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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 diciembre de 2020 actualizado 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.

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Descripción detallada

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 estudio

Intervencionista

Inscripción (Actual)

120

Fase

  • Fase 3

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Alemania
    • North Rhine-westphalia
      • Bonn, North Rhine-westphalia, Alemania, 53127
        • Research Site
  • 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
  • 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
  • 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
  • Francia
    • Bouches-Du-Rhône
      • Marseille, Bouches-Du-Rhône, Francia, 13385
        • 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
  • Japón
    • Aichi-Ken
      • Nagoya-Shi, Aichi-Ken, Japón, 466-8550
        • Research Site
    • Fukuoka-Ken
      • Kitakyushu, Fukuoka-Ken, Japón, 807-8555
        • Research Site
    • Kanagawa-Ken
      • Kawasaki, Kanagawa-Ken, Japón, 216-8511
        • Research Site
    • Nara-Ken
      • Kashihara-shi, Nara-Ken, Japón, 634-8522
        • Research Site
    • Tokyo-To
      • Shinjuku-ku, Tokyo-To, Japón, 160-0023
        • Research Site
      • Tokyo, Tokyo-To, Japón, 167-8515
        • Research Site
  • Países Bajos
      • Utrecht, Países Bajos, 3584 CX
        • Research Site
  • Polonia
      • Lodz, Polonia, 93-510
        • Research Site
  • Porcelana
    • Beijingshì
      • Beijing, Beijingshì, Porcelana, 100005
        • Research Site
    • Guangdongsheng
      • Guangzhou, Guangdongsheng, Porcelana, 510515
        • Research Site
    • Shànghaishì
      • Shanghai, Shànghaishì, Porcelana, 200025
        • Research Site
    • Tianjinshì
      • Tianjing, Tianjinshì, Porcelana, 300020
        • 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
  • Sudáfrica
    • Gauteng
      • Johannesburg, Gauteng, Sudáfrica, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, Sudáfrica, 7925
        • Research Site
  • Suecia
      • Malmö, Suecia, 20502
        • Research Site
      • Stockholm, Suecia, 17176
        • Research Site

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Niño
  • Adulto
  • Adulto Mayor

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Masculino

Descripción

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.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
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.
Otros nombres:
  • Alprolix
  • BIIB029
  • factor de coagulación IX (recombinante) Proteína de fusión Fc
Experimental: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biológico: rFIXFc
Administered as specified in the treatment arm.
Otros nombres:
  • Alprolix
  • BIIB029
  • factor de coagulación IX (recombinante) Proteína de fusión Fc

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Number of Participants With Any Positive Inhibitor Development
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Annualized Bleeding Rate (ABR)
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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])
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Bioverativ Therapeutics Inc.

Investigadores

  • Director de estudio: Medical Director, Bioverativ Therapeutics Inc.

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

8 de diciembre de 2011

Finalización primaria (Actual)

1 de octubre de 2017

Finalización del estudio (Actual)

1 de octubre de 2017

Fechas de registro del estudio

Enviado por primera vez

19 de agosto de 2011

Primero enviado que cumplió con los criterios de control de calidad

29 de agosto de 2011

Publicado por primera vez (Estimar)

30 de agosto de 2011

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

19 de diciembre de 2020

Última actualización enviada que cumplió con los criterios de control de calidad

16 de diciembre de 2020

Última verificación

1 de noviembre de 2018

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • 9HB01EXT
  • 2011-003075-11

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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