Registry For Patients Treated With BeneFix In Usual Care Setting In Germany
28. september 2018 opdateret af: Pfizer
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.
Studieoversigt
Detaljeret beskrivelse
Non-interventional study: subjects to be selected according to the usual clinical practice of their physician
Undersøgelsestype
Observationel
Tilmelding (Faktiske)
80
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
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Berlin, Tyskland, 10249
- Vivantes Klinikum im Friedrichshain
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Berlin, Tyskland, 13353
- Charite Campus Virchow-Klinikum, Padiatrie mit S. Hamatologie und Onkologie
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Blaubeuren, Tyskland, 89143
- Kinder- und Jugendarzt-Praxis Blaubeuren
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Bonn, Tyskland, 53127
- Institute of Experimental Haematology and Transfusion Medicine
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Brannenburg, Tyskland, 83098
- Praxis fur Kinder- und Jugendmedizin, Homoopathie
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Bremen, Tyskland, 28177
- Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
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Delmenhorst, Tyskland, 27753
- Klinikum Delmehorst gGmbH, Padiatrie
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Duesseldorf, Tyskland, 40225
- Universitaetsklinikum Duesseldorf, Klinik f. Kinder-Onkologie, Haematologie u. Klinische Immunologie
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Duisburg, Tyskland, 47051
- CRC Coagulation Research Centre GmbH
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Halle, Tyskland, 06120
- Klinikum der Martin-Luther-Universitaet Halle-Wittenberg
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Hamburg, Tyskland, 20246
- Universitaetsklinikum Hamburg-Eppendorf
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Hamburg, Tyskland, 20251
- Universitaetsklinikum Eppendorf
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Heidelberg, Tyskland, 69123
- SRH Kurpfalzkrankenhaus Heidelberg
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Koeln, Tyskland, 50677
- Gemeinschaftspraxis fuer Haematologie und Onkologie
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Memmingen, Tyskland, 87700
- Klinikum Memmingen, Kinderklinik
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Muenchen, Tyskland, 80337
- Universitaetskinderklinik und Poliklinik im Dr. von Haunerschen
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Tuebingen, Tyskland, 72076
- Universitaetsklinik fuer Kinder- und Jugendmedizin
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Bayern
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München, Bayern, Tyskland, 80336
- Sonnengesundheitszentrum
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Niedersachsen
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Hannover, Niedersachsen, Tyskland, 30159
- Werlhof-Institut für Haemostaseologie GmbH
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Nordrhein-westfalen
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Muenster, Nordrhein-westfalen, Tyskland, 48143
- Institut für Thrombophilie und Hämostaseologie
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Linz, Østrig, 4020
- Allgemeines Krankenhaus Linz, Kinderklinik
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Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
- Barn
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Ingen
Køn, der er berettiget til at studere
Alle
Prøveudtagningsmetode
Ikke-sandsynlighedsprøve
Studiebefolkning
Patients with hemophilia B
Beskrivelse
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®.
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
Kohorter og interventioner
Gruppe / kohorte |
Intervention / Behandling |
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A
Patients with Hemophilia B
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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.
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Tidsramme: Baseline until last visit (up to 8.7 years)
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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.
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Baseline until last visit (up to 8.7 years)
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Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
Tidsramme: Baseline until last visit (up to 8.7 years)
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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.
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Baseline until last visit (up to 8.7 years)
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Number of Participants With Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
Tidsramme: Baseline until last visit (up to 8.7 years)
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FIX inhibitor development was defined as measured inhibitor titer of greater than (>) 0.6 Bethesda Units (BU) using the Nijmegen-modified Bethesda assay.
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Baseline until last visit (up to 8.7 years)
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Number of Participants With Adverse Events (AEs) of Special Interest
Tidsramme: Baseline until last visit (up to 8.7 years)
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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.
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Baseline until last visit (up to 8.7 years)
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Investigator Assessment of Treatment Tolerability of Participants
Tidsramme: End of study visit (any time up to 8.7 years)
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Investigator assessed the tolerability of participants and categorized as very good, good, moderate and poor.
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End of study visit (any time up to 8.7 years)
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Participant Assessment of Treatment Tolerability
Tidsramme: End of study visit (any time up to 8.7 years)
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Participants evaluated their treatment (BeneFIX) tolerability and rated it in 4 categories as very good, good, moderate and poor.
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End of study visit (any time up to 8.7 years)
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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Mean Total Number of Bleeding Episodes in Participants
Tidsramme: Baseline until last visit (up to 8.7 years)
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Participants documented all bleeding episodes in a diary during the study.
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Baseline until last visit (up to 8.7 years)
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Mean Total Number of Bleeding Episodes Per Year in Participants
Tidsramme: Baseline until last visit (up to 8.7 years)
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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.
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Baseline until last visit (up to 8.7 years)
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Number of Participants With Change From Baseline Status in Number of Days Missed From School or Work
Tidsramme: Baseline, up to 8.7 years
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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.
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Baseline, up to 8.7 years
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Investigator Assessment of Treatment Efficacy of Participants
Tidsramme: End of study visit (any time up to 8.7 years)
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Investigator evaluated the efficacy of BeneFIX in participants and rated it in 4 categories as very good, good, moderate and poor.
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End of study visit (any time up to 8.7 years)
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Investigator Assessment of Treatment Handling of Participants
Tidsramme: End of study visit (any time up to 8.7 years)
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Investigator evaluated the handling (administration) of BeneFIX by participants and rated it in 4 categories as very good, good, moderate and poor.
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End of study visit (any time up to 8.7 years)
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Assessment of Treatment Efficacy by the Participants
Tidsramme: End of study visit (any time up to 8.7 years)
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Participants evaluated the efficacy of BeneFIX and rated it in 4 categories as very good, good, moderate and poor.
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End of study visit (any time up to 8.7 years)
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Assessment of Treatment Handling by the Participants
Tidsramme: End of study visit (any time up to 8.7 years)
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Participants evaluated the handling (administration) of BeneFIX and rated it in 4 categories as very good, good, moderate and poor.
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End of study visit (any time up to 8.7 years)
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Investigator Assessment of Treatment Satisfaction of Participants
Tidsramme: Baseline up to 8.7 years
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Investigator evaluated the participant's satisfaction of treatment with BeneFIX and rated it in 4 categories as very satisfied, satisfied, unsatisfied and very unsatisfied.
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Baseline up to 8.7 years
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Publikationer og nyttige links
Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Faktiske)
1. januar 2008
Primær færdiggørelse (Faktiske)
1. oktober 2016
Studieafslutning (Faktiske)
1. oktober 2016
Datoer for studieregistrering
Først indsendt
8. juli 2008
Først indsendt, der opfyldte QC-kriterier
11. juli 2008
Først opslået (Skøn)
14. juli 2008
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
25. oktober 2018
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
28. september 2018
Sidst verificeret
1. september 2018
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- 3090A1-4406
- B1821011 (Anden identifikator: Alias Study Number)
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .