Ignite Creation Date:
2025-12-24 @ 4:14 PM
Ignite Modification Date:
2026-01-22 @ 8:42 PM
Study NCT ID:
NCT01810666
Status:
COMPLETED
Last Update Posted:
2015-05-19
First Post:
2013-03-12
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Prophylaxis Versus on Demand Treatment for Children With Hemophilia 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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005169', 'term': 'Factor VIII'}, {'id': 'C078147', 'term': 'F8 protein, human'}, {'id': 'C414350', 'term': 'BAY 14-2222'}], 'ancestors': [{'id': 'D001779', 'term': 'Blood Coagulation Factors'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011498', 'term': 'Protein Precursors'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinical-trials-contact@bayerhealthcare.com', 'title': 'Therapeutic Area Head', 'organization': 'BAYER'}, 'certainAgreement': {'restrictionType': 'GT60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From first treatment through 3 days after the last dose of study drug, up to 6 months', 'eventGroups': [{'id': 'EG000', 'title': 'Rec. Factor VIII On-demand Followed by Prophylaxis', 'description': 'Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.', 'otherNumAtRisk': 30, 'otherNumAffected': 2, 'seriousNumAtRisk': 30, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Rec. Factor VIII On-demand Followed by Prophylaxis', 'description': 'Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.'}], 'classes': [{'categories': [{'measurements': [{'value': '56.00', 'groupId': 'OG000', 'lowerLimit': '48.80', 'upperLimit': '62.54'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)', 'description': 'Annualized bleedings period 1 minus period 2 ITT analysis set.', 'unitOfMeasure': 'Bleeds', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Rec. Factor VIII On-demand Followed by Prophylaxis', 'description': 'Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.'}], 'classes': [{'categories': [{'measurements': [{'value': '37.71', 'groupId': 'OG000', 'lowerLimit': '30.42', 'upperLimit': '46.02'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)', 'description': 'Annualized joint bleedings period 1 minus period 2 ITT analysis set.', 'unitOfMeasure': 'Bleeds', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Rec. Factor VIII On-demand Followed by Prophylaxis', 'description': 'Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.'}], 'classes': [{'categories': [{'measurements': [{'value': '-3.00', 'groupId': 'OG000', 'lowerLimit': '-5.00', 'upperLimit': '0.00'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)', 'description': 'Hemophilia Joint Health Score(HJHS) ranges from 0 to 124. Higher values in the HJHS represent worse situation for the subject. 2-sided Hodges Lehmann estimates for median 95% CI HJHS values difference of changes ITT analysis set.', 'unitOfMeasure': 'Scores on scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Rec. Factor VIII On-demand Followed by Prophylaxis', 'description': 'Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '30'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '30'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Subject recruitment period was between 20-Mar-2013 to 28-Jun-2013.', 'preAssignmentDetails': 'Of 33 participants who were screened for inclusion in the study, 30 were enrolled and received treatment.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Rec. Factor VIII On-demand Followed by Prophylaxis', 'description': 'Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '10', 'spread': '3.9', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '30', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Asian', 'categories': [{'measurements': [{'value': '30', 'groupId': 'BG000'}]}]}, {'title': 'Others', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'Participants'}, {'title': 'Baseline weight', 'classes': [{'categories': [{'measurements': [{'value': '41.42', 'spread': '19.02', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Kg', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Baseline body mass index', 'classes': [{'categories': [{'measurements': [{'value': '18.65', 'spread': '4.16', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Kg/m2', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Target Joint for Bleeds', 'classes': [{'title': 'No', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}]}]}, {'title': 'Yes', 'categories': [{'measurements': [{'value': '22', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'Participants'}, {'title': 'Number of Target Joint', 'classes': [{'title': '0', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}]}]}, {'title': '1', 'categories': [{'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}, {'title': '2', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'Participants'}, {'title': 'Prior FVIII Treatment On-demand', 'classes': [{'categories': [{'measurements': [{'value': '30', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'Participants'}, {'title': 'Number of Bleeds in Last 3 Months', 'classes': [{'categories': [{'measurements': [{'value': '12.4', 'spread': '8.71', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Bleeds', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Number of Joint Bleeds in Last 3 Months', 'classes': [{'categories': [{'measurements': [{'value': '8.73', 'spread': '8.07', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Bleeds', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 30}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-04', 'completionDateStruct': {'date': '2014-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-04-30', 'studyFirstSubmitDate': '2013-03-12', 'resultsFirstSubmitDate': '2014-12-22', 'studyFirstSubmitQcDate': '2013-03-12', 'lastUpdatePostDateStruct': {'date': '2015-05-19', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2014-12-22', 'studyFirstPostDateStruct': {'date': '2013-03-13', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-01-05', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period', 'timeFrame': 'Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)', 'description': 'Annualized bleedings period 1 minus period 2 ITT analysis set.'}], 'secondaryOutcomes': [{'measure': 'Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period', 'timeFrame': 'Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)', 'description': 'Annualized joint bleedings period 1 minus period 2 ITT analysis set.'}, {'measure': 'Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period', 'timeFrame': 'From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)', 'description': 'Hemophilia Joint Health Score(HJHS) ranges from 0 to 124. Higher values in the HJHS represent worse situation for the subject. 2-sided Hodges Lehmann estimates for median 95% CI HJHS values difference of changes ITT analysis set.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Hemophilia A']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.clinicaltrialsregister.eu/', 'label': 'Click here to find information about studies related to Bayer Healthcare products conducted in Europe'}]}, 'descriptionModule': {'briefSummary': 'Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '16 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male, aged 2-16yrs\n* Severe hemophilia A (\\<1% FVIII:C \\[Blood Clotting Factor VIII:C\\] )\n* Minimum of at least 50 documented ED (exposure day) prior to enrolment\n* No measurable inhibitor activity at baseline and history of FVIII inhibitor antibody formation\n* Parents or legal guardians document, sign, and date informed consent\n\nExclusion Criteria:\n\n* Another bleeding disease that is different from hemophilia A\n* Known hypersensitivity to the active substance, mouse or hamster protein\n* Thrombocytopenia (platelet count \\<100 000/mm3) based on previous medical records'}, 'identificationModule': {'nctId': 'NCT01810666', 'briefTitle': 'Prophylaxis Versus on Demand Treatment for Children With Hemophilia A', 'organization': {'class': 'INDUSTRY', 'fullName': 'Bayer'}, 'officialTitle': 'Routine Prophylaxis Treatment Versus On-demand Treatment for Children With Severe Hemophilia A: Comparison of All Bleeding Events in Chinese Hemophilia Patients', 'orgStudyIdInfo': {'id': '16287'}, 'secondaryIdInfos': [{'id': '2014-001362-10', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Recombinant Factor VIII', 'interventionNames': ['Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)']}], 'interventions': [{'name': 'Recombinant Factor VIII (Kogenate FS, BAY14-2222)', 'type': 'BIOLOGICAL', 'description': 'Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.', 'armGroupLabels': ['Recombinant Factor VIII']}]}, 'contactsLocationsModule': {'locations': [{'zip': '510515', 'city': 'Guangzhou', 'state': 'Guangdong', 'country': 'China', 'geoPoint': {'lat': 23.11667, 'lon': 113.25}}, {'zip': '430022', 'city': 'Wuhan', 'state': 'Hubei', 'country': 'China', 'geoPoint': {'lat': 30.58333, 'lon': 114.26667}}, {'zip': '100730', 'city': 'Beijing', 'country': 'China', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}, {'city': 'Beijing', 'country': 'China', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}, {'city': 'Tianjin', 'country': 'China', 'geoPoint': {'lat': 39.14222, 'lon': 117.17667}}], 'overallOfficials': [{'name': 'Bayer Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Bayer'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Bayer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}