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

September 28, 2018 updated by: Pfizer

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
80

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with hemophilia B

Description

Inclusion Criteria:

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

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®.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
A
Patients with Hemophilia B
Drug: BeneFIX
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.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
Baseline until last visit (up to 8.7 years)
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.
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
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.
Baseline until last visit (up to 8.7 years)
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.
Baseline until last visit (up to 8.7 years)
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.
End of study visit (any time up to 8.7 years)
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.
End of study visit (any time up to 8.7 years)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Mean Total Number of Bleeding Episodes in Participants
Time Frame: Baseline until last visit (up to 8.7 years)
Participants documented all bleeding episodes in a diary during the study.
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)
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.
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
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.
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 evaluated the efficacy of BeneFIX in participants and rated it in 4 categories as very good, good, moderate and poor.
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)
Investigator evaluated the handling (administration) of BeneFIX by participants and rated it in 4 categories as very good, good, moderate and poor.
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)
Participants evaluated the efficacy of BeneFIX and rated it in 4 categories as very good, good, moderate and poor.
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)
Participants evaluated the handling (administration) of BeneFIX and rated it in 4 categories as very good, good, moderate and poor.
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
Investigator evaluated the participant's satisfaction of treatment with BeneFIX and rated it in 4 categories as very satisfied, satisfied, unsatisfied and very unsatisfied.
Baseline up to 8.7 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 1, 2008

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 1, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 1, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 8, 2008

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 11, 2008

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 14, 2008

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 25, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 28, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 3090A1-4406
  • B1821011 (Other Identifier: Alias Study Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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