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Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B (B-YOND)

16. Dezember 2020 aktualisiert von: Bioverativ Therapeutics Inc.

An Open-Label, Multicenter, Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B

The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B.

The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.

Studienübersicht

Status

Abgeschlossen

Bedingungen

Intervention / Behandlung

Detaillierte Beschreibung

Participants will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the patient in the preceding studies, B-LONG 998HB102 (NCT01027364) and Kids B-LONG study 9HB02PED (NCT01440946)

Studientyp

Interventionell

Einschreibung (Tatsächlich)

120

Phase

  • Phase 3

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienorte

  • Australien
    • South Australia
      • Adelaide, South Australia, Australien, 5000
        • Research Site
    • Victoria
      • Parkville, Victoria, Australien, 3052
        • Research Site
    • Western Australia
      • Murdoch, Western Australia, Australien, 6150
        • Research Site
      • Perth, Western Australia, Australien, 6008
        • Research Site
  • Belgien
      • Bruxelles, Belgien, 1200
        • Research Site
      • Leuven, Belgien, 3000
        • Research Site
  • Brasilien
    • Sao Paulo
      • Campinas, Sao Paulo, Brasilien, 13083-878
        • Research Site
  • China
    • Beijingshì
      • Beijing, Beijingshì, China, 100005
        • Research Site
    • Guangdongsheng
      • Guangzhou, Guangdongsheng, China, 510515
        • Research Site
    • Shànghaishì
      • Shanghai, Shànghaishì, China, 200025
        • Research Site
    • Tianjinshì
      • Tianjing, Tianjinshì, China, 300020
        • Research Site
  • Deutschland
    • North Rhine-westphalia
      • Bonn, North Rhine-westphalia, Deutschland, 53127
        • Research Site
  • Frankreich
    • Bouches-Du-Rhône
      • Marseille, Bouches-Du-Rhône, Frankreich, 13385
        • Research Site
  • Hongkong
      • Hong Kong, Hongkong
        • Research Site
    • New Territories
      • Hong Kong, New Territories, Hongkong
        • Research Site
  • Indien
    • Karnataka
      • Bangalore, Karnataka, Indien, 560034
        • Research Site
    • Maharashtra
      • Pune, Maharashtra, Indien, 411004
        • Research Site
    • Tamil Nadu
      • Vellore, Tamil Nadu, Indien, 632004
        • Research Site
  • Irland
      • Dublin, Irland, D12 N512
        • Research Site
  • Italien
      • Florence, Italien, 50134
        • Research Site
      • Milano, Italien, 20122
        • Research Site
  • Japan
    • Aichi-Ken
      • Nagoya-Shi, Aichi-Ken, Japan, 466-8550
        • Research Site
    • Fukuoka-Ken
      • Kitakyushu, Fukuoka-Ken, Japan, 807-8555
        • Research Site
    • Kanagawa-Ken
      • Kawasaki, Kanagawa-Ken, Japan, 216-8511
        • Research Site
    • Nara-Ken
      • Kashihara-shi, Nara-Ken, Japan, 634-8522
        • Research Site
    • Tokyo-To
      • Shinjuku-ku, Tokyo-To, Japan, 160-0023
        • Research Site
      • Tokyo, Tokyo-To, Japan, 167-8515
        • Research Site
  • Kanada
    • Ontario
      • Toronto, Ontario, Kanada, M5B 1W8
        • Research Site
    • Quebec
      • Montreal, Quebec, Kanada, H3T 1C5
        • Research Site
  • Niederlande
      • Utrecht, Niederlande, 3584 CX
        • Research Site
  • Polen
      • Lodz, Polen, 93-510
        • Research Site
  • Schweden
      • Malmö, Schweden, 20502
        • Research Site
      • Stockholm, Schweden, 17176
        • Research Site
  • Südafrika
    • Gauteng
      • Johannesburg, Gauteng, Südafrika, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, Südafrika, 7925
        • Research Site
  • Vereinigte Staaten
    • Arizona
      • Phoenix, Arizona, Vereinigte Staaten, 85016
        • Research Site
    • California
      • Sacramento, California, Vereinigte Staaten, 95817
        • Research Site
    • Colorado
      • Aurora, Colorado, Vereinigte Staaten, 80045
        • Research Site
    • Georgia
      • Atlanta, Georgia, Vereinigte Staaten, 30322
        • Research Site
    • Hawaii
      • Honolulu, Hawaii, Vereinigte Staaten, 96826
        • Research Site
    • Indiana
      • Indianapolis, Indiana, Vereinigte Staaten, 46260
        • Research Site
    • Louisiana
      • New Orleans, Louisiana, Vereinigte Staaten, 70112
        • Research Site
    • Michigan
      • East Lansing, Michigan, Vereinigte Staaten, 48823
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, Vereinigte Staaten, 15213
        • Research Site
    • Washington
      • Seattle, Washington, Vereinigte Staaten, 98104
        • Research Site
  • Vereinigtes Königreich
    • Cambridgeshire
      • Cambridge, Cambridgeshire, Vereinigtes Königreich, CB2 0QQ
        • Research Site
    • Greater London
      • London, Greater London, Vereinigtes Königreich, E1 1BB
        • Research Site
      • London, Greater London, Vereinigtes Königreich, SE1 7EH
        • Research Site
    • Hampshire
      • Basingstoke, Hampshire, Vereinigtes Königreich, RG24 9NA
        • Research Site

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Kind
  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Nein

Studienberechtigte Geschlechter

Männlich

Beschreibung

Key Inclusion Criteria:

  • Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
  • Ability to understand the purposes & risks of the study and provide signed and dated informed consent.

Key Exclusion Criteria:

  • High-titer inhibitor (>/=5.00 BU/mL)

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Hauptzweck: Behandlung
  • Zuteilung: Nicht randomisiert
  • Interventionsmodell: Parallele Zuordnung
  • Maskierung: Keine (Offenes Etikett)

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: On-Demand
The individual dose of rFIXFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor IX peak (recovery) levels.
Biologisch: rFIXFc
Administered as specified in the treatment arm.
Andere Namen:
  • Alprolix
  • BIIB029
  • Gerinnungsfaktor IX (rekombinantes) Fc-Fusionsprotein
Experimental: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biologisch: rFIXFc
Administered as specified in the treatment arm.
Andere Namen:
  • Alprolix
  • BIIB029
  • Gerinnungsfaktor IX (rekombinantes) Fc-Fusionsprotein

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Number of Participants With Any Positive Inhibitor Development
Zeitfenster: Approximately 5 years
An inhibitor test result greater than or equal to (>=)0.6 Bethesda units per milliliter (BU/mL), confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Data was summarized by treatment regimen for participants from Study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from Study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Annualized Bleeding Rate (ABR)
Zeitfenster: Approximately 5 years
ABR is annualized number of bleeding episodes per participant per year. Bleeding episodes were classified as spontaneous if participant records bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity and classified as traumatic if participant records bleeding event when there is known reason for bleed. ABR=(Number of bleeding episodes during efficacy period/number of days during efficacy period)*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. ABR was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years
Annualized Spontaneous Joint Bleeding Episodes
Zeitfenster: Approximately 5 years
Bleeding episodes were classified as spontaneous if participant records a bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity. In addition, location of bleed (joint, internal, skin/mucosa or muscle) were also collected. Annualized spontaneous joint bleeding episodes=(Number of spontaneous joint bleeding episodes during efficacy period/number of days during efficacy period)*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Bleeding episodes were summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years
Total Number of Exposure Days (EDs)
Zeitfenster: Approximately 5 years
An exposure day is a 24-hour period in which one or more rFIXFc injections are given. The total number of days of exposure to rFIXFc were summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years
Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
Zeitfenster: Approximately 5 years
Annualized consumption = (total international unit per kilogram [IU/kg] of study treatment received during the efficacy period / total number of days during the efficacy period) multiplied by 365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Annualized consumption was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Approximately 5 years
Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
Zeitfenster: Approximately 5 years
Participants were assessed for response to their rFIXFc regimen using following 4-point scale: 1=Excellent: bleeding episodes responded to less than or equal to (<=)usual number of injections or dose of rFIXFc or rate of breakthrough bleeding during prophylaxis was <= that usually observed; 2=Effective: most bleeding episodes responded to same number of injections and dose, but some required more injections or higher doses, or there was minor increase in rate of breakthrough; 3=Partially Effective: bleeding episodes most often required more injections and/or higher doses than expected or adequate breakthrough bleeding prevention during prophylaxis required more frequent injections and/or higher doses and 4=Ineffective: routine failure to control hemostasis/hemostatic control require additional agents. Total number of scale responses =total count of scale responses for all participants; multiple responses per participant including those at scheduled and unscheduled visits are counted.
Approximately 5 years
Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
Zeitfenster: Approximately 5 years
Using eDiary, participant received rating for treatment response to any bleeding episode (BE) using 4-point scale- 1=Excellent: Abrupt pain relief and/or improvement in signs of bleeding within approximately (approx.) 8 hours (h) after initial injection (inj.); 2=Good: Definite pain relief and/or improvement in signs of bleeding within approx. 8h after an injection, but possibly requiring more than 1 injection after 24-48h for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8h after initial injection and requires more than 1 injection and 4=None: No improvement, or condition worsens within approx. 8h after initial injection. This assessment was to be made approx. 8 to 12h from time the injection was given to treat BE and prior to any additional doses of rFIXFc given for same bleeding episode. Percentages are based on the number of bleeding episodes for which a response (excellent or good) was provided for the first injection during the efficacy period.
Approximately 5 years

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

Bioverativ Therapeutics Inc.

Ermittler

  • Studienleiter: Medical Director, Bioverativ Therapeutics Inc.

Publikationen und hilfreiche Links

Die Bereitstellung dieser Publikationen erfolgt freiwillig durch die für die Eingabe von Informationen über die Studie verantwortliche Person. Diese können sich auf alles beziehen, was mit dem Studium zu tun hat.

Allgemeine Veröffentlichungen

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Tatsächlich)

8. Dezember 2011

Primärer Abschluss (Tatsächlich)

1. Oktober 2017

Studienabschluss (Tatsächlich)

1. Oktober 2017

Studienanmeldedaten

Zuerst eingereicht

19. August 2011

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

29. August 2011

Zuerst gepostet (Schätzen)

30. August 2011

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

19. Dezember 2020

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

16. Dezember 2020

Zuletzt verifiziert

1. November 2018

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Schlüsselwörter

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • 9HB01EXT
  • 2011-003075-11

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