Ignite Creation Date:
2025-12-24 @ 11:46 AM
Ignite Modification Date:
2025-12-27 @ 8:14 AM
Study NCT ID:
NCT01335061
Status:
COMPLETED
Last Update Posted:
2015-06-08
First Post:
2011-03-30
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United States']}, '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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005164', 'term': 'Factor IX'}], 'ancestors': [{'id': 'D004792', 'term': 'Enzyme Precursors'}, {'id': 'D045762', 'term': 'Enzymes and Coenzymes'}, {'id': 'D001779', 'term': 'Blood Coagulation Factors'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011498', 'term': 'Protein Precursors'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTrials.gov_Inquiries@pfizer.com', 'phone': '1-800-718-1021', 'title': 'Pfizer ClinicalTrials.gov Call Center', 'organization': 'Pfizer, Inc.'}, 'certainAgreement': {'otherDetails': 'Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Until follow-up 82 weeks +/- 4 days.', 'description': 'Five participants experienced six SAEs since one participant experienced the same event twice (nephrolithiasis).', 'eventGroups': [{'id': 'EG000', 'title': 'On-Demand Therapy', 'description': 'Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.', 'otherNumAtRisk': 25, 'otherNumAffected': 16, 'seriousNumAtRisk': 25, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Prophylaxis Therapy', 'description': 'The prophylaxis regimen of approximately 100IU/kg once weekly was initiated at Visit 4.', 'otherNumAtRisk': 25, 'otherNumAffected': 20, 'seriousNumAtRisk': 25, 'seriousNumAffected': 4}], 'otherEvents': [{'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Inappropriate schedule of drug administration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 6}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Drug dose omission', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Medication error', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Underdose', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Wrong dose administration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Head injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Gastritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Local swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}], 'seriousEvents': [{'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Varicella', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Blood pressure decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Lipoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 25, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 17.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Annualized Number of Bleeding Episodes.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'On-Demand Therapy', 'description': 'Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.'}, {'id': 'OG001', 'title': 'Prophylaxis Therapy', 'description': 'The prophylaxis regimen of approximately 100 IU/kg once weekly was initiated at Visit 4.'}], 'classes': [{'categories': [{'measurements': [{'value': '32.9', 'spread': '17.4', 'groupId': 'OG000'}, {'value': '3.6', 'spread': '4.6', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Paired t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': '2 years', 'description': 'The annualized bleed rate (ABR) or the annualized number of bleeding episodes per year, will be derived for each participant for each treatment period by using the following formula: ABR = number of bleeds / (Days on treatment period / 365.25) The number of bleeds for the ABR calculation includes all bleeds requiring treatment with factor IX product during the time on treatment.', 'unitOfMeasure': 'Number of bleeds per year', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The efficacy analysis set (EAS) was used for the primary efficacy analyses with respect to ABR. It includes all participants who participated in at least one day of the routine prophylaxis period (ie, in the study through at least Visit 4).'}, {'type': 'SECONDARY', 'title': 'Response to On-Demand Treatment for All Bleeding Episodes.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}]}, {'units': 'bleeds', 'counts': [{'value': '507', 'groupId': 'OG000'}, {'value': '152', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'On-Demand Therapy-First Infusion', 'description': 'Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.'}, {'id': 'OG001', 'title': 'On-Demand Therapy-Follow-up Infusions', 'description': 'Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.'}], 'classes': [{'title': 'Excellent', 'categories': [{'measurements': [{'value': '271', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}]}, {'title': 'Good', 'categories': [{'measurements': [{'value': '177', 'groupId': 'OG000'}, {'value': '80', 'groupId': 'OG001'}]}]}, {'title': 'Moderate', 'categories': [{'measurements': [{'value': '55', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}]}, {'title': 'No response', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Data not recorded', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '2 years', 'description': 'Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by participant/caregiver or investigator/qualified staff. The 4-point scale assessments are Excellent, Good, Moderate or No response. Responses to number of observations were noted.', 'unitOfMeasure': 'Number of observations with response', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'bleeds', 'denomUnitsSelected': 'Participants', 'populationDescription': 'The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. Follow-up infusion was only required for 18 participants.'}, {'type': 'SECONDARY', 'title': 'Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}, {'units': 'bleeds', 'counts': [{'value': '507', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Population', 'description': 'The data for all the participants is presented.'}], 'classes': [{'title': 'Number of Infusion: 1', 'categories': [{'measurements': [{'value': '416', 'groupId': 'OG000'}]}]}, {'title': 'Number of Infusions: 2', 'categories': [{'measurements': [{'value': '69', 'groupId': 'OG000'}]}]}, {'title': 'Number of Infusions: 3', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Number of Infusions: 4', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Number of Infusions: >4', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '2 years', 'description': 'The number of study drug infusions administered to treat a bleed will be calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). The number of infusions needed to treat a bleed will be classified into the following categories: 1, 2, 3, 4 and \\>4 infusions. If there were more than one bleed location (e.g., ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.', 'unitOfMeasure': 'Number of bleeds requiring infusion', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'bleeds', 'denomUnitsSelected': 'Participants', 'populationDescription': 'The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study.'}, {'type': 'SECONDARY', 'title': 'Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}, {'units': 'breakthrough bleeds', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'The data for all the participants is presented.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.0', 'spread': '0.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '2 years', 'description': 'The number of spontaneous, non-traumatic breakthrough bleeds within 48 hours following a prophylaxis dose of BeneFIX were summarized. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.', 'unitOfMeasure': 'Number of breakthrough bleeds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'breakthrough bleeds', 'denomUnitsSelected': 'Participants', 'populationDescription': 'The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. Three participants experienced 1 spontaneous bleeding episode each within 48 hours of a previous prophylaxis infusion.'}, {'type': 'SECONDARY', 'title': 'Average Infusion Dose.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'On-Demand Therapy', 'description': 'Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.'}, {'id': 'OG001', 'title': 'Prophylaxis Therapy', 'description': 'The prophylaxis regimen of approximately 100IU/kg once weekly was initiated at Visit 4.'}], 'classes': [{'categories': [{'measurements': [{'value': '52', 'spread': '16', 'groupId': 'OG000', 'lowerLimit': '20', 'upperLimit': '54'}, {'value': '99', 'spread': '2', 'groupId': 'OG001', 'lowerLimit': '93', 'upperLimit': '104'}]}]}], 'paramType': 'MEAN', 'timeFrame': '2 years', 'description': 'The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions', 'unitOfMeasure': 'IU/Kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study.'}, {'type': 'SECONDARY', 'title': 'Total Factor Consumption.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'On-Demand Therapy', 'description': 'Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.'}, {'id': 'OG001', 'title': 'Prophylaxis Therapy', 'description': 'The prophylaxis regimen of approximately 100IU/kg once weekly was initiated at Visit 4.'}], 'classes': [{'categories': [{'measurements': [{'value': '707', 'spread': '519', 'groupId': 'OG000'}, {'value': '4985', 'spread': '233', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '2 years', 'description': 'The total amount (IU) infused for each infusion recorded were summed to calculate the total factor consumption for each participant. For each infusion, IU/kg was calculated, using the most recently recorded weight measurement and the total factor consumption, divided by number of infusions, and was summarized similarly to average infusion dose (IU). Annualized TFC by weight was reported. Annualized TFC by weight = (Total IU/kg / treatment interval duration)\\*365.25.', 'unitOfMeasure': 'IU/Kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study.'}, {'type': 'SECONDARY', 'title': 'Incidence of Less Than Expected Therapeutic Effect (LETE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'The data for all the participants is presented.'}], 'classes': [{'title': 'LETE in On-Demand setting', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'LETE in prophylaxis setting', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'LETE (Low recovery)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '2 years', 'description': 'The following criteria are the definitions for LETE in this study: 1. LETE in the On-Demand Setting: LETE occurs in the on-demand setting if 2 successive "No Response" ratings are recorded after 2 successive BeneFIX drug infusions in the absence of confounding factors. 2. LETE in the Prophylaxis Setting: LETE occurs in the prophylaxis setting if there is a spontaneous bleed within 48 hours (≤ 48 hours) after a regularly scheduled prophylactic dose of BeneFIX in the absence of confounding factors. 3. LETE (Low Recovery): LETE can also be lower than expected recovery of FIX in the opinion of the investigator following infusion of BeneFIX in the absence of confounding factors. Each reported occurrence of low recovery LETE was listed.', 'unitOfMeasure': 'Percentage of occurence', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'All Participants', 'description': 'The data for all the participants is presented.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '25'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '25'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Participation included 25 enrolled participants from 15 study centers and 9 countries.', 'preAssignmentDetails': 'The duration of participation was approximately 86 weeks, consisting of a Screening period (1 day to 4 weeks), Period 1 (on-demand treatment for 26 weeks), Period 2 (prophylaxis therapy for 52 weeks), and a follow-up safety period (4 weeks).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'All Participants', 'description': 'The data for all the participants is presented.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '31.3', 'spread': '12.6', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '25', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 25}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-05', 'completionDateStruct': {'date': '2014-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-05-12', 'studyFirstSubmitDate': '2011-03-30', 'resultsFirstSubmitDate': '2015-03-18', 'studyFirstSubmitQcDate': '2011-04-12', 'lastUpdatePostDateStruct': {'date': '2015-06-08', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2015-04-30', 'studyFirstPostDateStruct': {'date': '2011-04-13', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-05-04', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Annualized Number of Bleeding Episodes.', 'timeFrame': '2 years', 'description': 'The annualized bleed rate (ABR) or the annualized number of bleeding episodes per year, will be derived for each participant for each treatment period by using the following formula: ABR = number of bleeds / (Days on treatment period / 365.25) The number of bleeds for the ABR calculation includes all bleeds requiring treatment with factor IX product during the time on treatment.'}], 'secondaryOutcomes': [{'measure': 'Response to On-Demand Treatment for All Bleeding Episodes.', 'timeFrame': '2 years', 'description': 'Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by participant/caregiver or investigator/qualified staff. The 4-point scale assessments are Excellent, Good, Moderate or No response. Responses to number of observations were noted.'}, {'measure': 'Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode.', 'timeFrame': '2 years', 'description': 'The number of study drug infusions administered to treat a bleed will be calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). The number of infusions needed to treat a bleed will be classified into the following categories: 1, 2, 3, 4 and \\>4 infusions. If there were more than one bleed location (e.g., ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.'}, {'measure': 'Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX.', 'timeFrame': '2 years', 'description': 'The number of spontaneous, non-traumatic breakthrough bleeds within 48 hours following a prophylaxis dose of BeneFIX were summarized. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.'}, {'measure': 'Average Infusion Dose.', 'timeFrame': '2 years', 'description': 'The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions'}, {'measure': 'Total Factor Consumption.', 'timeFrame': '2 years', 'description': 'The total amount (IU) infused for each infusion recorded were summed to calculate the total factor consumption for each participant. For each infusion, IU/kg was calculated, using the most recently recorded weight measurement and the total factor consumption, divided by number of infusions, and was summarized similarly to average infusion dose (IU). Annualized TFC by weight was reported. Annualized TFC by weight = (Total IU/kg / treatment interval duration)\\*365.25.'}, {'measure': 'Incidence of Less Than Expected Therapeutic Effect (LETE)', 'timeFrame': '2 years', 'description': 'The following criteria are the definitions for LETE in this study: 1. LETE in the On-Demand Setting: LETE occurs in the on-demand setting if 2 successive "No Response" ratings are recorded after 2 successive BeneFIX drug infusions in the absence of confounding factors. 2. LETE in the Prophylaxis Setting: LETE occurs in the prophylaxis setting if there is a spontaneous bleed within 48 hours (≤ 48 hours) after a regularly scheduled prophylactic dose of BeneFIX in the absence of confounding factors. 3. LETE (Low Recovery): LETE can also be lower than expected recovery of FIX in the opinion of the investigator following infusion of BeneFIX in the absence of confounding factors. Each reported occurrence of low recovery LETE was listed.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Hemophilia B', 'on-demand', 'prophylaxis', 'nonacog alfa', 'BeneFIX', 'FIX'], 'conditions': ['Hemophilia B']}, 'referencesModule': {'references': [{'pmid': '40750723', 'type': 'DERIVED', 'citation': 'Wojciechowski J, Gaitonde P, Hughes JH, Ravva P. Population Modeling of Factor IX Activity Following Administration of Fidanacogene Elaparvovec Gene Therapy in Participants with Hemophilia B. Clin Pharmacokinet. 2025 Oct;64(10):1531-1548. doi: 10.1007/s40262-025-01535-y. Epub 2025 Aug 1.'}, {'pmid': '33631774', 'type': 'DERIVED', 'citation': 'Tortella BJ, Carr ME, Rendo P, Korth-Bradley J, Smith LM, Kavakli K. Once-weekly prophylaxis regimen of nonacog alfa in patients with hemophilia B: an analysis of timing of bleeding event onset. Blood Coagul Fibrinolysis. 2021 Apr 1;32(3):180-185. doi: 10.1097/MBC.0000000000001012.'}], 'seeAlsoLinks': [{'url': 'https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=B1821010&StudyName=Study%20To%20Compare%20On-Demand%20Treatment%20To%20A%20Prophylaxis%20Regimen%20Of%20BeneFIX%20In%20Subjects%20With%20Moderately%20Severe%20to%20Severe%20Hemophilia%20B', 'label': 'To obtain contact information for a study center near you, click here.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Documented history of moderately-severe to severe hemophilia B (FIX activity \\</=2%).\n* Male subjects, aged 12 years to 65 years.\n* Subjects with at least 100 exposure days (EDs) to factor IX products.\n* Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12-month period before the Screening visit.\n\nExclusion Criteria:\n\n* Subjects who have received FIX as a primary or secondary prophylaxis regimen within the last 12 months prior to the Screening visit.\n* Subjects who have had major surgery or an orthopedic surgical procedure within the past 3 months prior to Screening visit.\n* Subjects for which major surgery or orthopedic surgery is planned within the duration of study participation.\n* Subjects with a past history of, or current FIX inhibitor, defined as \\>ULN (upper limit of normal) of the reporting laboratory.\n* Subjects with a known hypersensitivity to any FIX product or hamster protein.'}, 'identificationModule': {'nctId': 'NCT01335061', 'briefTitle': 'Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pfizer'}, 'officialTitle': 'A Multicenter, Open-label Study To Compare On-demand Treatment To A Prophylaxis Regimen Of Nonacog-alfa (Benefix) In Subjects With Moderately Severe To Severe Hemophilia B (Fix:c</=2%)', 'orgStudyIdInfo': {'id': 'B1821010'}, 'secondaryIdInfos': [{'id': '3090A1-3306', 'type': 'OTHER', 'domain': 'Alias Study Number'}, {'id': '2011-000520-15', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'BeneFIX', 'interventionNames': ['Biological: Nonacog alfa']}], 'interventions': [{'name': 'Nonacog alfa', 'type': 'BIOLOGICAL', 'otherNames': ['BeneFIX'], 'description': 'Period 1: During on-demand period, dosing at the discretion of investigator.', 'armGroupLabels': ['BeneFIX']}, {'name': 'Nonacog alfa', 'type': 'BIOLOGICAL', 'otherNames': ['BeneFIX'], 'description': 'Period 2: During the prophylaxis period, 100 IU/kg once weekly', 'armGroupLabels': ['BeneFIX']}]}, 'contactsLocationsModule': {'locations': [{'zip': '4002', 'city': 'Plovdiv', 'country': 'Bulgaria', 'facility': 'UMBAL Sveti Georgi, Klinika po hematologia', 'geoPoint': {'lat': 42.15387, 'lon': 24.75001}}, {'zip': 'K1H 8L6', 'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': 'The Ottawa Hospital', 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'zip': '10000', 'city': 'Zagreb', 'country': 'Croatia', 'facility': 'University Hospital Center Zagreb', 'geoPoint': {'lat': 45.81444, 'lon': 15.97798}}, {'zip': '50400', 'city': 'Kuala Lumpur', 'state': 'Kuala Lumpur', 'country': 'Malaysia', 'facility': 'National Blood Centre', 'geoPoint': {'lat': 3.1412, 'lon': 101.68653}}, {'zip': '25200', 'city': 'Kuantan', 'state': 'Pahang', 'country': 'Malaysia', 'facility': 'Hospital Tengku Ampuan Afzan', 'geoPoint': {'lat': 3.8077, 'lon': 103.326}}, {'zip': '20127', 'city': 'Aguascalientes', 'state': 'Aguascalientes', 'country': 'Mexico', 'facility': 'Instituto Biomedico de Investigacion A.C.', 'geoPoint': {'lat': 21.88262, 'lon': -102.2843}}, {'zip': '64000', 'city': 'Monterrey', 'state': 'Nuevo León', 'country': 'Mexico', 'facility': 'Hospital y Clinica OCA', 'geoPoint': {'lat': 25.68435, 'lon': -100.31721}}, {'zip': '02-797', 'city': 'Warsaw', 'country': 'Poland', 'facility': 'Nzoz Triclinium', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'zip': '50-367', 'city': 'Wroclaw', 'country': 'Poland', 'facility': 'Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku', 'geoPoint': {'lat': 51.10286, 'lon': 17.03006}}, {'zip': '169608', 'city': 'Singapore', 'country': 'Singapore', 'facility': 'Singapore General Hospital', 'geoPoint': {'lat': 1.28967, 'lon': 103.85007}}, {'zip': '302-799', 'city': 'Daejeon', 'country': 'South Korea', 'facility': 'Eulji University Hospital', 'geoPoint': {'lat': 36.34913, 'lon': 127.38493}}, {'zip': '34098', 'city': 'Fatih', 'state': 'Istanbul', 'country': 'Turkey (Türkiye)', 'facility': 'Istanbul Universitesi Cerrahpasa Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bilim Dali', 'geoPoint': {'lat': 41.0225, 'lon': 28.94083}}, {'zip': '06100', 'city': 'Ankara', 'country': 'Turkey (Türkiye)', 'facility': 'Hacettepe Universitesi Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bolumu', 'geoPoint': {'lat': 39.91987, 'lon': 32.85427}}, {'zip': '35100', 'city': 'Bornova/Izmir', 'country': 'Turkey (Türkiye)', 'facility': 'Ege Universitesi Tip Fakultesi'}, {'zip': '27300', 'city': 'Gaziantep', 'country': 'Turkey (Türkiye)', 'facility': 'Gaziantep Universitesi Tip Fakultesi Sahinbey Arastirma ve Uygulama Hastanesi Hematoloji Poliklinigi', 'geoPoint': {'lat': 37.05944, 'lon': 37.3825}}, {'zip': '38039', 'city': 'Kayseri', 'country': 'Turkey (Türkiye)', 'facility': 'Erciyes Universitesi Tip Fakultesi', 'geoPoint': {'lat': 38.73222, 'lon': 35.48528}}, {'zip': '38039', 'city': 'Kayseri̇, Erciyes', 'country': 'Turkey (Türkiye)', 'facility': 'Erciyes Universitesi Tip Fakultesi Hastaneleri M. Kemal Dedeman Onkoloji Hastanesi', 'geoPoint': {'lat': 38.65634, 'lon': 35.58772}}], 'overallOfficials': [{'name': 'Pfizer CT.gov Call Center', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Pfizer'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pfizer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}