Ignite Creation Date:
2025-12-25 @ 12:10 AM
Ignite Modification Date:
2026-02-21 @ 6:45 PM
Study NCT ID:
NCT01863758
Status:
COMPLETED
Last Update Posted:
2018-01-30
First Post:
2013-05-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'sylvia.werner@octapharma.com', 'phone': '415 260-9577', 'title': 'Sylvia Werner', 'organization': 'Octapharma'}, 'certainAgreement': {'otherDetails': 'Octapharma agreements may vary with individual investigators, but will not prohibit any investigator from publishing. Octapharma supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial. Octapharma also reserves the right to review data prior to publishing and provide comments/changes within a certain time period.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'description': 'Safety population: All participants who received at least 1 dose of Human-cl rhFVIII.', 'eventGroups': [{'id': 'EG000', 'title': 'Human-cl rhFVIII', 'description': 'All subject who received at least one dose of intravenous Human-cl rhFVIII (human cell line recombinant Factor VIII).', 'otherNumAtRisk': 66, 'otherNumAffected': 24, 'seriousNumAtRisk': 66, 'seriousNumAffected': 5}], 'otherEvents': [{'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}], 'seriousEvents': [{'term': 'Haematemesis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Fracture of Lower Leg', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Tenotomy of muculus gastrocnemius medialis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Appendicitis Acuta', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'wound inflammation due to surgery', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Pain of lumbar spine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 66, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}], 'frequencyThreshold': '3'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Annualized Number of Bleeding Episodes (BE) in Phase II', 'denoms': [{'units': 'Participants', 'counts': [{'value': '66', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Human-cl rhFVIII', 'description': 'Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.05', 'spread': '13.43', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '<0.05', 'groupIds': ['OG000'], 'paramType': 'Annualized number of bleeding episodes', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.13', 'ciLowerLimit': '2.56', 'ciUpperLimit': '3.80', 'estimateComment': 'The annualized number of bleeding episodes in study GENA-01 was 49.36.', 'groupDescription': 'This study was considered as showing efficacy if the annualized number of bleeding episodes (BE) was reduced by 50% compared to the number of BEs observed in study GENA-01 (NCT00989196).', 'statisticalMethod': '2-sided, 1-sample Poisson test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The annualized number of total BEs was calculated for each participant as follows: d\\*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196).', 'unitOfMeasure': 'Annualized number of bleeding episodes', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII.'}, {'type': 'SECONDARY', 'title': 'Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II', 'denoms': [{'units': 'Participants', 'counts': [{'value': '66', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Human-cl rhFVIII', 'description': 'Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.84', 'spread': '8.81', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '<0.05', 'groupIds': ['OG000'], 'paramType': 'Annualized number of spontaneous BEs', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.90', 'ciLowerLimit': '1.46', 'ciUpperLimit': '2.43', 'estimateComment': 'The annualized number of spontaneous bleeding episodes in study GENA-01 was 32.23', 'groupDescription': 'This study was considered as showing efficacy if the annualized number of spontaneous bleeding episodes (BE) was reduced by 50% compared to the number of spontaneous BEs observed in study GENA-01 (NCT00989196).', 'statisticalMethod': '2-sided, 1-sample Poisson test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The annualized number of spontaneous BEs was calculated for each participant as follows: d\\*y/t, where y = the number of spontaneous BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A spontaneous bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery and BEs due to trauma or due to other causes were not included.', 'unitOfMeasure': 'Annualized number of bleeding episodes', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII.'}, {'type': 'SECONDARY', 'title': 'Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Human-cl rhFVIII', 'description': 'Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval.'}], 'classes': [{'categories': [{'measurements': [{'value': '4.1', 'spread': '17.4', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The annualized number of BEs was calculated for each participant as follows: d\\*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery were not included.', 'unitOfMeasure': 'Annualized number of bleeding episodes', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII. Only participants who received ≤ 2 treatments/week were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Median Dosing Interval During Individually Tailored Prophylaxis', 'denoms': [{'units': 'Participants', 'counts': [{'value': '66', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Human-cl rhFVIII', 'description': 'Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval.'}], 'classes': [{'title': 'Beginning of Phase II', 'categories': [{'measurements': [{'value': '83.1', 'groupId': 'OG000', 'lowerLimit': '56.2', 'upperLimit': '94.4'}]}]}, {'title': 'End of Phase II', 'categories': [{'measurements': [{'value': '83.3', 'groupId': 'OG000', 'lowerLimit': '56.2', 'upperLimit': '95.6'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The median time between 2 prophylactic doses of Human-cl rhFVIII in the prophylactic treatment phase II were determined per patient', 'unitOfMeasure': 'Hours', 'dispersionType': 'Inter-Quartile Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII.'}, {'type': 'SECONDARY', 'title': 'Dosage Per Week in Phase II', 'denoms': [{'units': 'Participants', 'counts': [{'value': '66', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Human-cl rhFVIII', 'description': 'Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval.'}], 'classes': [{'categories': [{'measurements': [{'value': '97.7', 'spread': '23.1', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The mean dosage per week during Phase II of the study are reported.', 'unitOfMeasure': 'IU/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Human-cl rhFVIII', 'description': 'Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval.'}], 'periods': [{'title': 'Pharmacokinetic Assessment', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '66'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '66'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}, {'title': 'Prophylactic Treatment-Phase I', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '66'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '66'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}, {'title': 'Prophylactic Treatment-Phase II', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '66'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '64'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'investigator Error of Early Termination', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '66', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Human-cl rhFVIII', 'description': 'Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '65', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '66', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '65', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Austria', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Romania', 'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}]}]}, {'title': 'Hungary', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}, {'title': 'Poland', 'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}, {'title': 'Slovakia', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}, {'title': 'Bulgaria', 'categories': [{'measurements': [{'value': '31', 'groupId': 'BG000'}]}]}, {'title': 'Germany', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Age', 'classes': [{'categories': [{'measurements': [{'value': '33.6', 'spread': '9.9', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Weight', 'classes': [{'categories': [{'measurements': [{'value': '80.5', 'spread': '19.9', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kg', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'BMI', 'classes': [{'categories': [{'measurements': [{'value': '25.4', 'spread': '5.4', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kg/m^2', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Hemophilia Joint Health Score (HJHS)', 'classes': [{'categories': [{'measurements': [{'value': '37.4', 'spread': '25.3', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'The HJHS is determined through a physical examination of the 6 joints most affected by hemophilia; the elbows, knees, and ankles. Knees and ankles are assessed on 11 items: Swelling, duration of swelling, muscle atrophy, axial alignment, crepitus on motion, flexion loss, extension loss, instability, joint pain, strength, and gait. Elbows are assessed on 9 of the 11 items (axial alignment and gait are not assessed). Each item is assessed on a scale that ranges from 0-1 to 0-4. The total score can range from 0 to 148, with a higher score indicating a bigger effect of hemophilia.', 'unitOfMeasure': 'Units on a scale', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Intent-to-treat/prophylactic/safety population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 66}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-07', 'completionDateStruct': {'date': '2015-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-07-05', 'studyFirstSubmitDate': '2013-05-23', 'resultsFirstSubmitDate': '2016-01-25', 'studyFirstSubmitQcDate': '2013-05-23', 'lastUpdatePostDateStruct': {'date': '2018-01-30', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-07-05', 'studyFirstPostDateStruct': {'date': '2013-05-29', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-01-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2015-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Annualized Number of Bleeding Episodes (BE) in Phase II', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The annualized number of total BEs was calculated for each participant as follows: d\\*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196).'}], 'secondaryOutcomes': [{'measure': 'Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The annualized number of spontaneous BEs was calculated for each participant as follows: d\\*y/t, where y = the number of spontaneous BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A spontaneous bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery and BEs due to trauma or due to other causes were not included.'}, {'measure': 'Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The annualized number of BEs was calculated for each participant as follows: d\\*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery were not included.'}, {'measure': 'Median Dosing Interval During Individually Tailored Prophylaxis', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The median time between 2 prophylactic doses of Human-cl rhFVIII in the prophylactic treatment phase II were determined per patient'}, {'measure': 'Dosage Per Week in Phase II', 'timeFrame': 'Beginning to the end of Phase II (6 months)', 'description': 'The mean dosage per week during Phase II of the study are reported.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Severe Haemophilia A']}, 'descriptionModule': {'briefSummary': 'To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand treatment.', 'detailedDescription': 'There were 3 phases in this study: (1) An initial pharmacokinetic (PK) assessment in which participants received a single infusion of 60±5 IU/kg of Human-cl rhFVIII; blood samples were collected for 72 hours following the infusion. (2) Prophylactic Treatment-Phase I during which participants received infusions of 30-40 IU/kg of human-cl rhFVIII every other day or 3x/week for 1-3 months. (3) Prophylactic Treatment-Phase II during which the dose and dosing interval were determined individually from data gathered in the initial PK assessment. The maximum dosing interval with a dose of ≤ 60-80 IU/kg that maintains a trough level of ≥ 0.01 IU/mL was determined. Participants were treated for 6 months.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Severe haemophilia A (FVIII:C \\< 1%) according to medical history.\n* Male patients ≥ 18 years old.\n* Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 exposure days (EDs).\n* Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start.\n* Immunocompetence (CD4+ count \\> 200/microliter).\n* HIV-negative, if positive, viral load \\< 200 particles/microliter or \\< 400,000 copies/mL.\n* Freely given written informed consent\n\nExclusion Criteria:\n\n* Any coagulation disorder other than haemophilia A.\n* Present or past FVIII inhibitor activity (\\> 0.6 Bethesda Unit \\[BU\\])\n* Severe liver or kidney disease.'}, 'identificationModule': {'nctId': 'NCT01863758', 'briefTitle': 'Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A', 'organization': {'class': 'INDUSTRY', 'fullName': 'Octapharma'}, 'officialTitle': 'Prospective, Open-label, Multicenter Phase 3b Study to Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A', 'orgStudyIdInfo': {'id': 'GENA-21'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Human-cl rhFVIII', 'description': 'Up to 60-80 IU/kg of intravenous Human-cl rhFVIII was administered at an individually determined dose and dose interval.', 'interventionNames': ['Biological: Human-cl rhFVIII']}], 'interventions': [{'name': 'Human-cl rhFVIII', 'type': 'BIOLOGICAL', 'description': 'Human-cl rhFVIII was provided as a freeze-dried concentrate to be reconstituted in water for injection.', 'armGroupLabels': ['Human-cl rhFVIII']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Vienna', 'country': 'Austria', 'facility': 'Medical University Vienna', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'city': 'Plovdiv', 'country': 'Bulgaria', 'facility': 'University Multiprofile Hospital for Active Treatment', 'geoPoint': {'lat': 42.15387, 'lon': 24.75001}}, {'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'Specialized Hospital for Active Treatment', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'city': 'Varna', 'country': 'Bulgaria', 'facility': 'Multiprofile Hospital for Active Treatment', 'geoPoint': {'lat': 43.21912, 'lon': 27.91024}}, {'city': 'Berlin', 'country': 'Germany', 'facility': 'Vivantes Hospital in Friedrichshain', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Heidelberg', 'country': 'Germany', 'facility': 'SRH Kurpfalzklinik Heidelberg GMBH', 'geoPoint': {'lat': 49.40768, 'lon': 8.69079}}, {'city': 'Budapest', 'country': 'Hungary', 'facility': 'Hungarian National Healthcare Center', 'geoPoint': {'lat': 47.49835, 'lon': 19.04045}}, {'city': 'Debrecen', 'country': 'Hungary', 'facility': 'University of Debrecen, Medical and Health Science Center', 'geoPoint': {'lat': 47.53167, 'lon': 21.62444}}, {'city': 'Bialystok', 'country': 'Poland', 'facility': 'University Teaching Hospital in Bialystok, Teaching Department of Hematology with a Subdepartment of Vascular Diseases', 'geoPoint': {'lat': 53.13333, 'lon': 23.16433}}, {'city': 'Gdansk', 'country': 'Poland', 'facility': 'University Clinical Center, Teaching Department of Hematology and Transplantology', 'geoPoint': {'lat': 54.35227, 'lon': 18.64912}}, {'city': 'Torun', 'country': 'Poland', 'facility': 'Nicolaus Copernicus Municipal Specialist Hospital, Department of Hematology', 'geoPoint': {'lat': 53.01375, 'lon': 18.59814}}, {'city': 'Warsaw', 'country': 'Poland', 'facility': 'Institute of Hematology and Transfusion Medicine, Depart. of Hemostatic Disorders and Internal Diseases', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'city': 'Bucharest', 'country': 'Romania', 'facility': 'Sanador SRL', 'geoPoint': {'lat': 44.43225, 'lon': 26.10626}}, {'city': 'Timișoara', 'country': 'Romania', 'facility': "Louis Turcanu Emergency Clinical Children's Hospital", 'geoPoint': {'lat': 45.75372, 'lon': 21.22571}}, {'city': 'Bratislava', 'country': 'Slovakia', 'facility': 'University Hospital Saint Cyril and Metod', 'geoPoint': {'lat': 48.14816, 'lon': 17.10674}}, {'city': 'Martin', 'country': 'Slovakia', 'facility': 'University Hospital Martin, Department of Hematology and Transfusiology', 'geoPoint': {'lat': 49.06651, 'lon': 18.92399}}, {'city': 'Basingstoke', 'country': 'United Kingdom', 'facility': 'Basingstoke and North Hampshire Hospital, Hemophilia, Hemostasis and Thrombosis Center', 'geoPoint': {'lat': 51.26249, 'lon': -1.08708}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal London Hospital, Barts and the London Hemophilia Center', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Manchester Royal Infirmary, Department of Clinical Hematology', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}, {'city': 'Sheffield', 'country': 'United Kingdom', 'facility': 'Royal Hallamshire Hospital', 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Octapharma', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}