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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 de dezembro de 2020 atualizado por: 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.

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Descrição detalhada

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 de estudo

Intervencional

Inscrição (Real)

120

Estágio

  • Fase 3

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

  • Alemanha
    • North Rhine-westphalia
      • Bonn, North Rhine-westphalia, Alemanha, 53127
        • Research Site
  • Austrália
    • South Australia
      • Adelaide, South Australia, Austrália, 5000
        • Research Site
    • Victoria
      • Parkville, Victoria, Austrália, 3052
        • Research Site
    • Western Australia
      • Murdoch, Western Australia, Austrália, 6150
        • Research Site
      • Perth, Western Australia, Austrália, 6008
        • Research Site
  • Brasil
    • Sao Paulo
      • Campinas, Sao Paulo, Brasil, 13083-878
        • Research Site
  • Bélgica
      • Bruxelles, Bélgica, 1200
        • Research Site
      • Leuven, Bélgica, 3000
        • Research Site
  • Canadá
    • Ontario
      • Toronto, Ontario, Canadá, M5B 1W8
        • Research Site
    • Quebec
      • Montreal, Quebec, Canadá, H3T 1C5
        • 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
  • Estados Unidos
    • Arizona
      • Phoenix, Arizona, Estados Unidos, 85016
        • Research Site
    • California
      • Sacramento, California, Estados Unidos, 95817
        • Research Site
    • Colorado
      • Aurora, Colorado, Estados Unidos, 80045
        • Research Site
    • Georgia
      • Atlanta, Georgia, Estados Unidos, 30322
        • Research Site
    • Hawaii
      • Honolulu, Hawaii, Estados Unidos, 96826
        • Research Site
    • Indiana
      • Indianapolis, Indiana, Estados Unidos, 46260
        • Research Site
    • Louisiana
      • New Orleans, Louisiana, Estados Unidos, 70112
        • Research Site
    • Michigan
      • East Lansing, Michigan, Estados Unidos, 48823
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, Estados Unidos, 15213
        • Research Site
    • Washington
      • Seattle, Washington, Estados Unidos, 98104
        • Research Site
  • França
    • Bouches-Du-Rhône
      • Marseille, Bouches-Du-Rhône, França, 13385
        • Research Site
  • Holanda
      • Utrecht, Holanda, 3584 CX
        • Research Site
  • Hong Kong
      • Hong Kong, Hong Kong
        • Research Site
    • New Territories
      • Hong Kong, New Territories, Hong Kong
        • Research Site
  • Irlanda
      • Dublin, Irlanda, D12 N512
        • Research Site
  • Itália
      • Florence, Itália, 50134
        • Research Site
      • Milano, Itália, 20122
        • Research Site
  • Japão
    • Aichi-Ken
      • Nagoya-Shi, Aichi-Ken, Japão, 466-8550
        • Research Site
    • Fukuoka-Ken
      • Kitakyushu, Fukuoka-Ken, Japão, 807-8555
        • Research Site
    • Kanagawa-Ken
      • Kawasaki, Kanagawa-Ken, Japão, 216-8511
        • Research Site
    • Nara-Ken
      • Kashihara-shi, Nara-Ken, Japão, 634-8522
        • Research Site
    • Tokyo-To
      • Shinjuku-ku, Tokyo-To, Japão, 160-0023
        • Research Site
      • Tokyo, Tokyo-To, Japão, 167-8515
        • Research Site
  • Polônia
      • Lodz, Polônia, 93-510
        • Research Site
  • Reino Unido
    • Cambridgeshire
      • Cambridge, Cambridgeshire, Reino Unido, CB2 0QQ
        • Research Site
    • Greater London
      • London, Greater London, Reino Unido, E1 1BB
        • Research Site
      • London, Greater London, Reino Unido, SE1 7EH
        • Research Site
    • Hampshire
      • Basingstoke, Hampshire, Reino Unido, RG24 9NA
        • Research Site
  • Suécia
      • Malmö, Suécia, 20502
        • Research Site
      • Stockholm, Suécia, 17176
        • Research Site
  • África do Sul
    • Gauteng
      • Johannesburg, Gauteng, África do Sul, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, África do Sul, 7925
        • Research Site
  • Índia
    • Karnataka
      • Bangalore, Karnataka, Índia, 560034
        • Research Site
    • Maharashtra
      • Pune, Maharashtra, Índia, 411004
        • Research Site
    • Tamil Nadu
      • Vellore, Tamil Nadu, Índia, 632004
        • Research Site

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Filho
  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Macho

Descrição

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.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Não randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
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.
Biológico: rFIXFc
Administered as specified in the treatment arm.
Outros nomes:
  • Alprolix
  • BIIB029
  • fator de coagulação IX (recombinante) proteína de fusão Fc
Experimental: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biológico: rFIXFc
Administered as specified in the treatment arm.
Outros nomes:
  • Alprolix
  • BIIB029
  • fator de coagulação IX (recombinante) proteína de fusão Fc

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Number of Participants With Any Positive Inhibitor Development
Prazo: 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

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Annualized Bleeding Rate (ABR)
Prazo: 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
Prazo: 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)
Prazo: 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])
Prazo: 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
Prazo: 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
Prazo: 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

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Bioverativ Therapeutics Inc.

Investigadores

  • Diretor de estudo: Medical Director, Bioverativ Therapeutics Inc.

Publicações e links úteis

A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.

Publicações Gerais

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

8 de dezembro de 2011

Conclusão Primária (Real)

1 de outubro de 2017

Conclusão do estudo (Real)

1 de outubro de 2017

Datas de inscrição no estudo

Enviado pela primeira vez

19 de agosto de 2011

Enviado pela primeira vez que atendeu aos critérios de CQ

29 de agosto de 2011

Primeira postagem (Estimativa)

30 de agosto de 2011

Atualizações de registro de estudo

Última Atualização Postada (Real)

19 de dezembro de 2020

Última atualização enviada que atendeu aos critérios de controle de qualidade

16 de dezembro de 2020

Última verificação

1 de novembro de 2018

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • 9HB01EXT
  • 2011-003075-11

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

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