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

NCT00714415 | Completed Results

• 2 other identifiers: 3090A1-4406B1821011
• Study Type: observational
• Target: 80
• Locations: 2 countries
• Sites: 21

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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (6)

• 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 Outcomes (8)

• Mean Total Number of Bleeding Episodes in Participants

  Baseline until last visit (up to 8.7 years)

• Mean Total Number of Bleeding Episodes Per Year in Participants

  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

  Baseline, up to 8.7 years

• Investigator Assessment of Treatment Efficacy of Participants

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

• Investigator Assessment of Treatment Handling of Participants

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

Study Arms (1)

A

Patients with Hemophilia B

Drug: BeneFIX

Interventions

BeneFIX DRUG

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.

A

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with hemophilia B

You may qualify if:

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

You may not qualify if:

• Patients with hemophilia B treated with a product other than BeneFIX®.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (21)

Allgemeines Krankenhaus Linz, Kinderklinik

Linz, 4020, Austria

Location

Sonnengesundheitszentrum

München, Bavaria, 80336, Germany

Location

Werlhof-Institut für Haemostaseologie GmbH

Hanover, Lower Saxony, 30159, Germany

Location

Institut für Thrombophilie und Hämostaseologie

Münster, North Rhine-Westphalia, 48143, Germany

Location

Vivantes Klinikum im Friedrichshain

Berlin, 10249, Germany

Location

Charite Campus Virchow-Klinikum, Padiatrie mit S. Hamatologie und Onkologie

Berlin, 13353, Germany

Location

Kinder- und Jugendarzt-Praxis Blaubeuren

Blaubeuren Abbey, 89143, Germany

Location

Institute of Experimental Haematology and Transfusion Medicine

Bonn, 53127, Germany

Location

Praxis fur Kinder- und Jugendmedizin, Homoopathie

Brannenburg, 83098, Germany

Location

Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik

Bremen, 28177, Germany

Location

Gemeinschaftspraxis fuer Haematologie und Onkologie

Cologne, 50677, Germany

Location

Klinikum Delmehorst gGmbH, Padiatrie

Delmenhorst, 27753, Germany

Location

CRC Coagulation Research Centre GmbH

Duisburg, 47051, Germany

Location

Universitaetsklinikum Duesseldorf, Klinik f. Kinder-Onkologie, Haematologie u. Klinische Immunologie

Düsseldorf, 40225, Germany

Location

Klinikum der Martin-Luther-Universitaet Halle-Wittenberg

Halle, 06120, Germany

Location

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, 20246, Germany

Location

Universitaetsklinikum Eppendorf

Hamburg, 20251, Germany

Location

SRH Kurpfalzkrankenhaus Heidelberg

Heidelberg, 69123, Germany

Location

Klinikum Memmingen, Kinderklinik

Memmingen, 87700, Germany

Location

Universitaetskinderklinik und Poliklinik im Dr. von Haunerschen

München, 80337, Germany

Location

Universitaetsklinik fuer Kinder- und Jugendmedizin

Tübingen, 72076, Germany

Location

Related Links

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

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

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.

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2008
• First Posted: July 14, 2008
• Study Start: January 1, 2008
• Primary Completion: October 1, 2016
• Study Completion: October 1, 2016
• Last Updated: October 25, 2018
• Results First Posted: September 24, 2018

Record last verified: 2018-09

Locations

AU (1); DE (20)