Study Evaluating Pharmacovigilance Of Refacto AF

Pharmacovigilance Evaluation Of Refacto Af (Registered) In Germany And Austria

The purpose of this observational study is to describe the incidence of adverse events among patients treated with Refacto AF in usual health care settings in Germany and Austria.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: ReFacto AF (Moroctocog alfa)

Detailed Description

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

• Study Type: Observational
• Enrollment (Actual): 101

Contacts and Locations

Study Locations

• Austria
  • Graz, Austria, 8036
    • LKH - Univ. Klinikum Graz,Abt. fur Hamatologie
  • Linz, Austria, 4020
    • Allgemeines Krankenhaus Linz, Kinderklinik
  • Wien, Austria, 1090
    • Universitaetsklinik fuer Innere Medizin 1
• Germany
  • Berlin, Germany, 10249
    • Vivantes Klinikum im Friedrichshain
  • Bremen, Germany, 28205
    • Klinikum Bremen Mitte
  • Duesseldorf, Germany, 40225
    • Universitaetsklinikum Duesseldorf
  • Duesseldorf, Germany, 40225
    • Universitaetsklinikum Duesseldorf, Klinik f. Kinder-Onkologie, Haematologie u. Klinische Immunologie
  • Duisburg, Germany, 47051
    • CRC Coagulation Research Centre GmbH
  • Frankfurt a. M., Germany, 60596
    • Praxis zur Diagnostik und Therapie Von Blutgerinnungstoerungen
  • Grunwald, Germany, 82031
    • Praxis fur Kinder- und Jugendmedizin
  • Hannover, Germany, 30625
    • Medizinische Hochschule Hannover
  • Heidelberg, Germany, 69123
    • SRH Kurpfalzkrankenhaus Heidelberg
  • Memmingen, Germany, 87700
    • Donaustrasse 78
  • Metzingen, Germany, 72555
    • Praxis Dr. Autenrieth
  • Muenster, Germany, 48143
    • Institut for Thrombophilia and Hemastaseologie
  • Mutlangen, Germany, 73557
    • Stauferklinikum Schwaebisch Gmuend
  • Mörfelden-Walldorf, Germany, 64546
    • Hämophilie-Zentrum Rhein Main GmbH
  • Regensburg, Germany, 93042
    • Universitat Regensburg
  • Stadtroda, Germany, 07646
    • Asklepios Fachklinikum Stadtroda GmbH
  • Stuttgart, Germany, 70176
    • Klinikum Stuttgart
  • Tuebingen, Germany, 72076
    • Universitaetsklinik fuer Kinder- und Jugendmedizin
  • Wiesbaden, Germany, 65191
    • Stiftung Deutsche Klinik für Diagnostik GmbH
  • Bayern
    • München, Bayern, Germany, 80336
      • Sonnengesundheitszentrum
  • Niedersachsen
    • Hannover, Niedersachsen, Germany, 30159
      • Werlhof-Institut für Haemostaseologie GmbH

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 A

Description

Inclusion Criteria:

• Patients with hemophilia A of any severity already receiving or starting treatment with ReFacto AF.

Exclusion Criteria:

• Patients with Hemophilia A treated with a product other than Refacto AF.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
1; Patients treated with Refacto AF	Drug: ReFacto AF (Moroctocog alfa); Patients will be treated with intravenous infusion of ReFacto AF per dosing and frequency as prescribed by the subjects' treating physician. ReFacto AF® will be prescribed in the context of routine clinical practice in Germany and Austria, respectively

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 87 months) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.	Baseline until last visit (up to 87 months)
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 adverse events. Relatedness of AEs with Refacto AF was assessed by the investigator.	Baseline until last visit (up to 87 months)
Number of Participants With Factor VIII (FVIII) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay	FVIII 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 87 months)
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 87 months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 as: mean total number of bleeding episodes divided by duration of observation period (in years) for bleeding documentation.	Baseline until last visit (up to 87 months)
Number of Participants With Change From Baseline Status in 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 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 until last visit (up to 87 months)
Participant Assessment of Satisfaction With Treatment Handling	Participants evaluated their satisfaction with handing (administration) of Refacto AF and rated it in 4 categories as: very satisfied, satisfied, unsatisfied and very unsatisfied.	End of study visit (any time up to 87 months)
Investigator Assessment of Treatment Satisfaction of Participants	Investigator assessed the treatment satisfaction of participants and categorized as very satisfied, satisfied, unsatisfied, or very unsatisfied.	End of study visit (any time up to 87 months)

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): July 17, 2009
• Primary Completion (Actual): October 19, 2016
• Study Completion (Actual): October 19, 2016

Study Registration Dates

• First Submitted: May 1, 2009
• First Submitted That Met QC Criteria: May 5, 2009
• First Posted (Estimate): May 7, 2009

Study Record Updates

• Last Update Posted (Actual): July 17, 2018
• Last Update Submitted That Met QC Criteria: September 22, 2017
• Last Verified: September 1, 2017

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; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: 3082B2-4420; B1831016 (Other Identifier: Alias Study Number)