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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. prosince 2020 aktualizováno: 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.

Přehled studie

Postavení

Dokončeno

Podmínky

Intervence / Léčba

Detailní popis

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)

Typ studie

Intervenční

Zápis (Aktuální)

120

Fáze

  • Fáze 3

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní místa

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

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

  • Dítě
  • Dospělý
  • Starší dospělý

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Mužský

Popis

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.

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: Nerandomizované
  • Intervenční model: Paralelní přiřazení
  • Maskování: Žádné (otevřený štítek)

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: 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.
Biologický: rFIXFc
Administered as specified in the treatment arm.
Ostatní jména:
  • Alprolix
  • BIIB029
  • koagulační faktor IX (rekombinantní) Fc fúzní protein
Experimentální: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biologický: rFIXFc
Administered as specified in the treatment arm.
Ostatní jména:
  • Alprolix
  • BIIB029
  • koagulační faktor IX (rekombinantní) Fc fúzní protein

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Number of Participants With Any Positive Inhibitor Development
Časové okno: 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

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Annualized Bleeding Rate (ABR)
Časové okno: 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
Časové okno: 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)
Časové okno: 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])
Časové okno: 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
Časové okno: 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
Časové okno: 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

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Bioverativ Therapeutics Inc.

Vyšetřovatelé

  • Ředitel studie: Medical Director, Bioverativ Therapeutics Inc.

Publikace a užitečné odkazy

Osoba odpovědná za zadávání informací o studiu tyto publikace poskytuje dobrovolně. Mohou se týkat čehokoli, co souvisí se studiem.

Obecné publikace

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Aktuální)

8. prosince 2011

Primární dokončení (Aktuální)

1. října 2017

Dokončení studie (Aktuální)

1. října 2017

Termíny zápisu do studia

První předloženo

19. srpna 2011

První předloženo, které splnilo kritéria kontroly kvality

29. srpna 2011

První zveřejněno (Odhad)

30. srpna 2011

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

19. prosince 2020

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

16. prosince 2020

Naposledy ověřeno

1. listopadu 2018

Více informací

Termíny související s touto studií

Klíčová slova

Další relevantní podmínky MeSH

Další identifikační čísla studie

  • 9HB01EXT
  • 2011-003075-11

Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .

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