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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. desember 2020 oppdatert av: 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.

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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)

Studietype

Intervensjonell

Registrering (Faktiske)

120

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • 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
  • Belgia
      • Bruxelles, Belgia, 1200
        • Research Site
      • Leuven, Belgia, 3000
        • Research Site
  • Brasil
    • Sao Paulo
      • Campinas, Sao Paulo, Brasil, 13083-878
        • Research Site
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5B 1W8
        • Research Site
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • Research Site
  • Forente stater
    • Arizona
      • Phoenix, Arizona, Forente stater, 85016
        • Research Site
    • California
      • Sacramento, California, Forente stater, 95817
        • Research Site
    • Colorado
      • Aurora, Colorado, Forente stater, 80045
        • Research Site
    • Georgia
      • Atlanta, Georgia, Forente stater, 30322
        • Research Site
    • Hawaii
      • Honolulu, Hawaii, Forente stater, 96826
        • Research Site
    • Indiana
      • Indianapolis, Indiana, Forente stater, 46260
        • Research Site
    • Louisiana
      • New Orleans, Louisiana, Forente stater, 70112
        • Research Site
    • Michigan
      • East Lansing, Michigan, Forente stater, 48823
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, Forente stater, 15213
        • Research Site
    • Washington
      • Seattle, Washington, Forente stater, 98104
        • Research Site
  • Frankrike
    • Bouches-Du-Rhône
      • Marseille, Bouches-Du-Rhône, Frankrike, 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
  • Irland
      • Dublin, Irland, D12 N512
        • Research Site
  • Italia
      • Florence, Italia, 50134
        • Research Site
      • Milano, Italia, 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
  • Kina
    • Beijingshì
      • Beijing, Beijingshì, Kina, 100005
        • Research Site
    • Guangdongsheng
      • Guangzhou, Guangdongsheng, Kina, 510515
        • Research Site
    • Shànghaishì
      • Shanghai, Shànghaishì, Kina, 200025
        • Research Site
    • Tianjinshì
      • Tianjing, Tianjinshì, Kina, 300020
        • Research Site
  • Nederland
      • Utrecht, Nederland, 3584 CX
        • Research Site
  • Polen
      • Lodz, Polen, 93-510
        • Research Site
  • Storbritannia
    • Cambridgeshire
      • Cambridge, Cambridgeshire, Storbritannia, CB2 0QQ
        • Research Site
    • Greater London
      • London, Greater London, Storbritannia, E1 1BB
        • Research Site
      • London, Greater London, Storbritannia, SE1 7EH
        • Research Site
    • Hampshire
      • Basingstoke, Hampshire, Storbritannia, RG24 9NA
        • Research Site
  • Sverige
      • Malmö, Sverige, 20502
        • Research Site
      • Stockholm, Sverige, 17176
        • Research Site
  • Sør-Afrika
    • Gauteng
      • Johannesburg, Gauteng, Sør-Afrika, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, Sør-Afrika, 7925
        • Research Site
  • Tyskland
    • North Rhine-westphalia
      • Bonn, North Rhine-westphalia, Tyskland, 53127
        • Research Site

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn
  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Mann

Beskrivelse

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.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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.
Biologisk: rFIXFc
Administered as specified in the treatment arm.
Andre navn:
  • Alprolix
  • BIIB029
  • koagulasjonsfaktor IX (rekombinant) Fc-fusjonsprotein
Eksperimentell: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biologisk: rFIXFc
Administered as specified in the treatment arm.
Andre navn:
  • Alprolix
  • BIIB029
  • koagulasjonsfaktor IX (rekombinant) Fc-fusjonsprotein

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Number of Participants With Any Positive Inhibitor Development
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Annualized Bleeding Rate (ABR)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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])
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Bioverativ Therapeutics Inc.

Etterforskere

  • Studieleder: Medical Director, Bioverativ Therapeutics Inc.

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

8. desember 2011

Primær fullføring (Faktiske)

1. oktober 2017

Studiet fullført (Faktiske)

1. oktober 2017

Datoer for studieregistrering

Først innsendt

19. august 2011

Først innsendt som oppfylte QC-kriteriene

29. august 2011

Først lagt ut (Anslag)

30. august 2011

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

19. desember 2020

Siste oppdatering sendt inn som oppfylte QC-kriteriene

16. desember 2020

Sist bekreftet

1. november 2018

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 9HB01EXT
  • 2011-003075-11

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