Hemophilia BNCT00835068

Post Marketing Observational Study of Reformulated BeneFIX

An observational study of Hemophilia B, run by Pfizer at 17 sites in France.

Completed

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

Trial summary

The primary objective of this observational study is to collect safety data on reformulated BeneFIX as prescribed in routine clinical practice conditions in France. The secondary objectives are to collect data on the clinical course of individuals treated with reformulated BeneFIX and on the ease of reformulated… Read more about this study

Who is it for?: Any age
Conditions the study is for: Hemophilia B
How many people: 58 participants
Where: 17 sitesFrance
Study dates: Jan 2009to Oct 2013
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: Hemophilia B patients already receiving or starting treatment with reformulated BeneFIX

Who may be able to join

Subjects with Hemophilia B already receiving or starting treatment with reformulated BeneFIX.
Subjects who have dated and signed the informed consent form.

Who cannot join

Ongoing treatment of Hemophilia B by a product other than reformulated BeneFIX.
Participation in the European prospective registry of patients with Hemophilia B treated with BeneFIX (Wyeth protocol 3090A-101039).

Trial locations and contacts

17 sites in 1 country.

City, postcode or hospital

17 sites in 1 country

France17 sites

Pfizer Investigational Site
Caen, 14033
Pfizer Investigational Site
Chamberry, Cedex, 73011
Pfizer Investigational Site
Clermont-Ferrand, 63003
Pfizer Investigational Site
Dijon, 21070
Pfizer Investigational Site
Le Chesnay, Cedex, 78157
Pfizer Investigational Site
leKremlin-Bicetre, 94275
Pfizer Investigational Site
Lyon, 69003
Pfizer Investigational Site
Marseille, 13385
Pfizer Investigational Site
Montmorency, 95160
Pfizer Investigational Site
Montpellier, 34 34295
Pfizer Investigational Site
Nantes, 44093
Pfizer Investigational Site
Paris, 75014
Pfizer Investigational Site
Paris, F-75015
Pfizer Investigational Site
Rouen, 76031
Pfizer Investigational Site
Saint-Priest-en-Jarez, 42277
Pfizer Investigational Site
Tours, 37044
Pfizer Investigational Site
Vandœuvre-lès-Nancy, 54511

Other trials in France

About this study

Official title: Post Marketing Observational Study Of Reformulated BeneFIX

Read the detailed description

No sampling

Conditions: Hemophilia B
Being tested: No intervention
Keywords: Hemophilia B, observational, post marketing study
Medical subject headings: Hemophilia B

What participants receive

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

No interventionOther
As it is a non interventional study, patient receives his usual treatment (BeneFIX) as determined by the physician

Study groups in NCT00835068
Study group	Role	What they receive
BeneFIX		Not described Receives: Other: No intervention

What is being measured

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

Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) Prior to Safety Amendment
A treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included SAEs as well as non-serious AEs which occurred prior to safety amendment. Prior to safety amendment, only AEs/SAEs deemed related to BeneFIX as per participating physician were collected.
Measured: Baseline up to Year 3.5
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Relationship After Safety Amendment
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. A treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug as per participating physician. AEs included SAEs as well as non-serious AEs which occurred after the safety amendment. After the safety amendment, all AEs/SAEs were collected irrespective of their relationship to BeneFIX.
Measured: Year 3.5 up to 4.75
Number of Participants With Events of Special Interest
Events of special interest included allergic reactions, red blood cell (RBC) agglutination phenomena, lack of efficacy/low recovery, thrombotic events and onset of factor IX (FIX) inhibitor. Participants may be represented in more than 1 category.
Measured: Baseline up to Year 4.75

Secondary outcomes (7)

Number of Bleeding Episodes
Number of bleeding episode during prophylaxis and on demand period were reported. All periods with at least one injection per week were considered as prophylaxis period. All prophylaxis periods of less than a month were reviewed and cross-checked with the treatment scheme planned at the previous visit to confirm if they were real prophylaxis periods or preventive injections periods. On demand treatment period included the total duration of follow up excluding duration of both prophylaxis and preventive injection treatment scheme. Efficacy population included only those participants who were previously treated with BeneFIX. Here, 'n' signifies participants evaluable for this measure during the specified treatment period. Participants may be represented in more than 1 category.
Measured: Baseline up to Year 4.75
Number of Bleeding Episodes Requiring Treatment by Injection
Number of injections (1, 2, 3 or greater than or equal to [>= 4]) required to treat the bleeding episodes during prophylaxis and on demand period were reported. All periods with at least one injection per week were considered as prophylaxis period. All prophylaxis periods of less than a month were reviewed and cross-checked with the treatment scheme planned at the previous visit to confirm if they were real prophylaxis periods or preventive injections periods. On demand treatment period included the total duration of follow up excluding duration of both prophylaxis and preventive injection treatment scheme.
Measured: Baseline up to Year 4.75
Total Consumption of BeneFIX
Total consumption of BeneFIX included consumption during prophylaxis, on demand, during bleeding episodes, preventive injections, surgeries or immune tolerance.
Measured: Baseline up to Year 4.75
Subjective Assessment of Efficacy by Participant
Participants assessed efficacy of BeneFIX as very good, good, moderate and bad at each follow-up visit. Results were summarized for the latest (most recent), worst and best assessment of BeneFIX done by the participant.
Measured: Baseline up to Year 4.75
Dose Per Injection of BeneFIX
Dose per injection during prophylaxis and on demand period were reported. All periods with at least one injection per week were considered as prophylaxis period. All prophylaxis periods of less than a month were reviewed and cross-checked with the treatment scheme planned at the previous visit to confirm if they were real prophylaxis periods or preventive injections periods. On demand treatment period included the total duration of follow up excluding duration of both prophylaxis and preventive injection treatment scheme. Participants may be represented in more than 1 category.
Measured: Baseline up to Year 4.75
Subjective Assessment of Efficacy by Physician
Participating physician assessed efficacy of BeneFIX as very good, good, moderate and bad at follow-up visit. Results were summarized for the latest (most recent), worst and best assessment of BeneFIX done by the physician.
Measured: Baseline up to Year 4.75
Subjective Assessment of Ease of Use by Participant
Participants assessed ease of use of BeneFIX as very good, good, moderate and bad at each follow-up visit. Results were summarized for the latest (most recent), worst and best assessment of BeneFIX done by the participant.
Measured: Baseline up to Year 4.75

Timeline

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

Jan 2009Oct 2013

First posted3 Feb 2009 (estimated)
Study startJan 2009
Primary completionOct 2013
Study completionOct 2013

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: 58 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00835068
Sponsor's protocol ID: 3090X1-4409
Other IDs: B1821007
Status last verified: Sep 2014
First submitted: 2 Feb 2009
Last updated: 17 Sep 2014
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

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 17 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Post Marketing Observational Study Of Reformulated BeneFIX
ClinicalTrials.gov ID: NCT00835068
Acronym / sponsor ID: 3090X1-4409
Conditions: Hemophilia B
Keywords: Hemophilia B, observational, post marketing study
Study type: Observational
Recruitment status: Completed
Enrolment: 58 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Jan 2009
Primary completion: Oct 2013 (actual)
Study completion: Oct 2013 (actual)
First posted: 3 Feb 2009 (estimated)
Results: Posted 17 Sep 2014

Clinical summary

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

Detailed description

No sampling

Study design

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

Arm	Type	Receives
BeneFIX	–	Other: No intervention

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: Hemophilia B patients already receiving or starting treatment with reformulated BeneFIX

Key inclusion criteria (2 listed)

Subjects with Hemophilia B already receiving or starting treatment with reformulated BeneFIX.
Subjects who have dated and signed the informed consent form.

Key exclusion criteria (2 listed)

Ongoing treatment of Hemophilia B by a product other than reformulated BeneFIX.
Participation in the European prospective registry of patients with Hemophilia B treated with BeneFIX (Wyeth protocol 3090A-101039).

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
No interventionAs it is a non interventional study, patient receives his usual treatment (BeneFIX) as determined by the physician	Other	BeneFIX

Primary outcome

Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) Prior to Safety Amendment
Time frame: Baseline up to Year 3.5
A treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included SAEs as well as non-serious AEs which occurred prior to safety amendment. Prior to safety amendment, only AEs/SAEs deemed related to BeneFIX as per participating physician were collected.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Relationship After Safety Amendment
Time frame: Year 3.5 up to 4.75
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. A treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug as per participating physician. AEs included SAEs as well as non-serious AEs which occurred after the safety amendment. After the safety amendment, all AEs/SAEs were collected irrespective of their relationship to BeneFIX.
Number of Participants With Events of Special Interest
Time frame: Baseline up to Year 4.75
Events of special interest included allergic reactions, red blood cell (RBC) agglutination phenomena, lack of efficacy/low recovery, thrombotic events and onset of factor IX (FIX) inhibitor. Participants may be represented in more than 1 category.

7 secondary outcomes

Number of Bleeding Episodes
Time frame: Baseline up to Year 4.75
Number of Bleeding Episodes Requiring Treatment by Injection
Time frame: Baseline up to Year 4.75
Total Consumption of BeneFIX
Time frame: Baseline up to Year 4.75
Subjective Assessment of Efficacy by Participant
Time frame: Baseline up to Year 4.75
Dose Per Injection of BeneFIX
Time frame: Baseline up to Year 4.75
Subjective Assessment of Efficacy by Physician
Time frame: Baseline up to Year 4.75
Subjective Assessment of Ease of Use by Participant
Time frame: Baseline up to Year 4.75

Results and safety

Results posted to the registry on 17 Sep 2014, 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 (Previously Treated Participants)	57	47
BeneFIX (Previously Untreated Participants)	1	0

Primary outcome: Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) Prior to Safety Amendment

Time frame: Baseline up to Year 3.5. Number. Unit: participants

Measure	BeneFIX (Previously Treated Participants) n = 57	BeneFIX (Previously Untreated Participants) n = 1
Treatment-related AE	1	1
Treatment-related SAE	0	1

Population analysed: Safety population included all participants who received at least one dose of BeneFIX.

Primary outcome: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Relationship After Safety Amendment

Time frame: Year 3.5 up to 4.75. Number. Unit: participants

Measure	BeneFIX (Previously Treated Participants) n = 57	BeneFIX (Previously Untreated Participants) n = 0
AE	26	–
SAE	10	–
Treatment-related AE	1	–
Treatment-related SAE	0	–

Population analysed: Safety population included all participants who received at least one dose of BeneFIX. Previously untreated participants were not evaluable for this outcome measure due to discontinuation prior to safety amendment.

Primary outcome: Number of Participants With Events of Special Interest

Time frame: Baseline up to Year 4.75. Number. Unit: participants

Measure	BeneFIX (Previously Treated Participants) n = 57	BeneFIX (Previously Untreated Participants) n = 1
FIX inhibitor	0	1
Allergic reactions	0	0
RBC agglutination	0	0
Lack of efficacy/ low recovery	0	0
Thrombotic event	0	0

Population analysed: Safety population included all participants who received at least one dose of BeneFIX.

7 secondary outcomes are 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 (Previously Treated Participants)	10 of 57 (17.5%)	21 of 57 (36.8%)	Not reported
BeneFIX (Previously Untreated Participants)	1 of 1 (100%)	0 of 1 (0%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2014.
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.

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

17 sites 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 17 sites in France.

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 2 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 17 Sep 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 17 sites in France. 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 17 Sep 2014). 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 Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) Prior to Safety Amendment (time frame: Baseline up to Year 3.5). The study lists 3 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 NCT00835068.

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 17 Sep 2014.