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CompletedNCT00714415Updated Oct 25, 2018Results posted

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

An observational study in Hemophilia B, sponsored by Pfizer. Completed at 21 sites in 2 countries. Per ClinicalTrials.gov, last updated 2018-10-25.

Sponsored by Pfizer · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
80
Sex
All
01

Study 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 the detailed description

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

02

Conditions studied

  • Hemophilia B

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03

In context

Hemophilia B

271 studies on the registry are indexed under Hemophilia B; 51 are open to participants now.

This study's enrollment of 80 is below the median of 91 across 105 observational studies indexed under Hemophilia B.

Browse Hemophilia B studies →

Lead sponsor

Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.

Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Patients with hemophilia B

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

  • Patients with hemophilia B treated with a product other than BeneFIX®.
  • Inclusion in the ongoing prospective registry of European hemophilia B patients using BeneFIX®.
05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
80 participants (actual)
Patient registry
No

Groups and cohorts

  • A

    Patients with Hemophilia B

    Drug: BeneFIX

Interventions

  • DrugBeneFIX

    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.

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline until last visit (up to 8.7 years)

  2. 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.

    Time frame: Baseline until last visit (up to 8.7 years)

  3. 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.

    Time frame: Baseline until last visit (up to 8.7 years)

  4. 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.

    Time frame: Baseline until last visit (up to 8.7 years)

  5. Investigator Assessment of Treatment Tolerability of Participants

    Investigator assessed the tolerability of participants and categorized as very good, good, moderate and poor.

    Time frame: End of study visit (any time up to 8.7 years)

  6. Participant Assessment of Treatment Tolerability

    Participants evaluated their treatment (BeneFIX) tolerability and rated it in 4 categories as very good, good, moderate and poor.

    Time frame: End of study visit (any time up to 8.7 years)

Secondary outcomes

  1. Mean Total Number of Bleeding Episodes in Participants

    Participants documented all bleeding episodes in a diary during the study.

    Time frame: Baseline until last visit (up to 8.7 years)

  2. 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.

    Time frame: Baseline until last visit (up to 8.7 years)

  3. 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.

    Time frame: Baseline, up to 8.7 years

  4. 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.

    Time frame: End of study visit (any time up to 8.7 years)

  5. 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.

    Time frame: End of study visit (any time up to 8.7 years)

  6. 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.

    Time frame: End of study visit (any time up to 8.7 years)

  7. 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.

    Time frame: End of study visit (any time up to 8.7 years)

  8. 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.

    Time frame: Baseline up to 8.7 years

07

Results

Posted Sep 24, 2018
Limitations and caveats
Prioritization of outcome measures as primary and secondary was based on the study team's discretion, as it was not specified in source documents.

Participant flow

Participant flow — Overall Study
MilestoneBeneFIX: On-DemandBeneFIX: Prophylaxis
Started2555
Completed2355
Not completed20
Withdrew: Lack of efficacy10
Withdrew: Death10

Outcome measures

PrimaryNumber 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.

Time frame:
Baseline until last visit (up to 8.7 years)
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
AEs1949
SAEs829
PrimaryNumber 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.

Time frame:
Baseline until last visit (up to 8.7 years)
Reported as:
Number · participants
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
AEs28
SAEs12
PrimaryNumber 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.

Time frame:
Baseline until last visit (up to 8.7 years)
Reported as:
Number · participants
Number of Participants With Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Number of Participants With Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay13
PrimaryNumber 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.

Time frame:
Baseline until last visit (up to 8.7 years)
Reported as:
Number · participants
Number of Participants With Adverse Events (AEs) of Special Interest
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Number of Participants With Adverse Events (AEs) of Special Interest00
PrimaryInvestigator Assessment of Treatment Tolerability of Participants

Investigator assessed the tolerability of participants and categorized as very good, good, moderate and poor.

Time frame:
End of study visit (any time up to 8.7 years)
Reported as:
Number · participants
Investigator Assessment of Treatment Tolerability of Participants
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Very good1635
Good718
Moderate01
Poor00
PrimaryParticipant Assessment of Treatment Tolerability

Participants evaluated their treatment (BeneFIX) tolerability and rated it in 4 categories as very good, good, moderate and poor.

Time frame:
End of study visit (any time up to 8.7 years)
Reported as:
Number · participants
Participant Assessment of Treatment Tolerability
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Very good1637
Good614
Moderate01
Poor00
SecondaryMean Total Number of Bleeding Episodes in Participants

Participants documented all bleeding episodes in a diary during the study.

Time frame:
Baseline until last visit (up to 8.7 years)
Reported as:
Mean · bleeding episodes
Mean Total Number of Bleeding Episodes in Participants
bleeding episodesBeneFIX: On DemandBeneFIX: Prophylaxis
Mean Total Number of Bleeding Episodes in Participants16.7 ± 21.217.6 ± 23.1
SecondaryMean 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.

Time frame:
Baseline until last visit (up to 8.7 years)
Reported as:
Mean · bleeding episodes per year
Mean Total Number of Bleeding Episodes Per Year in Participants
bleeding episodes per yearBeneFIX: On DemandBeneFIX: Prophylaxis
Mean Total Number of Bleeding Episodes Per Year in Participants4.7 ± 4.74.2 ± 4.3
SecondaryNumber 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.

Time frame:
Baseline, up to 8.7 years
Reported as:
Number · participants
Number of Participants With Change From Baseline Status in Number of Days Missed From School or Work
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Improved47
Unchanged920
Worsened27
SecondaryInvestigator 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.

Time frame:
End of study visit (any time up to 8.7 years)
Reported as:
Number · participants
Investigator Assessment of Treatment Efficacy of Participants
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Very good1024
Good1130
Moderate10
Poor10
SecondaryInvestigator 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.

Time frame:
End of study visit (any time up to 8.7 years)
Reported as:
Number · participants
Investigator Assessment of Treatment Handling of Participants
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Very good1334
Good1020
Moderate00
Poor00
SecondaryAssessment 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.

Time frame:
End of study visit (any time up to 8.7 years)
Reported as:
Number · participants
Assessment of Treatment Efficacy by the Participants
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Very good1127
Good1025
Moderate00
Poor10
SecondaryAssessment 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.

Time frame:
End of study visit (any time up to 8.7 years)
Reported as:
Number · participants
Assessment of Treatment Handling by the Participants
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Very good1337
Good815
Moderate10
Poor00
SecondaryInvestigator 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.

Time frame:
Baseline up to 8.7 years
Reported as:
Number · participants
Investigator Assessment of Treatment Satisfaction of Participants
participantsBeneFIX: On DemandBeneFIX: Prophylaxis
Very satisfied1227
Satisfied927
Unsatisfied10
Very unsatisfied10

Adverse events

Collected over Baseline until last visit (up to 8.7 years). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
BeneFIX: On Demand—8/25 (32%)18/25 (72%)
BeneFIX: Prophylaxis—29/55 (52.7%)46/55 (83.6%)
Most frequent serious events
Showing 10 of 104
Most frequent serious events
EventBeneFIX: On DemandBeneFIX: Prophylaxis
AppendicitisInfections and infestations0/253/55
GastritisGastrointestinal disorders1/250/55
Gastrooesophageal reflux diseaseGastrointestinal disorders1/250/55
Condition aggravatedGeneral disorders1/250/55
PainGeneral disorders1/250/55
Pilonidal cystInfections and infestations1/252/55
Craniocerebral injuryInjury, poisoning and procedural complications1/250/55
FallInjury, poisoning and procedural complications1/252/55
Femur fractureInjury, poisoning and procedural complications1/250/55
Meniscus injuryInjury, poisoning and procedural complications1/250/55
Most frequent other events
Showing 10 of 229
Most frequent other events
EventBeneFIX: On DemandBeneFIX: Prophylaxis
FallInjury, poisoning and procedural complications7/2521/55
ArthralgiaMusculoskeletal and connective tissue disorders2/2517/55
ContusionInjury, poisoning and procedural complications4/2514/55
Ligament sprainInjury, poisoning and procedural complications1/2512/55
Traumatic haemorrhageInjury, poisoning and procedural complications5/2512/55
HaemarthrosisMusculoskeletal and connective tissue disorders3/2512/55
HaematomaVascular disorders3/2512/55
LacerationInjury, poisoning and procedural complications3/2510/55
Limb injuryInjury, poisoning and procedural complications3/2510/55
Traumatic haematomaInjury, poisoning and procedural complications1/2510/55

Baseline characteristics

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

Age, Continuous
Age, Continuous(years)BeneFIX: On DemandBeneFIX: ProphylaxisTotal
Mean20.2 ± 18.816.0 ± 12.217.3 ± 14.6
Sex: Female, Male
Sex: Female, Male(Participants)BeneFIX: On DemandBeneFIX: ProphylaxisTotal
Female000
Male255580
08

Study locations

21 sites
  • Allgemeines Krankenhaus Linz, Kinderklinik
    Linz, 4020, Austria
  • Sonnengesundheitszentrum
    München, Bayern 80336, Germany
  • Werlhof-Institut für Haemostaseologie GmbH
    Hannover, Niedersachsen 30159, Germany
  • Institut für Thrombophilie und Hämostaseologie
    Muenster, Nordrhein-westfalen 48143, Germany
  • Vivantes Klinikum im Friedrichshain
    Berlin, 10249, Germany
  • Charite Campus Virchow-Klinikum, Padiatrie mit S. Hamatologie und Onkologie
    Berlin, 13353, Germany
  • Kinder- und Jugendarzt-Praxis Blaubeuren
    Blaubeuren, 89143, Germany
  • Institute of Experimental Haematology and Transfusion Medicine
    Bonn, 53127, Germany
  • Praxis fur Kinder- und Jugendmedizin, Homoopathie
    Brannenburg, 83098, Germany
  • Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
    Bremen, 28177, Germany
  • Klinikum Delmehorst gGmbH, Padiatrie
    Delmenhorst, 27753, Germany
  • Universitaetsklinikum Duesseldorf, Klinik f. Kinder-Onkologie, Haematologie u. Klinische Immunologie
    Duesseldorf, 40225, Germany
  • CRC Coagulation Research Centre GmbH
    Duisburg, 47051, Germany
  • Klinikum der Martin-Luther-Universitaet Halle-Wittenberg
    Halle, 06120, Germany
  • Universitaetsklinikum Hamburg-Eppendorf
    Hamburg, 20246, Germany
  • Universitaetsklinikum Eppendorf
    Hamburg, 20251, Germany
  • SRH Kurpfalzkrankenhaus Heidelberg
    Heidelberg, 69123, Germany
  • Gemeinschaftspraxis fuer Haematologie und Onkologie
    Koeln, 50677, Germany
  • Klinikum Memmingen, Kinderklinik
    Memmingen, 87700, Germany
  • Universitaetskinderklinik und Poliklinik im Dr. von Haunerschen
    Muenchen, 80337, Germany
  • Universitaetsklinik fuer Kinder- und Jugendmedizin
    Tuebingen, 72076, Germany
09

References and documents

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 25, 2018, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT00714415
Lead sponsor
Pfizer
Responsible party
Sponsor
First posted
Jul 14, 2008
Start date
Jan 2008
Primary completion
Oct 2016
Completion
Oct 2016
Results posted
Sep 24, 2018
Last update
Oct 25, 2018

Study contacts

Pfizer CT.gov Call Center
study director · Pfizer

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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