Ignite Creation Date:
2025-12-25 @ 1:33 AM
Ignite Modification Date:
2025-12-27 @ 6:50 AM
Study NCT ID:
NCT01488994
Status:
COMPLETED
Last Update Posted:
2021-05-20
First Post:
2011-12-06
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
BAX 326 Pediatric Study
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Bulgaria']}, 'conditionBrowseModule': {'meshes': [{'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'}]}}, '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 individual PI, but contain common elements. For this study, PIs may be restricted from independently publishing results without prior approval.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'AEs were monitored from the screening visit until the study completion/termination visit.', 'eventGroups': [{'id': 'EG000', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product', 'otherNumAtRisk': 23, 'otherNumAffected': 15, 'seriousNumAtRisk': 23, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Immunology test abnormal', 'notes': 'Total binding antibodies (ABs) to FIX developed in 4 subjects, AB to FIX and rFurin developed in 1 subject, and AB to rFurin developed twice in 1 subject. All ABs were of indeterminate specificity and too low to be verified in the confirmatory assay.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 8, 'numAffected': 6}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}], 'seriousEvents': [{'term': 'Device related infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Humerus fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Hemarthrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Subcutaneous hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 23, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Adverse Events (AEs) Possibly or Probably Related to BAX326', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'AEs considered related to BAX326', 'reportingStatus': 'POSTED', 'populationDescription': 'Full analysis set'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Overall Study Arm', 'description': 'No participants'}], 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints', 'reportingStatus': 'POSTED', 'populationDescription': 'On completion of the study, prospective changes to the planned statistical analysis were made not to analyze AUC 0-72h due to the different time points for the last PK blood sample. Only total AUC \\[i.e. AUC 0-infinity\\] was included in the PK analysis.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Pharmacokinetic Full Analysis Set', 'description': 'Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product.'}], 'classes': [{'categories': [{'measurements': [{'value': '723.7', 'spread': '119', 'groupId': 'OG000'}, {'value': '886', 'spread': '133.66', 'groupId': 'OG001'}, {'value': '808.4', 'spread': '149.14', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'unitOfMeasure': 'IU*hour (hr)/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Mean Residence Time (MRT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Pharmacokinetic Full Analysis Set', 'description': 'Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product.'}], 'classes': [{'categories': [{'measurements': [{'value': '30.62', 'spread': '3.266', 'groupId': 'OG000'}, {'value': '25.31', 'spread': '1.830', 'groupId': 'OG001'}, {'value': '27.85', 'spread': '3.726', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'description': 'Computed as total area under the first moment curve (total AUMC) divided by the total area under the concentration versus time curve (total AUC)', 'unitOfMeasure': 'hours (hr)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Pharmacokinetic Full Analysis Set', 'description': 'Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.1058', 'spread': '0.01650', 'groupId': 'OG000'}, {'value': '0.0874', 'spread': '0.01213', 'groupId': 'OG001'}, {'value': '0.0962', 'spread': '0.01689', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'description': 'Computed as the dose divided by total Area under the curve (AUC)', 'unitOfMeasure': 'dL/(kg*hr)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Incremental Recovery (IR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'BAX326 < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'BAX326 6 to <12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Pharmacokinetic Full Analysis Set', 'description': 'Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.586', 'spread': '0.1320', 'groupId': 'OG000'}, {'value': '0.731', 'spread': '0.1615', 'groupId': 'OG001'}, {'value': '0.665', 'spread': '0.1632', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 30 mins pre-infusion and 30 mins post-infusion', 'description': 'The rise in FIX activity in IU/dL per unit dose administered in IU/kg. Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose', 'unitOfMeasure': 'IU/dL : IU/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Pharmacokinetic Full Analysis Set', 'description': 'Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product.'}], 'classes': [{'categories': [{'measurements': [{'value': '27.67', 'spread': '2.658', 'groupId': 'OG000'}, {'value': '23.15', 'spread': '1.582', 'groupId': 'OG001'}, {'value': '25.31', 'spread': '3.130', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'description': 'Calculated as log\\_e2/λ, where λ is the regression slope in the terminal phase of the least absolute deviations regression model', 'unitOfMeasure': 'hours (hr)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Pharmacokinetic Full Analysis Set', 'description': 'Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.225', 'spread': '0.5233', 'groupId': 'OG000'}, {'value': '2.209', 'spread': '0.3165', 'groupId': 'OG001'}, {'value': '2.695', 'spread': '0.6662', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'description': 'Computed as Clearance (CL) \\* Mean residence time (MRT)', 'unitOfMeasure': 'dL/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Incremental Recovery (IR) Over Time', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Pharmacokinetic Full Analysis Set', 'description': 'Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product.'}], 'classes': [{'title': 'Baseline (N=10, 12, 22)', 'categories': [{'measurements': [{'value': '0.586', 'spread': '0.1320', 'groupId': 'OG000'}, {'value': '0.731', 'spread': '0.1615', 'groupId': 'OG001'}, {'value': '0.665', 'spread': '0.1632', 'groupId': 'OG002'}]}]}, {'title': 'Week 5 (N=11, 12, 23)', 'categories': [{'measurements': [{'value': '0.630', 'spread': '0.1028', 'groupId': 'OG000'}, {'value': '0.726', 'spread': '0.1291', 'groupId': 'OG001'}, {'value': '0.680', 'spread': '0.1245', 'groupId': 'OG002'}]}]}, {'title': 'Week 13 (N=10, 11, 21)', 'categories': [{'measurements': [{'value': '0.676', 'spread': '0.1211', 'groupId': 'OG000'}, {'value': '0.733', 'spread': '0.1400', 'groupId': 'OG001'}, {'value': '0.706', 'spread': '0.1313', 'groupId': 'OG002'}]}]}, {'title': 'Week 26 (N=10, 11, 21)', 'categories': [{'measurements': [{'value': '0.647', 'spread': '0.1274', 'groupId': 'OG000'}, {'value': '0.795', 'spread': '0.1445', 'groupId': 'OG001'}, {'value': '0.724', 'spread': '0.1533', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Within 30 mins pre-infusion and 30 mins post-infusion at baseline, Week 5, Week 13 and Week 26.', 'description': 'IR calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose. IR is determined at baseline (PK analysis), Week 5, Week 13 and Week 26 timepoints. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\> 6 years of age; pediatric participants 6 to \\<12 years of age; pharmacokinetic Full Analysis Set (PKFAS).', 'unitOfMeasure': 'IU/dL : IU/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '14', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'Bleeding Episodes controlled with 1 infusion', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '15', 'groupId': 'OG002'}]}]}, {'title': 'Bleeding Episodes controlled with 2 infusions', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}]}]}, {'title': 'Bleeding episodes controlled with ≥ 3 infusions', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'Bleeding Episodes', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants in the Full Analysis Set who had bleeding episodes'}, {'type': 'SECONDARY', 'title': 'Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '14', 'groupId': 'OG002'}]}, {'units': 'Bleeding Episodes', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}, {'value': '26', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'Excellent', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}, {'value': '13', 'groupId': 'OG002'}]}]}, {'title': 'Good', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}, {'value': '12', 'groupId': 'OG002'}]}]}, {'title': 'Fair', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'None', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'Rating Scale for Treatment of bleeding episodes (4-point ordinal scale): - Excellent: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion required for the control of bleeding. Administration of further infusions to maintain hemostasis did not affect this scoring. - Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. - Fair: Probable and/or slight relief of pain and slight improvement in signs of bleeding after single infusion. Required more than 1 infusion for complete resolution. - None: No improvement or condition worsens.', 'unitOfMeasure': 'Bleeding Episodes', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Bleeding Episodes', 'denomUnitsSelected': 'Bleeding Episodes', 'populationDescription': 'Participants in the Full Analysis Set who had bleeding episodes'}, {'type': 'SECONDARY', 'title': 'Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '1.9', 'spread': '1.89', 'groupId': 'OG000'}, {'value': '3.4', 'spread': '3.93', 'groupId': 'OG001'}, {'value': '2.7', 'spread': '3.14', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'The annualized bleeding rate (ABR) during prophylaxis was calculated only for participants who had adequate treatment time for bleeding rate assessment (i.e., more than 3 months of prophylaxis treatment). The observation period for prophylaxis was to be the time between the first and the last prophylactic infusions. The treatment period for surgery was to be excluded from the bleed rate calculation. ABR calculated as (Number of bleeding episodes/observed treatment period in days) \\* 365.25.', 'unitOfMeasure': 'Bleeding episodes per year', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Consumption of BAX326: Number of Infusions Per Month', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '6.8', 'spread': '0.44', 'groupId': 'OG000'}, {'value': '7.2', 'spread': '0.40', 'groupId': 'OG001'}, {'value': '7.0', 'spread': '0.44', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'Infusions per month', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Consumption of BAX326: Number of Infusions Per Year', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '82.1', 'spread': '5.27', 'groupId': 'OG000'}, {'value': '85.9', 'spread': '4.79', 'groupId': 'OG001'}, {'value': '84.1', 'spread': '5.27', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'Infusions per year', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Consumption of BAX326: Weight-adjusted Consumption Per Month', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '393.4', 'spread': '50.53', 'groupId': 'OG000'}, {'value': '414.8', 'spread': '58.44', 'groupId': 'OG001'}, {'value': '404.6', 'spread': '54.66', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'IU/kg per month', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '4720.9', 'spread': '606.31', 'groupId': 'OG000'}, {'value': '4978.2', 'spread': '701.26', 'groupId': 'OG001'}, {'value': '4855.1', 'spread': '655.93', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'IU/kg per year', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Consumption of BAX326: Weight-adjusted Consumption Per Event', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'Consumption of BAX326 for prophylactic infusions', 'categories': [{'measurements': [{'value': '56.3', 'spread': '10.29', 'groupId': 'OG000'}, {'value': '56.2', 'spread': '6.55', 'groupId': 'OG001'}, {'value': '56.2', 'spread': '8.34', 'groupId': 'OG002'}]}]}, {'title': 'Consumption of BAX326 for infusions to treat BEs', 'categories': [{'measurements': [{'value': '57.6', 'spread': '11.87', 'groupId': 'OG000'}, {'value': '62.1', 'spread': '16.00', 'groupId': 'OG001'}, {'value': '59.9', 'spread': '13.74', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'Event includes prophylactic infusions of study product and infusions of study product for treatment of bleeding episodes (BEs).', 'unitOfMeasure': 'IU/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. AB=antibodies in category for outcome measure data.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Safety: Number of Participants With Thrombotic Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'Number with CS changes in haematology params', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Number with CS changes in clin. chemistry params', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Number with CS changes in vital signs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'Categories consist of Clinically Significant (CS) changes in haemaotology parameters, clinical chemistry parameters and vital signs. Abbreviations in categories; Clin=clinical; params=parameters', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'Number who developed antibodies to CHO proteins', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Number who developed antibodies to rFurin', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'Peds-QL 2-4 (N=4)', 'categories': [{'measurements': [{'value': '3.27', 'spread': '10.119', 'groupId': 'OG000'}]}]}, {'title': 'Peds-QL 5-7 (N=2)', 'categories': [{'measurements': [{'value': '-7.07', 'spread': '6.917', 'groupId': 'OG000'}]}]}, {'title': 'Peds-QL 8-12 (N=10)', 'categories': [{'measurements': [{'value': '4.02', 'spread': '11.038', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and 6 months', 'description': 'For this study, the PedsQL™ questionnaires for participants 2 to 7 years of age (parent-proxy versions for age groups 2-4 years and 5-7 years) and PedsQL™ Child version for participants 8 to 12 years of age were used. The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. A 5-point score is used for each domain: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0 so that higher scores indicate better quality of life (QoL). The total score is the mean (average) of all scores from the 4 domains. The change from baseline in total score is reported- a positive score indicates a better QoL compared to baseline and a negative score indicates a poorer QoL compared to baseline.', 'unitOfMeasure': 'Scores on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants in the Full Analysis Set who had PedsQL™ data for baseline and 6 months'}, {'type': 'SECONDARY', 'title': 'Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.18', 'spread': '0.456', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and 6 months', 'description': 'The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100.', 'unitOfMeasure': 'Scores on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants in the Full Analysis Set who had Haemo-QoL data for baseline and 6 months'}, {'type': 'SECONDARY', 'title': 'Health Resource Use: Number of Hospitalizations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'Week 5 (N=11, 12, 23)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '0'}]}]}, {'title': 'Week 13 (N=11, 11, 22)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '1'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '1'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '1'}]}]}, {'title': 'Week 26 (N=11, 11, 22)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '1'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '1'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': 'The number of hospitalizations per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.', 'unitOfMeasure': 'Hospitalizations', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Health Resource Use: Length of Hospitalization', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'PK assessment', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'No participants were hospitalized during this timepoint.', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'No participants were hospitalized during this timepoint.', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'No participants were hospitalized during this timepoint.', 'groupId': 'OG002', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}, {'title': 'Week 5', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'No participants were hospitalized during this timepoint.', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'No participants were hospitalized during this timepoint.', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'No participants were hospitalized during this timepoint.', 'groupId': 'OG002', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}, {'title': 'Week 13 (N=1, 1, 2)', 'categories': [{'measurements': [{'value': '2.0', 'groupId': 'OG000', 'lowerLimit': '2', 'upperLimit': '2'}, {'value': '4.0', 'groupId': 'OG001', 'lowerLimit': '4', 'upperLimit': '4'}, {'value': '3.0', 'groupId': 'OG002', 'lowerLimit': '2', 'upperLimit': '4'}]}]}, {'title': 'Week 26 (N=NA, 1, 1)', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'No participants were hospitalized during this timepoint.', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': '13.0', 'groupId': 'OG001', 'lowerLimit': '13', 'upperLimit': '13'}, {'value': '13.0', 'groupId': 'OG002', 'lowerLimit': '13', 'upperLimit': '13'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': 'The length of hospitalization per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.', 'unitOfMeasure': 'Days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': "Health Resource Use: Unscheduled Doctor's Office Visits", 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'PK assessment (N=11, 12, 23)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '0'}]}]}, {'title': 'Week 5 (N=11, 12, 23)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '1'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '1'}]}]}, {'title': 'Week 13 (N=11, 11, 22)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '1'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '2'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '2'}]}]}, {'title': 'Week 26 (N=11, 11, 22)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '6'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '6'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': "The number of unscheduled doctor's Office visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.", 'unitOfMeasure': 'Visits', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': "Participants who had unscheduled visits to a doctor's office"}, {'type': 'SECONDARY', 'title': 'Health Resource Use: Emergency Room Visits', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'PK assessment (N=11, 12, 23)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '0'}]}]}, {'title': 'Week 5 (N=11, 12, 23)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '0'}]}]}, {'title': 'Week 13 (N=11, 11, 22)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '2'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '2'}]}]}, {'title': 'Week 26 (N=11, 11, 22)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '4'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '1'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '4'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': 'The number of Emergency Room visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.', 'unitOfMeasure': 'Visits', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Health Resource Use: Days Lost From School', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG001', 'title': 'Pediatric Participants 6 to < 12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'OG002', 'title': 'Full Analysis Set', 'description': 'Comprised of all participants who received at least one infusion of study product'}], 'classes': [{'title': 'PK assessment (N=11, 12, 23)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '6'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '6'}]}]}, {'title': 'Week 5 (N=11, 12, 23)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '9'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '9'}]}]}, {'title': 'Week 13 (N=11, 11, 22)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '14'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '4'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '14'}]}]}, {'title': 'Week 26 (N=11, 11, 22)', 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '6'}, {'value': '0.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '11'}, {'value': '0.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '11'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': 'The number of days lost from school per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.', 'unitOfMeasure': 'Days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who missed days from school'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'FG001', 'title': 'Pediatric Participants 6 to <12 Years of Age', 'description': 'Pediatric participants 6 to \\< 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}, {'groupId': 'FG001', 'numSubjects': '12'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}, {'groupId': 'FG001', 'numSubjects': '11'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Enrollment was conducted at 11 clinical sites in 6 countries (United Kingdom, Poland, Romania, Russian Federation, Ukraine, India). A total of 23 participants were enrolled in the study. Of these, 11 were \\< 6 years of age and 12 were 6 to \\<12 years of age.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Pediatric Participants < 6 Years of Age', 'description': 'Pediatric participants \\< 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'BG001', 'title': 'Pediatric Participants 6 to <12 Years of Age', 'description': 'Pediatric participants 6 to \\<12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '3.83', 'groupId': 'BG000', 'lowerLimit': '1.8', 'upperLimit': '6.0'}, {'value': '9.8', 'groupId': 'BG001', 'lowerLimit': '7.1', 'upperLimit': '11.8'}, {'value': '6.94', 'groupId': 'BG002', 'lowerLimit': '1.8', 'upperLimit': '11.8'}]}]}], 'paramType': 'MEAN', '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': '11', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 23}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-12-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-04', 'dispFirstSubmitDate': '2014-05-29', 'completionDateStruct': {'date': '2013-05-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-04-30', 'studyFirstSubmitDate': '2011-12-06', 'dispFirstSubmitQcDate': '2014-05-29', 'resultsFirstSubmitDate': '2016-03-24', 'studyFirstSubmitQcDate': '2011-12-07', 'dispFirstPostDateStruct': {'date': '2014-06-02', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2021-05-20', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2016-07-21', 'studyFirstPostDateStruct': {'date': '2011-12-09', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2016-09-01', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-05-14', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Adverse Events (AEs) Possibly or Probably Related to BAX326', 'timeFrame': 'Throughout study period (approximately 17 months)'}], 'secondaryOutcomes': [{'measure': 'Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose)', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints'}, {'measure': 'Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose)', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.'}, {'measure': 'Pharmacokinetics (PK): Mean Residence Time (MRT)', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'description': 'Computed as total area under the first moment curve (total AUMC) divided by the total area under the concentration versus time curve (total AUC)'}, {'measure': 'Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL)', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'description': 'Computed as the dose divided by total Area under the curve (AUC)'}, {'measure': 'Pharmacokinetics (PK): Incremental Recovery (IR)', 'timeFrame': 'Within 30 mins pre-infusion and 30 mins post-infusion', 'description': 'The rise in FIX activity in IU/dL per unit dose administered in IU/kg. Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose'}, {'measure': 'Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2)', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'description': 'Calculated as log\\_e2/λ, where λ is the regression slope in the terminal phase of the least absolute deviations regression model'}, {'measure': 'Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss)', 'timeFrame': 'Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.', 'description': 'Computed as Clearance (CL) \\* Mean residence time (MRT)'}, {'measure': 'Pharmacokinetics (PK): Incremental Recovery (IR) Over Time', 'timeFrame': 'Within 30 mins pre-infusion and 30 mins post-infusion at baseline, Week 5, Week 13 and Week 26.', 'description': 'IR calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose. IR is determined at baseline (PK analysis), Week 5, Week 13 and Week 26 timepoints. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\> 6 years of age; pediatric participants 6 to \\<12 years of age; pharmacokinetic Full Analysis Set (PKFAS).'}, {'measure': 'Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode', 'timeFrame': 'Throughout study period (approximately 17 months)'}, {'measure': 'Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'Rating Scale for Treatment of bleeding episodes (4-point ordinal scale): - Excellent: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion required for the control of bleeding. Administration of further infusions to maintain hemostasis did not affect this scoring. - Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. - Fair: Probable and/or slight relief of pain and slight improvement in signs of bleeding after single infusion. Required more than 1 infusion for complete resolution. - None: No improvement or condition worsens.'}, {'measure': 'Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR)', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'The annualized bleeding rate (ABR) during prophylaxis was calculated only for participants who had adequate treatment time for bleeding rate assessment (i.e., more than 3 months of prophylaxis treatment). The observation period for prophylaxis was to be the time between the first and the last prophylactic infusions. The treatment period for surgery was to be excluded from the bleed rate calculation. ABR calculated as (Number of bleeding episodes/observed treatment period in days) \\* 365.25.'}, {'measure': 'Consumption of BAX326: Number of Infusions Per Month', 'timeFrame': 'Throughout study period (approximately 17 months)'}, {'measure': 'Consumption of BAX326: Number of Infusions Per Year', 'timeFrame': 'Throughout study period (approximately 17 months)'}, {'measure': 'Consumption of BAX326: Weight-adjusted Consumption Per Month', 'timeFrame': 'Throughout study period (approximately 17 months)'}, {'measure': 'Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized)', 'timeFrame': 'Throughout study period (approximately 17 months)'}, {'measure': 'Consumption of BAX326: Weight-adjusted Consumption Per Event', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'Event includes prophylactic infusions of study product and infusions of study product for treatment of bleeding episodes (BEs).'}, {'measure': 'Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX)', 'timeFrame': 'Throughout study period (approximately 17 months)'}, {'measure': 'Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX)', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. AB=antibodies in category for outcome measure data.'}, {'measure': 'Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis', 'timeFrame': 'Throughout study period (approximately 17 months)'}, {'measure': 'Safety: Number of Participants With Thrombotic Events', 'timeFrame': 'Throughout study period (approximately 17 months)'}, {'measure': 'Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'Categories consist of Clinically Significant (CS) changes in haemaotology parameters, clinical chemistry parameters and vital signs. Abbreviations in categories; Clin=clinical; params=parameters'}, {'measure': 'Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin)', 'timeFrame': 'Throughout study period (approximately 17 months)', 'description': 'If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay.'}, {'measure': 'Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score', 'timeFrame': 'Baseline and 6 months', 'description': 'For this study, the PedsQL™ questionnaires for participants 2 to 7 years of age (parent-proxy versions for age groups 2-4 years and 5-7 years) and PedsQL™ Child version for participants 8 to 12 years of age were used. The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. A 5-point score is used for each domain: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0 so that higher scores indicate better quality of life (QoL). The total score is the mean (average) of all scores from the 4 domains. The change from baseline in total score is reported- a positive score indicates a better QoL compared to baseline and a negative score indicates a poorer QoL compared to baseline.'}, {'measure': 'Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score', 'timeFrame': 'Baseline and 6 months', 'description': 'The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100.'}, {'measure': 'Health Resource Use: Number of Hospitalizations', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': 'The number of hospitalizations per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.'}, {'measure': 'Health Resource Use: Length of Hospitalization', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': 'The length of hospitalization per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.'}, {'measure': "Health Resource Use: Unscheduled Doctor's Office Visits", 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': "The number of unscheduled doctor's Office visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set."}, {'measure': 'Health Resource Use: Emergency Room Visits', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': 'The number of Emergency Room visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.'}, {'measure': 'Health Resource Use: Days Lost From School', 'timeFrame': 'Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26', 'description': 'The number of days lost from school per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants \\< 6 years of age; pediatric participants 6 to \\<12 years of age; Full Analysis Set.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Hemophilia B']}, 'referencesModule': {'references': [{'pmid': '25495591', 'type': 'RESULT', 'citation': 'Urasinski T, Stasyshyn O, Andreeva T, Rusen L, Perina FG, Oh MS, Chapman M, Pavlova BG, Valenta-Singer B, Abbuehl BE. Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical trial. Haemophilia. 2015 Mar;21(2):196-203. doi: 10.1111/hae.12548. Epub 2014 Dec 11.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.', 'detailedDescription': 'The secondary outcome measure: Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours (h) Post-infusion analysis was not done due to the different time-points for the last PK blood sample, AUC0-72 h was redundant and only total AUC was included in the PK analysis.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Main Inclusion Criteria:\n\n* Participant and/or legal representative has/have voluntarily provided signed informed consent\n* Participant has severe (FIX level \\< 1%) or moderately severe (FIX level ≤ 2%) hemophilia B\n* Participant is \\< 12 years old at the time of screening\n* Participant has no evidence of a history of FIX inhibitors (based on the participant's medical records)\n* Participant is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm\\^3\n\nMain Exclusion Criteria:\n\n* Participant has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 Bethesda Unit (BU)\n* Participant has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)\n* Participant has evidence of an ongoing or recent thrombotic disease\n* Participant has an inherited or acquired hemostatic defect other than hemophilia B"}, 'identificationModule': {'nctId': 'NCT01488994', 'briefTitle': 'BAX 326 Pediatric Study', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'BAX 326 (Recombinant Factor IX): A Phase 2/3 Prospective, Uncontrolled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, and Immunogenicity in Previously Treated Pediatric Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level 1-2%) Hemophilia B', 'orgStudyIdInfo': {'id': '251101'}, 'secondaryIdInfos': [{'id': '2011-002437-19', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'BAX326 < 6 years of age', 'interventionNames': ['Biological: BAX326']}, {'type': 'EXPERIMENTAL', 'label': 'BAX326 6 to <12 years of age', 'interventionNames': ['Biological: BAX326']}], 'interventions': [{'name': 'BAX326', 'type': 'BIOLOGICAL', 'otherNames': ['BAX 326', 'RIXUBIS'], 'description': 'All participants underwent a pharmacokinetic evaluation with BAX326 (recombinant Factor IX) followed by twice weekly prophylactic treatment for 6 months or for at least 50 exposure days, whichever occurred last.', 'armGroupLabels': ['BAX326 6 to <12 years of age', 'BAX326 < 6 years of age']}]}, 'contactsLocationsModule': {'locations': [{'zip': '110002', 'city': 'New Delhi', 'country': 'India', 'facility': 'LNJP Maulana Azad Medical College & Associated Hospitals', 'geoPoint': {'lat': 28.62137, 'lon': 77.2148}}, {'zip': '30-663', 'city': 'Krakow', 'country': 'Poland', 'facility': 'University Pediatric Hospital', 'geoPoint': {'lat': 50.06143, 'lon': 19.93658}}, {'zip': '10-561', 'city': 'Olsztyn', 'country': 'Poland', 'facility': 'Stanislaw Popowski Provincial Specialist Pediatric Hospital', 'geoPoint': {'lat': 53.78376, 'lon': 20.49272}}, {'zip': '71-252', 'city': 'Szczecin', 'country': 'Poland', 'facility': 'Professor Tadeusz Sokolowski Independent Public Teaching Hospital of the Pomeranian Medical University in Szczecin', 'geoPoint': {'lat': 53.42894, 'lon': 14.55302}}, {'zip': '11156', 'city': 'Bucharest', 'country': 'Romania', 'facility': 'S.C. Sanador SRL', 'geoPoint': {'lat': 44.43225, 'lon': 26.10626}}, {'zip': '300011', 'city': 'Timișoara', 'country': 'Romania', 'facility': "Louis Turcanu Emergency Children's Hospital", 'geoPoint': {'lat': 45.75372, 'lon': 21.22571}}, {'zip': '350007', 'city': 'Krasnodar', 'country': 'Russia', 'facility': 'Pediatric Regional Clinical Hospital, Hematology Department', 'geoPoint': {'lat': 45.04534, 'lon': 38.98178}}, {'zip': '195213', 'city': 'Saint Petersburg', 'country': 'Russia', 'facility': 'Republican Center for Hemophilia Treatment', 'geoPoint': {'lat': 59.93863, 'lon': 30.31413}}, {'zip': '620149', 'city': 'Yekaterinburg', 'country': 'Russia', 'facility': 'Regional Clinical Hospital', 'geoPoint': {'lat': 56.85733, 'lon': 60.61529}}, {'zip': '79044', 'city': 'Lviv', 'country': 'Ukraine', 'facility': 'State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine"', 'geoPoint': {'lat': 49.83826, 'lon': 24.02324}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Manchester Children´s Hospital', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Takeda'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Baxalta now part of Shire', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}