Registry For Patients Treated With BeneFix In Usual Care Setting In Germany
- Registration Number
- NCT00714415
- Lead Sponsor
- Pfizer
- Brief 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.
- Detailed Description
Non-interventional study: subjects to be selected according to the usual clinical practice of their physician
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 80
- Patients with hemophilia B already receiving or starting treatment with reformulated BeneFIX®.
- 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 & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description A BeneFIX Patients with Hemophilia B
- Primary Outcome Measures
Name Time Method Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) 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 Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) 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 Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay 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 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 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 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.
- Secondary Outcome Measures
Name Time Method Mean Total Number of Bleeding Episodes in Participants 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 in Participants 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.
Number of Participants With Change From Baseline Status in Number of Days Missed From School or Work 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.
Investigator Assessment of Treatment Efficacy of Participants 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.
Investigator Assessment of Treatment Handling of Participants 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.
Assessment of Treatment Efficacy by the Participants 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.
Assessment of Treatment Handling by the Participants 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.
Investigator Assessment of Treatment Satisfaction of Participants 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.
Trial Locations
- Locations (21)
Allgemeines Krankenhaus Linz, Kinderklinik
🇦🇹Linz, Austria
Werlhof-Institut für Haemostaseologie GmbH
🇩🇪Hannover, Niedersachsen, Germany
Klinikum der Martin-Luther-Universitaet Halle-Wittenberg
🇩🇪Halle, Germany
Kinder- und Jugendarzt-Praxis Blaubeuren
🇩🇪Blaubeuren, Germany
Institute of Experimental Haematology and Transfusion Medicine
🇩🇪Bonn, Germany
Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
🇩🇪Bremen, Germany
Klinikum Delmehorst gGmbH, Padiatrie
🇩🇪Delmenhorst, Germany
Universitaetsklinikum Duesseldorf, Klinik f. Kinder-Onkologie, Haematologie u. Klinische Immunologie
🇩🇪Duesseldorf, Germany
Charite Campus Virchow-Klinikum, Padiatrie mit S. Hamatologie und Onkologie
🇩🇪Berlin, Germany
Sonnengesundheitszentrum
🇩🇪München, Bayern, Germany
Institut für Thrombophilie und Hämostaseologie
🇩🇪Muenster, Nordrhein-westfalen, Germany
Vivantes Klinikum im Friedrichshain
🇩🇪Berlin, Germany
Praxis fur Kinder- und Jugendmedizin, Homoopathie
🇩🇪Brannenburg, Germany
CRC Coagulation Research Centre GmbH
🇩🇪Duisburg, Germany
Universitaetsklinikum Hamburg-Eppendorf
🇩🇪Hamburg, Germany
Universitaetsklinikum Eppendorf
🇩🇪Hamburg, Germany
Gemeinschaftspraxis fuer Haematologie und Onkologie
🇩🇪Koeln, Germany
Universitaetsklinik fuer Kinder- und Jugendmedizin
🇩🇪Tuebingen, Germany
SRH Kurpfalzkrankenhaus Heidelberg
🇩🇪Heidelberg, Germany
Klinikum Memmingen, Kinderklinik
🇩🇪Memmingen, Germany
Universitaetskinderklinik und Poliklinik im Dr. von Haunerschen
🇩🇪Muenchen, Germany