: Some effect of the treatment on the bleed was noticed, e.g., pain decreased or bleeding signs improved, but bleed continued and required continued treatment with the study drug. Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug.\n\nExcellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required."}, {'measure': 'Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode', 'timeFrame': 'Within 24 hours of Bleeding Episode'}, {'measure': 'Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode', 'timeFrame': 'Through study completion'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Hemophilia A With Inhibitors', 'Hemophilia B With Inhibitors']}, 'referencesModule': {'references': [{'pmid': '40765904', 'type': 'DERIVED', 'citation': 'Young G, Mahlangu J, Boggio LN, Carcao M, Dargaud Y, Escobar M, Giermasz A, Hermans C, Kuriakose P, Miesbach W, Nance D, Rafique A, Sidonio RF Jr, Vilchevska KV, Wang M, Pipe SW. Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials. Blood Vessel Thromb Hemost. 2025 Mar 27;2(3):100069. doi: 10.1016/j.bvth.2025.100069. eCollection 2025 Aug.'}, {'pmid': '40674256', 'type': 'DERIVED', 'citation': 'Carcao M, Hermans C, Giermasz A, Kessler C, Miesbach W, Quon D, Windyga J, Mahlangu J. Safety and Use of Eptacog Beta 225 microg/kg in Patients With Haemophilia A or B With Inhibitors. Haemophilia. 2025 Sep;31(5):957-965. doi: 10.1111/hae.70083. Epub 2025 Jul 17.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX', 'detailedDescription': 'This was a global, multicenter, Phase III, prospective, open-label, randomized, crossover study. After obtaining informed consent and performance of screening procedures, patients who met all inclusion and exclusion criteria were randomized to one of two treatment regimens as follows:\n\n* 75 µg/kg treatment regimen\n* 225 µg/kg treatment regimen\n\nFor each treatment regimen there were two phases:\n\n* Phase A (Initial phase)\n* Phase B (Treatment phase)\n\nThe assigned treatment regimen was the dose administered in Phase A and was the starting dose in Phase B.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* be male with a diagnosis of congenital hemophilia A and/or B of any severity\n* have one of the following:\n* a positive inhibitor test Bethesda Unit (BU) ≥ 5 (as confirmed at screening by the institutional lab), OR\n* a BU\\<5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the subject's medical history, precluding the use of Factor VIII or IX products to treat bleedings, OR\n* a BU\\<5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the subject's medical history, precluding the use of Factor VIII or IX products to treat bleedings\n* be 12 years or older, up to and including 75 years of age (NOTE: different age restrictions may apply per local regulation and/or ethical considerations)\n* have at least 3 bleeding episodes of any severity in the past 6 months be capable of understanding and willing to comply with the conditions of the protocol\n* have read, understood and provided written informed consent (patient and/or parent(s)/legal guardian(s) if \\<18 years of age)\n\nExclusion Criteria:\n\n* have any coagulation disorder other than hemophilia A or B\n* be immuno-suppressed (i.e., the patient should not be receiving systemic immunosuppressive medication, cluster of differentiation 4 (CD4) counts at screening should be \\>200/µl)\n* have a known allergy or hypersensitivity to rabbits\n* have platelet count \\<100,000/mL\n* have had within one month prior to first administration of the study drug in this study a major surgical procedure (e.g. orthopedic, abdominal)\n* have received an investigational drug within 30 days of the first study drug administration, or is expected to receive such drug during participation in this study\n* have a clinically relevant hepatic (AST and/or alanine aminotransferase (ALT) \\>3 times the upper limit of normal) and/or renal impairment (creatinine \\>2 times the upper limit of normal)\n* have a history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis or pulmonary embolism) within 2 years prior to first dose of study drug, or current New York Heart Association (NYHA) functional classification score of stage II -IV\n* have an active malignancy (those with non-melanoma skin cancer are allowed)\n* have any life-threatening disease or other disease or condition which, according to the investigator's judgment, could imply a potential hazard to the patient, interfere with the trial participation or trial outcome (e.g., a history of non-responsiveness to bypassing products)."}, 'identificationModule': {'nctId': 'NCT02020369', 'briefTitle': 'Phase III Study of Coagulation FVIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors', 'organization': {'class': 'INDUSTRY', 'fullName': 'rEVO Biologics'}, 'officialTitle': 'A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors to Factor VIII or IX', 'orgStudyIdInfo': {'id': 'RB-FVIIa-006-13'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'FVIIa: 75 µg/kg first, then 225 µg/kg', 'description': 'Coagulation Factor VIIa (Recombinant): First Intervention (3 months), Second Intervention (3 months), repeat cycle until study completion.', 'interventionNames': ['Biological: Coagulation Factor VIIa (Recombinant)']}, {'type': 'EXPERIMENTAL', 'label': 'FVIIa: 225 µg/kg first, then 75 µg/kg', 'description': 'Coagulation Factor VIIa (Recombinant): First Intervention (3 months), Second Intervention (3 months), repeat cycle until study completion.', 'interventionNames': ['Biological: Coagulation Factor VIIa (Recombinant)']}], 'interventions': [{'name': 'Coagulation Factor VIIa (Recombinant)', 'type': 'BIOLOGICAL', 'description': 'A cross over design to assess the efficacy of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX', 'armGroupLabels': ['FVIIa: 225 µg/kg first, then 75 µg/kg', 'FVIIa: 75 µg/kg first, then 225 µg/kg']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90007', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Orthopaedic Hemophilia Treatment Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'University of California Davis Comprehensive Cancer Center', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado Hemophilia and Thrombosis Center', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Rush University Medical Center', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota Medical Center Fairview', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'city': 'Homyel', 'country': 'Belarus', 'facility': 'Republican Research Center for Radiation Medicine and Human Ecology', 'geoPoint': {'lat': 52.4345, 'lon': 30.9754}}, {'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'Specialized Hospital for Active Treatment of Hematological Diseases', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'city': 'Tbilisi', 'country': 'Georgia', 'facility': 'LTD HEMA', 'geoPoint': {'lat': 41.69143, 'lon': 44.83412}}, {'zip': '5261', 'city': 'Ramat Gan', 'country': 'Israel', 'facility': 'Chaim Sheba Medical Center, Tel-hashomer hospital', 'geoPoint': {'lat': 32.08227, 'lon': 34.81065}}, {'city': 'Warsaw', 'country': 'Poland', 'facility': 'Institute of Hematology and Transfusion Medicine', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'city': 'Bucharest', 'country': 'Romania', 'facility': 'Sandor SRL', 'geoPoint': {'lat': 44.43225, 'lon': 26.10626}}, {'city': 'Kirov', 'country': 'Russia', 'facility': 'Kirov Research Institute of Hematology and Blood Transfusion', 'geoPoint': {'lat': 58.59809, 'lon': 49.65783}}, {'city': 'Moscow', 'country': 'Russia', 'facility': 'Hematology Research Center', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'city': 'Saint Petersburg', 'country': 'Russia', 'facility': 'City Outpatient Clinic #37', 'geoPoint': {'lat': 59.93863, 'lon': 30.31413}}, {'city': 'Kyiv', 'country': 'Ukraine', 'facility': 'Kyiv City Clinical Hospital #9', 'geoPoint': {'lat': 50.45466, 'lon': 30.5238}}, {'city': 'Lviv', 'country': 'Ukraine', 'facility': 'Institute of Blood Pathology and Transfusion Medicine of Academy of Medical Sciences of Ukraine', 'geoPoint': {'lat': 49.83826, 'lon': 24.02324}}, {'city': 'Basingstoke', 'country': 'United Kingdom', 'facility': 'Basingstoke and North Hampshire Hospital, Hemophilia, Hemostasis and Thrombosis Center', 'geoPoint': {'lat': 51.26249, 'lon': -1.08708}}], 'overallOfficials': [{'name': 'Jean Francois Schved, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Saint Eloi Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'rEVO Biologics', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Laboratoire français de Fractionnement et de Biotechnologies', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}