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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 dicembre 2020 aggiornato da: 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.

Panoramica dello studio

Stato

Completato

Condizioni

Intervento / Trattamento

Descrizione dettagliata

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)

Tipo di studio

Interventistico

Iscrizione (Effettivo)

120

Fase

  • Fase 3

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

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

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Bambino
  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Sessi ammissibili allo studio

Maschio

Descrizione

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.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Non randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: 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.
Biologico: rFIXFc
Administered as specified in the treatment arm.
Altri nomi:
  • Alprolix
  • BIIB029
  • fattore IX della coagulazione (ricombinante) proteina di fusione Fc
Sperimentale: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biologico: rFIXFc
Administered as specified in the treatment arm.
Altri nomi:
  • Alprolix
  • BIIB029
  • fattore IX della coagulazione (ricombinante) proteina di fusione Fc

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Number of Participants With Any Positive Inhibitor Development
Lasso di tempo: 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

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Annualized Bleeding Rate (ABR)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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])
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Bioverativ Therapeutics Inc.

Investigatori

  • Direttore dello studio: Medical Director, Bioverativ Therapeutics Inc.

Pubblicazioni e link utili

La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.

Pubblicazioni generali

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

8 dicembre 2011

Completamento primario (Effettivo)

1 ottobre 2017

Completamento dello studio (Effettivo)

1 ottobre 2017

Date di iscrizione allo studio

Primo inviato

19 agosto 2011

Primo inviato che soddisfa i criteri di controllo qualità

29 agosto 2011

Primo Inserito (Stima)

30 agosto 2011

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

19 dicembre 2020

Ultimo aggiornamento inviato che soddisfa i criteri QC

16 dicembre 2020

Ultimo verificato

1 novembre 2018

Maggiori informazioni

Termini relativi a questo studio

Parole chiave

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • 9HB01EXT
  • 2011-003075-11

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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