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

Hemophilia B Clinical Trial

Official title:

Pharmacovigilance Evaluation Of Benefix (Registered) In Germany And Austria

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00714415
• Other study ID #: 3090A1-4406
• Secondary ID: B1821011
• Status: Completed
• Start date: January 2008
• Est. completion date: October 2016

Study information

• Verified date: September 2018
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 80
• Est. completion date: October 2016
• Est. primary completion date: October 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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®.

Related Conditions & MeSH terms

• Hemophilia B

Intervention

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.

Locations

Country	Name	City	State
Austria	Allgemeines Krankenhaus Linz, Kinderklinik	Linz
Germany	Charite Campus Virchow-Klinikum, Padiatrie mit S. Hamatologie und Onkologie	Berlin
Germany	Vivantes Klinikum im Friedrichshain	Berlin
Germany	Kinder- und Jugendarzt-Praxis Blaubeuren	Blaubeuren
Germany	Institute of Experimental Haematology and Transfusion Medicine	Bonn
Germany	Praxis fur Kinder- und Jugendmedizin, Homoopathie	Brannenburg
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	CRC Coagulation Research Centre GmbH	Duisburg
Germany	Klinikum der Martin-Luther-Universitaet Halle-Wittenberg	Halle
Germany	Universitaetsklinikum Eppendorf	Hamburg
Germany	Universitaetsklinikum Hamburg-Eppendorf	Hamburg
Germany	Werlhof-Institut für Haemostaseologie GmbH	Hannover	Niedersachsen
Germany	SRH Kurpfalzkrankenhaus Heidelberg	Heidelberg
Germany	Gemeinschaftspraxis fuer Haematologie und Onkologie	Koeln
Germany	Klinikum Memmingen, Kinderklinik	Memmingen
Germany	Universitaetskinderklinik und Poliklinik im Dr. von Haunerschen	Muenchen
Germany	Institut für Thrombophilie und Hämostaseologie	Muenster	Nordrhein-westfalen
Germany	Sonnengesundheitszentrum	München	Bayern
Germany	Universitaetsklinik fuer Kinder- und Jugendmedizin	Tuebingen

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

Austria,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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	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)
Secondary	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)
Secondary	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
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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

External Links

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