Hemophilia BNCT01154231

BeneFIX Drug Use Results Survey [All-Case Surveillance]

An observational study of Hemophilia B, run by Pfizer at 1 site in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

The survey is intended to investigate the following matters, etc. under the actual use status after marketing in all patients who are administered this drug for a certain period of time after the launch. 1. Occurrence status of adverse events 2. Factors that may influence the safety 3. Read more about this study

Who is it for?: Any age
Conditions the study is for: Hemophilia B
What is being tested?: Nonacog Alfa (Genetical Recombination)
How many people: 314 participants
Where: 1 siteJapan
Study dates: 29 Oct 2009to 27 Feb 2017
Phase: Observational
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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Study population: Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug.

Who may be able to join

All patients with hemophilia B scheduled to receive treatment with BeneFIX will be eligible for the surveillance.
No patient will be excluded because of prior inhibitor history; however, complete patient history and demographics will be collected.

Who cannot join

Patients not administered BeneFIX.

Trial locations and contacts

1 site in 1 country.

Japan1 site

Tokyo Medical University Hospital
Shinjuku-ku, Tokyo

Other trials in Japan

About this study

Official title: BENEFIX(REGISTERED) INTRAVENOUS 500 1000 2000 DRUG USE RESULTS SURVEY (ALL-CASE SURVEILLANCE)

Occurrence status of adverse events
Factors that may influence the safety
Efficacy In addition, the following occurrence statuses will be investigated as priority items of the survey: Incidence rate of inhibitor, reduction in drug, efficacy, Allergic reaction, and Thrombosis.

Read the detailed description

Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug.

Patients with hemophilia B are either patients for whom treatment was already started with a blood coagulation factor IX product ("Previously treated patients": hereinafter, "PTPs") or patients who have no history of treatment with a blood coagulation factor IX product in the past and for whom treatment will be started with this drug for the first time ("Previously untreated patients": hereinafter, "PUPs").

The definition of PUPs in this survey is "Patients in whom the total number of days of administration of a blood coagulation factor IX product other than this drug before administration of this drug was 3 exposure days (actual number of days of administration: hereinafter, "EDs") or less."

Conditions: Hemophilia B
Being tested: Nonacog Alfa (Genetical Recombination)
Keywords: BeneFIX Regulatory Post Marketing Commitment Plan
Medical subject headings: Hemophilia B

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Nonacog Alfa (Genetical Recombination)Drug
Also called BeneFIX
Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug.
Patients with hemophilia B are either patients for whom treatment was already started with a blood coagulation factor IX product ("Previously treated patients": hereinafter, "PTPs") or patients who have no history of treatment with a blood coagulation factor IX product in the past and for whom treatment will be started with this drug for the first time ("Previously untreated patients": hereinafter, "PUPs").
The definition of PUPs in this survey is "Patients in whom the total number of days of administration of a blood coagulation factor IX product other than this drug before administration of this drug was 3 exposure days (actual number of days of administration: hereinafter, "EDs") or less."

Study groups in NCT01154231
Study group	Role	What they receive
Nonacog Alfa (Genetical Recombination)		Not described Receives: Drug: Nonacog Alfa (Genetical Recombination)

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy
Annual bleeding rate (ABR) was calculated as the number of total bleeding events occured during prophylaxis period devided by the period (events/year). ABR for other replacement treatment period was also calculated to evaluate the differences between types of treatment. If the period used for ABR calculation was less or equal than 7 days, the relevant data was regarded as missing.
Measured: 2 years for PTPs, 1 year for PUPs
Number of Administrations Required for Hemostasis for Bleeding Events
Mean number of administrations for hemostasis in replacement therapy for bleeding events.
Measured: 2 years for PTPs, 1 year for PUPs

Secondary outcomes (1)

Subjective Evaluation of Each Therapeutic Administration for Bleeding Episodes
Propotion of subjects whose physicians evaluated the effect of BeneFIX for bleeding episodes as excellent or good was calculated.
Measured: 2 years for PTPs, 1 year for PUPs

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

29 Oct 200927 Feb 2017

First posted30 Jun 2010 (estimated)
Study start29 Oct 2009
Primary completion29 Aug 2014
Study completion27 Feb 2017

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.
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: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 314 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01154231
Sponsor's protocol ID: 3090X1-4415
Other IDs: B1821009
Status last verified: Jul 2025
First submitted: 28 Jun 2010
Last updated: 16 Jul 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: No: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia

All Hemophilia B trials

Study at a glance

From the registry record, last updated by the sponsor on 16 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: BENEFIX(REGISTERED) INTRAVENOUS 500 1000 2000 DRUG USE RESULTS SURVEY (ALL-CASE SURVEILLANCE)
ClinicalTrials.gov ID: NCT01154231
Acronym / sponsor ID: 3090X1-4415
Conditions: Hemophilia B
Keywords: BeneFIX Regulatory Post Marketing Commitment Plan
Study type: Observational
Recruitment status: Completed
Enrolment: 314 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: 29 Oct 2009 (actual)
Primary completion: 29 Aug 2014 (actual)
Study completion: 27 Feb 2017 (actual)
First posted: 30 Jun 2010 (estimated)
Results: Posted 11 Apr 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Occurrence status of adverse events
Factors that may influence the safety
Efficacy In addition, the following occurrence statuses will be investigated as priority items of the survey: Incidence rate of inhibitor, reduction in drug, efficacy, Allergic reaction, and Thrombosis.

Detailed description

Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug.

Study design

Observational model: Cohort
Time perspective: Prospective
Number of arms: 1

Arm	Type	Receives
Nonacog Alfa (Genetical Recombination)	–	Drug: Nonacog Alfa (Genetical Recombination)

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug.

Key inclusion criteria (2 listed)

All patients with hemophilia B scheduled to receive treatment with BeneFIX will be eligible for the surveillance.
No patient will be excluded because of prior inhibitor history; however, complete patient history and demographics will be collected.

Key exclusion criteria (1 listed)

Patients not administered BeneFIX.

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
Nonacog Alfa (Genetical Recombination)Also: BeneFIXPatients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug. Patients with hemophilia B are either patients for whom treatment was already started with a blood coagulation factor IX product ("Previously treated patients": hereinafter, "PTPs") or patients who have no history of treatment with a blood coagulation factor IX product in the past and for whom treatment will be started with this drug for the first time ("Previously untreated patients": hereinafter, "PUPs"). The definition of PUPs in this survey is "Patients in whom the total number of days of administration of a blood coagulation factor IX product other than this drug before administration of this drug was 3 exposure days (actual number of days of administration: hereinafter, "EDs") or less."	Drug	Nonacog Alfa (Genetical Recombination)

Primary outcome

Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy
Time frame: 2 years for PTPs, 1 year for PUPs
Annual bleeding rate (ABR) was calculated as the number of total bleeding events occured during prophylaxis period devided by the period (events/year). ABR for other replacement treatment period was also calculated to evaluate the differences between types of treatment. If the period used for ABR calculation was less or equal than 7 days, the relevant data was regarded as missing.
Number of Administrations Required for Hemostasis for Bleeding Events
Time frame: 2 years for PTPs, 1 year for PUPs
Mean number of administrations for hemostasis in replacement therapy for bleeding events.

1 secondary outcome

Subjective Evaluation of Each Therapeutic Administration for Bleeding Episodes
Time frame: 2 years for PTPs, 1 year for PUPs

Results and safety

Results posted to the registry on 11 Apr 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
BeneFIX (Nonacog Alfa)	314	312

Primary outcome: Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy

Time frame: 2 years for PTPs, 1 year for PUPs. Median (Inter-Quartile Range). Unit: Bleeding events/year

Measure	BeneFIX (Nonacog Alfa) n = 178
Value	2.00 (11.29)

Population analysed: Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.

Primary outcome: Number of Administrations Required for Hemostasis for Bleeding Events

Time frame: 2 years for PTPs, 1 year for PUPs. Mean (Full Range). Unit: administrations

Measure	BeneFIX (Nonacog Alfa) n = 112
Value	1.8 (4.18)

Population analysed: Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.

1 secondary outcome is reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BeneFIX (Nonacog Alfa)	5 of 312 (1.6%)	35 of 312 (11.2%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.
Individual participant dataSharing planned: No. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Related trials for Hemophilia B

Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia

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

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Hemophilia B, run by Pfizer at 1 site in Japan.

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 2 inclusion and 1 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 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Japan. 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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Apr 2017). 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?

Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy (time frame: 2 years for PTPs, 1 year for PUPs). The study lists 2 primary outcomes in all. 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 NCT01154231.

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 16 Jul 2025.