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

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: Completed
• Conditions: Hemophilia B
• Intervention / Treatment: Drug: BeneFIX

Detailed Description

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

• Study Type: Observational
• Enrollment (Actual): 80

Contacts and Locations

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

Eligibility Criteria

• 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

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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.	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.	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.	End of study visit (any time up to 8.7 years)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Total Number of Bleeding Episodes in Participants	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	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	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	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	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	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	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	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

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2008
• Primary Completion (Actual): October 1, 2016
• Study Completion (Actual): October 1, 2016

Study Registration Dates

• First Submitted: July 8, 2008
• First Submitted That Met QC Criteria: July 11, 2008
• First Posted (Estimate): July 14, 2008

Study Record Updates

• Last Update Posted (Actual): October 25, 2018
• Last Update Submitted That Met QC Criteria: September 28, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia B
• Other Study ID Numbers: 3090A1-4406; B1821011 (Other Identifier: Alias Study Number)