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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 december 2020 uppdaterad 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.

Studieöversikt

Status

Avslutad

Betingelser

Intervention / Behandling

Detaljerad beskrivning

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)

Studietyp

Interventionell

Inskrivning (Faktisk)

120

Fas

  • Fas 3

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

  • 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
  • Frankrike
    • Bouches-Du-Rhône
      • Marseille, Bouches-Du-Rhône, Frankrike, 13385
        • Research Site
  • Förenta staterna
    • Arizona
      • Phoenix, Arizona, Förenta staterna, 85016
        • Research Site
    • California
      • Sacramento, California, Förenta staterna, 95817
        • Research Site
    • Colorado
      • Aurora, Colorado, Förenta staterna, 80045
        • Research Site
    • Georgia
      • Atlanta, Georgia, Förenta staterna, 30322
        • Research Site
    • Hawaii
      • Honolulu, Hawaii, Förenta staterna, 96826
        • Research Site
    • Indiana
      • Indianapolis, Indiana, Förenta staterna, 46260
        • Research Site
    • Louisiana
      • New Orleans, Louisiana, Förenta staterna, 70112
        • Research Site
    • Michigan
      • East Lansing, Michigan, Förenta staterna, 48823
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, Förenta staterna, 15213
        • Research Site
    • Washington
      • Seattle, Washington, Förenta staterna, 98104
        • Research Site
  • Hong Kong
      • Hong Kong, Hong Kong
        • Research Site
    • New Territories
      • Hong Kong, New Territories, Hong Kong
        • 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
  • 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
  • Nederländerna
      • Utrecht, Nederländerna, 3584 CX
        • Research Site
  • Polen
      • Lodz, Polen, 93-510
        • Research Site
  • Storbritannien
    • Cambridgeshire
      • Cambridge, Cambridgeshire, Storbritannien, CB2 0QQ
        • Research Site
    • Greater London
      • London, Greater London, Storbritannien, E1 1BB
        • Research Site
      • London, Greater London, Storbritannien, SE1 7EH
        • Research Site
    • Hampshire
      • Basingstoke, Hampshire, Storbritannien, RG24 9NA
        • Research Site
  • Sverige
      • Malmö, Sverige, 20502
        • Research Site
      • Stockholm, Sverige, 17176
        • Research Site
  • Sydafrika
    • Gauteng
      • Johannesburg, Gauteng, Sydafrika, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, Sydafrika, 7925
        • Research Site
  • Tyskland
    • North Rhine-westphalia
      • Bonn, North Rhine-westphalia, Tyskland, 53127
        • Research Site

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Barn
  • Vuxen
  • Äldre vuxen

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Manlig

Beskrivning

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

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Icke-randomiserad
  • Interventionsmodell: Parallellt uppdrag
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: 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.
Andra namn:
  • Alprolix
  • BIIB029
  • koagulationsfaktor IX (rekombinant) Fc-fusionsprotein
Experimentell: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biologisk: rFIXFc
Administered as specified in the treatment arm.
Andra namn:
  • Alprolix
  • BIIB029
  • koagulationsfaktor IX (rekombinant) Fc-fusionsprotein

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Number of Participants With Any Positive Inhibitor Development
Tidsram: 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Annualized Bleeding Rate (ABR)
Tidsram: 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
Tidsram: 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)
Tidsram: 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])
Tidsram: 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
Tidsram: 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
Tidsram: 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

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Bioverativ Therapeutics Inc.

Utredare

  • Studierektor: Medical Director, Bioverativ Therapeutics Inc.

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Allmänna publikationer

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

8 december 2011

Primärt slutförande (Faktisk)

1 oktober 2017

Avslutad studie (Faktisk)

1 oktober 2017

Studieregistreringsdatum

Först inskickad

19 augusti 2011

Först inskickad som uppfyllde QC-kriterierna

29 augusti 2011

Första postat (Uppskatta)

30 augusti 2011

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

19 december 2020

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

16 december 2020

Senast verifierad

1 november 2018

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • 9HB01EXT
  • 2011-003075-11

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

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