Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B
NCT ID: NCT00364182
Last Updated: 2011-10-04
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
50 participants
INTERVENTIONAL
2007-05-31
2010-10-31
Brief Summary
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The primary endpoint is annualized number of bleeding episodes, compared between the first on-demand period and each of the prophylaxis regimens. A comparison of annualized bleeding episodes between the two prophylaxis regimens will also be performed. Subject-reported outcomes will also be collected using a patient diary. At 24 and 48 hours following the onset of each joint bleeding episode, information on pain, sleep, and work will be collected. Additional data on physical functioning will be recorded on the diary at the end of the 16-week on-demand period, and at the end of each prophylaxis period. Information on safety will also be collected.
Each subject will participate in this study for approximately 59 weeks (15 months), including a screening period of up to 3 weeks, an initial 16-week on-demand period, two prophylaxis treatment periods of 16 weeks each, separated by an 8-week on-demand treatment period.
A modified FIX recovery study will be performed once during each prophylaxis period.
The study diary will also be used by subjects to collect secondary endpoints. These endpoints, which are subject-reported outcomes, will be recorded in the diary at 24 and 48 hours following the onset of each joint bleeding episode. Additional data on physical functioning will be recorded on the diary at the end of the 16-week on-demand period, and at the end of each prophylaxis period.
Patients will be recruited in the United States, Canada, Europe and Russia.
Detailed Description
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Conditions
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Study Design
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RANDOMIZED
CROSSOVER
PREVENTION
NONE
Study Groups
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A
Recombinant Coagulation Factor IX (BeneFIX)
100 IU/kg once weekly then crossover to 50 IU/kg twice weekly
B
Recombinant Coagulation Factor IX (BeneFIX)
50 IU/kg twice weekly then crossover to 100 IU/kg once weekly
Interventions
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Recombinant Coagulation Factor IX (BeneFIX)
100 IU/kg once weekly then crossover to 50 IU/kg twice weekly
Recombinant Coagulation Factor IX (BeneFIX)
50 IU/kg twice weekly then crossover to 100 IU/kg once weekly
Eligibility Criteria
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Inclusion Criteria
* Male subjects, aged 6 years to 65 years.
* Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12 months before screening.
Exclusion Criteria
* Subjects with HIV+ who have a CD4 count less than 200
* Subjects with hepatic or renal impairment.
* Prothrombin time or International Normalized Ration more than 1.5 times the upper limit of normal.
* Major surgery or an orthopedic surgical procedure within the past 3 months or is planned within the duration of participation in this study.
* Past history of, or current FIX inhibitor.
* Hypersensitivity to any FIX product or hamster protein.
* Exposure to any investigational drug, except for rFIX-R, or device within 30 days of providing consent/assent (as appropriate) for this study.
* Bleeding disorders other than hemophilia B.
* Concurrent inflammatory disease that in the investigator's judgment could confound the study results.
6 Years
65 Years
MALE
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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Pfizer Investigational Site
Phoenix, Arizona, United States
Pfizer Investigational Site
Chicago, Illinois, United States
Pfizer Investigational Site
Chicago, Illinois, United States
Pfizer Investigational Site
New Brunswick, New Jersey, United States
Pfizer Investigational Site
Houston, Texas, United States
Pfizer Investigational Site
Edmonton, Alberta, Canada
Pfizer Investigational Site
Edmonton, Alberta, Canada
Pfizer Investigational Site
Ottawa, Ontario, Canada
Pfizer Investigational Site
Zagreb, , Croatia
Pfizer Investigational Site
Budapest, , Hungary
Pfizer Investigational Site
Castelfranco Veneto (TV), , Italy
Pfizer Investigational Site
Coppito (AQ), , Italy
Pfizer Investigational Site
Bucharest, , Romania
Pfizer Investigational Site
Moscow, , Russia
Pfizer Investigational Site
Saint Petersburg, , Russia
Pfizer Investigational Site
Belgrade, , Serbia
Pfizer Investigational Site
Niš, , Serbia
Pfizer Investigational Site
Madrid, Madrid, Spain
Pfizer Investigational Site
Seville, Sevilla, Spain
Countries
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References
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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.
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.
Valentino LA, Rusen L, Elezovic I, Smith LM, Korth-Bradley JM, Rendo P. Multicentre, randomized, open-label study of on-demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects. Haemophilia. 2014 May;20(3):398-406. doi: 10.1111/hae.12344. Epub 2014 Jan 13.
Shafer F, Smith L, Vendetti N, Rendo P, Carr M. Lack of seasonal variation in bleeding and patient-assessed pain patterns in patients with haemophilia B receiving on-demand therapy. Haemophilia. 2014 May;20(3):349-53. doi: 10.1111/hae.12305. Epub 2013 Nov 29.
Related Links
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Other Identifiers
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B1821002
Identifier Type: OTHER
Identifier Source: secondary_id
3090A1-400
Identifier Type: -
Identifier Source: org_study_id