Ignite Creation Date:
2025-12-24 @ 7:02 PM
Ignite Modification Date:
2026-01-01 @ 12:50 PM
Study NCT ID:
NCT00037557
Status:
COMPLETED
Last Update Posted:
2007-12-27
First Post:
2002-05-17
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study Evaluating rFIX; BeneFIX in Severe Hemophilia B
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, '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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'count': 20}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2002-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2007-12', 'completionDateStruct': {'date': '2007-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2007-12-18', 'studyFirstSubmitDate': '2002-05-17', 'studyFirstSubmitQcDate': '2002-05-17', 'lastUpdatePostDateStruct': {'date': '2007-12-27', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2002-05-20', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To characterize the safety and efficacy of rFIX in children less than 6 years of age with severe hemophilia B in the setting of acute bleeding episodes, prophylaxis, and/or surgery.'}], 'secondaryOutcomes': [{'measure': 'To measure the incremental recovery of rFIX in children following a 75-IU/kg bolus infusion.'}]}, 'conditionsModule': {'keywords': ['Hemophilia B'], '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': '26196195', 'type': 'DERIVED', 'citation': 'Rendo P, Smith L, Lee HY, Shafer F. Nonacog alfa: an analysis of safety data from six prospective clinical studies in different patient populations with haemophilia B treated with different therapeutic modalities. Blood Coagul Fibrinolysis. 2015 Dec;26(8):912-8. doi: 10.1097/MBC.0000000000000359.'}]}, 'descriptionModule': {'briefSummary': 'To characterize the safety and efficacy of rFIX in children less than 6 years of age with severe hemophilia B in the setting of acute bleeding episodes, prophylaxis, and/or surgery.\n\nThis study will provide an opportunity for systematic observation of treatment with rFIX in children less than 6 years of age regardless of prior FIX treatment. Younger patients exhibit a different pharmacokinetic profile and therefore may respond differently to rFIX infusions when compared with older children and adults. This evaluation will provide data from which recommendations can be made regarding rFIX dosing and treatment of these patients. Surveillance for certain observations that have been made in patients treated with rFIX in the clinical and postmarketing setting will be performed, including inhibitor development, thrombogenicity, FIX recovery/lack of effect, allergic-type manifestations, and RBC agglutination. Comparisons will be derived from published reports and communications describing experience with other FIX products and protein therapeutics in general.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Severe hemophilia B\n* Less than 5 years of age\n* In the investigator's judgment, the patient and/or caregiver will be compliant to study procedures\n\nExclusion Criteria:\n\n* A currently detectable FIX inhibitor. A family history of inhibitors will not exclude the patient.\n* Impaired liver function\n* Impaired renal function"}, 'identificationModule': {'nctId': 'NCT00037557', 'briefTitle': 'Study Evaluating rFIX; BeneFIX in Severe Hemophilia B', 'organization': {'class': 'INDUSTRY', 'fullName': 'Wyeth is now a wholly owned subsidiary of Pfizer'}, 'officialTitle': 'An Open-Label, Single-Arm, Safety and Efficacy Study of Recombinant Human Factor IX (rFIX; BeneFIX®) in Children Less Than 6 Years of Age With Severe Hemophilia B', 'orgStudyIdInfo': {'id': '3090A1-301'}}, 'armsInterventionsModule': {'interventions': [{'name': 'BeneFIX', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'locations': [{'zip': '80262', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '48201', 'city': 'Detroit', 'state': 'Michigan', 'country': 'United States', 'geoPoint': {'lat': 42.33143, 'lon': -83.04575}}, {'zip': '08903', 'city': 'New Brunswick', 'state': 'New Jersey', 'country': 'United States', 'geoPoint': {'lat': 40.48622, 'lon': -74.45182}}, {'zip': '27599-7220', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '45404', 'city': 'Dayton', 'state': 'Ohio', 'country': 'United States', 'geoPoint': {'lat': 39.75895, 'lon': -84.19161}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}], 'overallOfficials': [{'name': 'Medical Monitor, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Wyeth is now a wholly owned subsidiary of Pfizer'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Wyeth is now a wholly owned subsidiary of Pfizer', 'class': 'INDUSTRY'}}}}