Hemophilia BPhase 3NCT00364182
Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B
A Phase 3 study testing Recombinant Coagulation Factor IX (BeneFIX) in people with Hemophilia B, run by Pfizer at 19 sites in 9 countries.
Trial summary
This study is designed to evaluate BeneFIX infused as prophylaxis regimens, compared with BeneFIX administered as an on-demand regimen only. In the trial, subjects will participate in 4 study periods. Read more about this study
- Who is it for?
- Ages 6 to 65 years, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- Recombinant Coagulation Factor IX (BeneFIX)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 19 sitesin 9 countries
- Study dates
- May 2007to Oct 2010
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Documented history of moderately severe or severe hemophilia B (FIX:C less than or equal to 2%)
- 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.
Who cannot join
- Subjects currently utilizing FIX primary prophylaxis.
- 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.
Show all 10Show fewer
- 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.
Trial locations and contacts
19 sites in 9 countries.
19 sites in 9 countries
United States5 sites
Pfizer Investigational Site
Chicago, Illinois, 60612-3833
Pfizer Investigational Site
Chicago, Illinois, 60614
Pfizer Investigational Site
Houston, Texas, 77030-4009
Pfizer Investigational Site
New Brunswick, New Jersey, 08903-0019
Pfizer Investigational Site
Phoenix, Arizona, 85016
Canada3 sites
Pfizer Investigational Site
Edmonton, Alberta, T6G 2B7
Pfizer Investigational Site
Edmonton, Alberta, T6G 2C8
Pfizer Investigational Site
Ottawa, Ontario, K1H 8L6
Italy2 sites
Pfizer Investigational Site
Castelfranco Veneto (TV), 31033
Pfizer Investigational Site
Coppito (AQ), 67100
Russia2 sites
Pfizer Investigational Site
Moscow, 125167
Pfizer Investigational Site
Saint Petersburg, 191186
Serbia2 sites
Pfizer Investigational Site
Belgrade, 11000
Pfizer Investigational Site
Niš, 18000
Spain2 sites
Pfizer Investigational Site
Madrid, Madrid, 28046
Pfizer Investigational Site
Seville, Sevilla, 41013
About this study
Official title: A Multicenter, Open-Label Study To Compare On-Demand Treatment With 2 Prophylaxis Regimens of Recombinant Coagulation Factor IX (BeneFIX) Reformulated Drug Product (rFIX-R) In Subjects With Severe Hemophilia B
This study is designed to evaluate BeneFIX infused as prophylaxis regimens, compared with BeneFIX administered as an on-demand regimen only. In the trial, subjects will participate in 4 study periods. The first period is a 16-week on-demand treatment, during which subjects will utilize BeneFIX to treat bleeding events episodically only as they occur (i.e., on-demand treatment). At the end of this period, subjects will be randomly assigned to 1 of 2 BeneFIX prophylaxis regimens: either 100 IU/kg once weekly or 50 IU/kg twice weekly, and followed for 16 weeks. At the end of this period, subjects will use on-demand treatment for 8 weeks. The purpose of this 8-week period is to mitigate the potential carry-over effect of the prior prophylaxis regimen on bleeding frequency. Following this 8-week on-demand treatment, subjects will cross-over and receive the alternate study prophylactic regimen for 16 weeks.
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.
Read more
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.
- Conditions
- Hemophilia B
- Being tested
- Recombinant Coagulation Factor IX (BeneFIX)
- Keywords
- Hemophilia B, BeneFIX
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Coagulation Factor IX (BeneFIX)Drug
100 IU/kg once weekly then crossover to 50 IU/kg twice weekly
Recombinant Coagulation Factor IX (BeneFIX)Drug
50 IU/kg twice weekly then crossover to 100 IU/kg once weekly
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Not described Receives: Drug: Recombinant Coagulation Factor IX (BeneFIX) |
| B | Experimental | Not described Receives: Drug: Recombinant Coagulation Factor IX (BeneFIX) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Number of Bleeding Episodes
Annualized bleed rate (ABR) or number of bleeds per year derived for each participant for each treatment regimen by using the following formula: ABR = number of bleeds / (days on treatment regimen / 365.25)
Measured: Baseline up to Week 56
Secondary outcomes (6)
Amount of Sleep Measured by Sleep Diary After Hemarthrosis
For each bleeding event, a diary was filled out that night and the subsequent night. Questions included: How long do you think you slept last night? Reported as average duration of sleep during study.
Measured: 24 and 48 hours post-bleed
Quality of Sleep Measured by Sleep Diary After Hemarthrosis
For each bleeding event, a diary was filled out that night and the subsequent night. Questions included: How would you describe the quality of your sleep last night? 1=Very Good, 2=Good, 3=Fair, 4=Poor, 5=Very Poor. Reported as quality of sleep during study.
Measured: 24 and 48 hours post-bleed
Acute Pain After Hemarthrosis
For each bleeding event, a diary was filled out that night and the subsequent night and included a Brief Pain Inventory (BPI): self-reported scale that measured severity of pain experienced over the past 24 hours. Questions included How much pain right now? 0 (no pain) to 10 (pain as severe as you can imagine).
Measured: 24 and 48 hours post-bleed
Health-Related Productivity Questionnaire (HRPQ) Score: Hours Lost From Work or School at 24 Hours Post-bleed
HRPQ: self-reported scale that measured for each bleed event, hours lost from work, school and housework because of hemophilia and its treatments. Mean and standard deviations calculated from measured values.
Measured: 24 hours post-bleed
HRPQ Score: Hours Lost From Work or School at 48 Hours Post-bleed
HRPQ: self-reported scale that measured for each bleed event, hours lost from work, school and housework because of hemophilia and its treatments. Mean and standard deviations calculated from measured values.
Measured: 48 hours post-bleed
36-Item Short-Form Health Survey (SF-36): Physical Functioning Domain
SF-36: standardized survey evaluating 8 aspects of functional health and well being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, and mental health. The physical functioning domain score was an average of the individual physical functioning question scores across all time points, which was scaled 0-100 (100=highest level of functioning).
Measured: Weeks 16, 32, and 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2006 (estimated)
- Study startMay 2007
- Primary completionOct 2010
- Study completionOct 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00364182
- Sponsor's protocol ID
- 3090A1-400
- Other IDs
- B1821002
- Status last verified
- Aug 2011
- First submitted
- 14 Aug 2006
- Last updated
- 4 Oct 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 40750723Rendo 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.
PubMed 26196195Valentino 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.
PubMed 24418368Shafer 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.
PubMed 24286226
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label Study To Compare On-Demand Treatment With 2 Prophylaxis Regimens of Recombinant Coagulation Factor IX (BeneFIX) Reformulated Drug Product (rFIX-R) In Subjects With Severe Hemophilia B
- ClinicalTrials.gov ID
- NCT00364182
- Acronym / sponsor ID
- 3090A1-400
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B, BeneFIX
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- May 2007
- Primary completion
- Oct 2010 (actual)
- Study completion
- Oct 2010 (actual)
- First posted
- 15 Aug 2006 (estimated)
- Results
- Posted 23 Aug 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to evaluate BeneFIX infused as prophylaxis regimens, compared with BeneFIX administered as an on-demand regimen only. In the trial, subjects will participate in 4 study periods. The first period is a 16-week on-demand treatment, during which subjects will utilize BeneFIX to treat bleeding events episodically only as they occur (i.e., on-demand treatment). At the end of this period, subjects will be randomly assigned to 1 of 2 BeneFIX prophylaxis regimens: either 100 IU/kg once weekly or 50 IU/kg twice weekly, and followed for 16 weeks. At the end of this period, subjects will use on-demand treatment for 8 weeks. The purpose of this 8-week period is to mitigate the potential carry-over effect of the prior prophylaxis regimen on bleeding frequency. Following this 8-week on-demand treatment, subjects will cross-over and receive the alternate study prophylactic regimen for 16 weeks.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: Recombinant Coagulation Factor IX (BeneFIX) |
| B | Experimental | Drug: Recombinant Coagulation Factor IX (BeneFIX) |
Who may qualify
- Ages
- 6 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Documented history of moderately severe or severe hemophilia B (FIX:C less than or equal to 2%)
- 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.
Key exclusion criteria (10 listed)
- Subjects currently utilizing FIX primary prophylaxis.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant Coagulation Factor IX (BeneFIX)100 IU/kg once weekly then crossover to 50 IU/kg twice weekly | Drug | A |
| Recombinant Coagulation Factor IX (BeneFIX)50 IU/kg twice weekly then crossover to 100 IU/kg once weekly | Drug | B |
Primary outcome
Annualized Number of Bleeding Episodes
Time frame: Baseline up to Week 56
Annualized bleed rate (ABR) or number of bleeds per year derived for each participant for each treatment regimen by using the following formula: ABR = number of bleeds / (days on treatment regimen / 365.25)
6 secondary outcomes
Amount of Sleep Measured by Sleep Diary After Hemarthrosis
Time frame: 24 and 48 hours post-bleed
Quality of Sleep Measured by Sleep Diary After Hemarthrosis
Time frame: 24 and 48 hours post-bleed
Acute Pain After Hemarthrosis
Time frame: 24 and 48 hours post-bleed
Health-Related Productivity Questionnaire (HRPQ) Score: Hours Lost From Work or School at 24 Hours Post-bleed
Time frame: 24 hours post-bleed
HRPQ Score: Hours Lost From Work or School at 48 Hours Post-bleed
Time frame: 48 hours post-bleed
36-Item Short-Form Health Survey (SF-36): Physical Functioning Domain
Time frame: Weeks 16, 32, and 56
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40750723Wojciechowski 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.
- Derived from this study: PubMed 26196195Rendo 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.
- Derived from this study: PubMed 24418368Valentino 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.
- Derived from this study: PubMed 24286226Shafer 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.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
19 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Recombinant Coagulation Factor IX (BeneFIX) in people with Hemophilia B, run by Pfizer at 19 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Oct 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Canada, Italy, Russia, Serbia and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Aug 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Number of Bleeding Episodes (time frame: Baseline up to Week 56). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00364182, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Oct 2011; AzTrials last refreshed its copy on 26 Oct 2026.