Hemophilia BNCT00714415

Registry For Patients Treated With BeneFix In Usual Care Setting In Germany

An observational study of Hemophilia B, run by Pfizer at 21 sites in 2 countries.

Completed

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

Trial summary

The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany. Read more about this study

Who is it for?: Any age
Conditions the study is for: Hemophilia B
What is being tested?: BeneFIX
How many people: 80 participants
Where: 21 sitesin 2 countries
Study dates: Jan 2008to Oct 2016
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

Who may be able to join

Patients with hemophilia B already receiving or starting treatment with reformulated BeneFIX®.

Who cannot join

Patients with hemophilia B treated with a product other than BeneFIX®.
Inclusion in the ongoing prospective registry of European hemophilia B patients using BeneFIX®.

Trial locations and contacts

21 sites in 2 countries.

Country

City, postcode or hospital

21 sites in 2 countries

Germany20 sites

Vivantes Klinikum im Friedrichshain
Berlin, 10249
Charite Campus Virchow-Klinikum, Padiatrie mit S. Hamatologie und Onkologie
Berlin, 13353
Kinder- und Jugendarzt-Praxis Blaubeuren
Blaubeuren Abbey, 89143
Institute of Experimental Haematology and Transfusion Medicine
Bonn, 53127
Praxis fur Kinder- und Jugendmedizin, Homoopathie
Brannenburg, 83098
Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
Bremen, 28177
Gemeinschaftspraxis fuer Haematologie und Onkologie
Cologne, 50677
Klinikum Delmehorst gGmbH, Padiatrie
Delmenhorst, 27753
CRC Coagulation Research Centre GmbH
Duisburg, 47051
Universitaetsklinikum Duesseldorf, Klinik f. Kinder-Onkologie, Haematologie u. Klinische Immunologie
Düsseldorf, 40225
Klinikum der Martin-Luther-Universitaet Halle-Wittenberg
Halle, 06120
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246
Universitaetsklinikum Eppendorf
Hamburg, 20251
Werlhof-Institut für Haemostaseologie GmbH
Hanover, Lower Saxony, 30159
SRH Kurpfalzkrankenhaus Heidelberg
Heidelberg, 69123
Klinikum Memmingen, Kinderklinik
Memmingen, 87700
Sonnengesundheitszentrum
München, Bavaria, 80336
Universitaetskinderklinik und Poliklinik im Dr. von Haunerschen
München, 80337
Institut für Thrombophilie und Hämostaseologie
Münster, North Rhine-Westphalia, 48143
Universitaetsklinik fuer Kinder- und Jugendmedizin
Tübingen, 72076

Other trials in Germany

Austria1 site

Allgemeines Krankenhaus Linz, Kinderklinik
Linz, 4020

Other trials in Austria

About this study

Official title: Pharmacovigilance Evaluation Of Benefix (Registered) In Germany And Austria

The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.

Read the detailed description

Non-interventional study: subjects to be selected according to the usual clinical practice of their physician

Conditions: Hemophilia B
Being tested: 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.

BeneFIXDrug
Patients will be treated in accordance with the requirements of the labeling of BeneFIX in Germany. The dosage and duration of therapy is to be determined by the physician to meet the patients' individual needs for treatment.

Study groups in NCT00714415
Study group	Role	What they receive
A		Patients with Hemophilia B Receives: Drug: BeneFIX

What is being measured

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

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. 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 or incapacity; cancer; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to last visit (up to 8.7 years) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious.
Measured: Baseline until last visit (up to 8.7 years)
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. 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; cancer; congenital anomaly. AEs included both serious and non-serious. Relatedness to BeneFIX was assessed by the investigator.
Measured: Baseline until last visit (up to 8.7 years)
Number of Participants With Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
FIX inhibitor development was defined as measured inhibitor titer of greater than (>) 0.6 Bethesda Units (BU) using the Nijmegen-modified Bethesda assay.
Measured: Baseline until last visit (up to 8.7 years)
Number of Participants With Adverse Events (AEs) of Special Interest
An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. Adverse Events of special interest included allergic reactions, less than expected therapeutic effect (LETE) of drug, lack of efficacy/low recovery, erythrocyte agglutination in tube or syringe red blood cell (RBC) agglutination phenomena and thrombogenicity.
Measured: Baseline until last visit (up to 8.7 years)
Investigator Assessment of Treatment Tolerability of Participants
Investigator assessed the tolerability of participants and categorized as very good, good, moderate and poor.
Measured: End of study visit (any time up to 8.7 years)
Participant Assessment of Treatment Tolerability
Participants evaluated their treatment (BeneFIX) tolerability and rated it in 4 categories as very good, good, moderate and poor.
Measured: End of study visit (any time up to 8.7 years)

Secondary outcomes (8)

Mean Total Number of Bleeding Episodes in Participants
Participants documented all bleeding episodes in a diary during the study.
Measured: Baseline until last visit (up to 8.7 years)
Mean Total Number of Bleeding Episodes Per Year in Participants
Participants documented all bleeding episodes in a diary during the study. Mean total number of bleeding episodes per year was calculated by mean total number of bleeding episodes divided by duration of observation period (in years) for bleeding documentation.
Measured: Baseline until last visit (up to 8.7 years)
Number of Participants With Change From Baseline Status in Number of Days Missed From School or Work
Change from baseline status in days missed from school or work was categorized in 3 categories: Improvement, unchanged and worsening. Improvement was defined as a decrease in number of days missed by participants from school/work as compared to baseline; worsening was defined as an increase in number of days missed by participants from school/work as compared to baseline; unchanged was defined as no change in number of days missed by participants from school/work as compared to baseline. In this outcome measure, number of participants with change from baseline status (as improved, worsen, unchanged) in days missed from school/work were reported.
Measured: Baseline, up to 8.7 years
Investigator Assessment of Treatment Efficacy of Participants
Investigator evaluated the efficacy of BeneFIX in participants and rated it in 4 categories as very good, good, moderate and poor.
Measured: End of study visit (any time up to 8.7 years)
Investigator Assessment of Treatment Handling of Participants
Investigator evaluated the handling (administration) of BeneFIX by participants and rated it in 4 categories as very good, good, moderate and poor.
Measured: End of study visit (any time up to 8.7 years)
Assessment of Treatment Efficacy by the Participants
Participants evaluated the efficacy of BeneFIX and rated it in 4 categories as very good, good, moderate and poor.
Measured: End of study visit (any time up to 8.7 years)
Assessment of Treatment Handling by the Participants
Participants evaluated the handling (administration) of BeneFIX and rated it in 4 categories as very good, good, moderate and poor.
Measured: End of study visit (any time up to 8.7 years)
Investigator Assessment of Treatment Satisfaction of Participants
Investigator evaluated the participant's satisfaction of treatment with BeneFIX and rated it in 4 categories as very satisfied, satisfied, unsatisfied and very unsatisfied.
Measured: Baseline up to 8.7 years

Timeline

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

Jan 2008Oct 2016

First posted14 Jul 2008 (estimated)
Study startJan 2008
Primary completionOct 2016
Study completionOct 2016

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: 80 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00714415
Sponsor's protocol ID: 3090A1-4406
Other IDs: B1821011
Status last verified: Sep 2018
First submitted: 8 Jul 2008
Last updated: 25 Oct 2018
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 25 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Pharmacovigilance Evaluation Of Benefix (Registered) In Germany And Austria
ClinicalTrials.gov ID: NCT00714415
Acronym / sponsor ID: 3090A1-4406
Conditions: Hemophilia B
Study type: Observational
Recruitment status: Completed
Enrolment: 80 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Jan 2008 (actual)
Primary completion: Oct 2016 (actual)
Study completion: Oct 2016 (actual)
First posted: 14 Jul 2008 (estimated)
Results: Posted 24 Sep 2018

Clinical summary

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

The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.

Detailed description

Non-interventional study: subjects to be selected according to the usual clinical practice of their physician

Study design

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

Arm	Type	Receives
A	–	Drug: BeneFIXPatients with Hemophilia B

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: Patients with hemophilia B

Key inclusion criteria (1 listed)

Patients with hemophilia B already receiving or starting treatment with reformulated BeneFIX®.

Key exclusion criteria (2 listed)

Patients with hemophilia B treated with a product other than BeneFIX®.
Inclusion in the ongoing prospective registry of European hemophilia B patients using 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
BeneFIXPatients will be treated in accordance with the requirements of the labeling of BeneFIX in Germany. The dosage and duration of therapy is to be determined by the physician to meet the patients' individual needs for treatment.	Drug	A

Primary outcome

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline until last visit (up to 8.7 years)
An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. 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 or incapacity; cancer; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to last visit (up to 8.7 years) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious.
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline until last visit (up to 8.7 years)
Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. 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; cancer; congenital anomaly. AEs included both serious and non-serious. Relatedness to BeneFIX was assessed by the investigator.
Number of Participants With Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
Time frame: Baseline until last visit (up to 8.7 years)
FIX inhibitor development was defined as measured inhibitor titer of greater than (>) 0.6 Bethesda Units (BU) using the Nijmegen-modified Bethesda assay.
Number of Participants With Adverse Events (AEs) of Special Interest
Time frame: Baseline until last visit (up to 8.7 years)
An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. Adverse Events of special interest included allergic reactions, less than expected therapeutic effect (LETE) of drug, lack of efficacy/low recovery, erythrocyte agglutination in tube or syringe red blood cell (RBC) agglutination phenomena and thrombogenicity.
Investigator Assessment of Treatment Tolerability of Participants
Time frame: End of study visit (any time up to 8.7 years)
Investigator assessed the tolerability of participants and categorized as very good, good, moderate and poor.
Participant Assessment of Treatment Tolerability
Time frame: End of study visit (any time up to 8.7 years)
Participants evaluated their treatment (BeneFIX) tolerability and rated it in 4 categories as very good, good, moderate and poor.

8 secondary outcomes

Mean Total Number of Bleeding Episodes in Participants
Time frame: Baseline until last visit (up to 8.7 years)
Mean Total Number of Bleeding Episodes Per Year in Participants
Time frame: Baseline until last visit (up to 8.7 years)
Number of Participants With Change From Baseline Status in Number of Days Missed From School or Work
Time frame: Baseline, up to 8.7 years
Investigator Assessment of Treatment Efficacy of Participants
Time frame: End of study visit (any time up to 8.7 years)
Investigator Assessment of Treatment Handling of Participants
Time frame: End of study visit (any time up to 8.7 years)
Assessment of Treatment Efficacy by the Participants
Time frame: End of study visit (any time up to 8.7 years)
Assessment of Treatment Handling by the Participants
Time frame: End of study visit (any time up to 8.7 years)
Investigator Assessment of Treatment Satisfaction of Participants
Time frame: Baseline up to 8.7 years

Results and safety

Results posted to the registry on 24 Sep 2018, 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: On-Demand	25	23
BeneFIX: Prophylaxis	55	55

Primary outcome: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: Baseline until last visit (up to 8.7 years). Number. Unit: participants

Measure	BeneFIX: On Demand n = 25	BeneFIX: Prophylaxis n = 55
AEs	19	49
SAEs	8	29

Population analysed: Safety analysis set included all participants with informed consent and treated with at least 1 dose of Benefix.

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

Time frame: Baseline until last visit (up to 8.7 years). Number. Unit: participants

Measure	BeneFIX: On Demand n = 25	BeneFIX: Prophylaxis n = 55
AEs	2	8
SAEs	1	2

Population analysed: Safety analysis set included all participants with informed consent and treated with at least 1 dose of Benefix.

Primary outcome: Number of Participants With Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay

Time frame: Baseline until last visit (up to 8.7 years). Number. Unit: participants

Measure	BeneFIX: On Demand n = 25	BeneFIX: Prophylaxis n = 55
Value	1	3

Population analysed: Safety analysis set included all participants with informed consent and treated with at least 1 dose of Benefix.

Primary outcome: Number of Participants With Adverse Events (AEs) of Special Interest

Time frame: Baseline until last visit (up to 8.7 years). Number. Unit: participants

Measure	BeneFIX: On Demand n = 25	BeneFIX: Prophylaxis n = 55
Value	0	0

Population analysed: Safety analysis set included all participants with informed consent and treated with at least 1 dose of Benefix.

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: Baseline until last visit (up to 8.7 years). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BeneFIX: On Demand	8 of 25 (32%)	18 of 25 (72%)	Not reported
BeneFIX: Prophylaxis	29 of 55 (52.7%)	46 of 55 (83.6%)	Not reported

Limitations and caveats (from the sponsor): Prioritization of outcome measures as primary and secondary was based on the study team's discretion, as it was not specified in source documents.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 25 Oct 2018.
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
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Not yet recruitingPhase 1
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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

21 sites in 2 countries: 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 21 sites in 2 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 1 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 25 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 21 sites in Germany, Austria. 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 24 Sep 2018). 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-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Baseline until last visit (up to 8.7 years)). The study lists 6 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 NCT00714415.

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 25 Oct 2018.