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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 grudnia 2020 zaktualizowane przez: 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.

Przegląd badań

Status

Zakończony

Warunki

Interwencja / Leczenie

Szczegółowy opis

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 studiów

Interwencyjne

Zapisy (Rzeczywisty)

120

Faza

  • Faza 3

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Lokalizacje studiów

  • Afryka Południowa
    • Gauteng
      • Johannesburg, Gauteng, Afryka Południowa, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, Afryka Południowa, 7925
        • 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
  • Belgia
      • Bruxelles, Belgia, 1200
        • Research Site
      • Leuven, Belgia, 3000
        • Research Site
  • Brazylia
    • Sao Paulo
      • Campinas, Sao Paulo, Brazylia, 13083-878
        • Research Site
  • Chiny
    • Beijingshì
      • Beijing, Beijingshì, Chiny, 100005
        • Research Site
    • Guangdongsheng
      • Guangzhou, Guangdongsheng, Chiny, 510515
        • Research Site
    • Shànghaishì
      • Shanghai, Shànghaishì, Chiny, 200025
        • Research Site
    • Tianjinshì
      • Tianjing, Tianjinshì, Chiny, 300020
        • Research Site
  • Francja
    • Bouches-Du-Rhône
      • Marseille, Bouches-Du-Rhône, Francja, 13385
        • Research Site
  • Holandia
      • Utrecht, Holandia, 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
  • Irlandia
      • Dublin, Irlandia, D12 N512
        • Research Site
  • Japonia
    • Aichi-Ken
      • Nagoya-Shi, Aichi-Ken, Japonia, 466-8550
        • Research Site
    • Fukuoka-Ken
      • Kitakyushu, Fukuoka-Ken, Japonia, 807-8555
        • Research Site
    • Kanagawa-Ken
      • Kawasaki, Kanagawa-Ken, Japonia, 216-8511
        • Research Site
    • Nara-Ken
      • Kashihara-shi, Nara-Ken, Japonia, 634-8522
        • Research Site
    • Tokyo-To
      • Shinjuku-ku, Tokyo-To, Japonia, 160-0023
        • Research Site
      • Tokyo, Tokyo-To, Japonia, 167-8515
        • Research Site
  • Kanada
    • Ontario
      • Toronto, Ontario, Kanada, M5B 1W8
        • Research Site
    • Quebec
      • Montreal, Quebec, Kanada, H3T 1C5
        • Research Site
  • Niemcy
    • North Rhine-westphalia
      • Bonn, North Rhine-westphalia, Niemcy, 53127
        • Research Site
  • Polska
      • Lodz, Polska, 93-510
        • Research Site
  • Stany Zjednoczone
    • Arizona
      • Phoenix, Arizona, Stany Zjednoczone, 85016
        • Research Site
    • California
      • Sacramento, California, Stany Zjednoczone, 95817
        • Research Site
    • Colorado
      • Aurora, Colorado, Stany Zjednoczone, 80045
        • Research Site
    • Georgia
      • Atlanta, Georgia, Stany Zjednoczone, 30322
        • Research Site
    • Hawaii
      • Honolulu, Hawaii, Stany Zjednoczone, 96826
        • Research Site
    • Indiana
      • Indianapolis, Indiana, Stany Zjednoczone, 46260
        • Research Site
    • Louisiana
      • New Orleans, Louisiana, Stany Zjednoczone, 70112
        • Research Site
    • Michigan
      • East Lansing, Michigan, Stany Zjednoczone, 48823
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, Stany Zjednoczone, 15213
        • Research Site
    • Washington
      • Seattle, Washington, Stany Zjednoczone, 98104
        • Research Site
  • Szwecja
      • Malmö, Szwecja, 20502
        • Research Site
      • Stockholm, Szwecja, 17176
        • Research Site
  • Włochy
      • Florence, Włochy, 50134
        • Research Site
      • Milano, Włochy, 20122
        • Research Site
  • Zjednoczone Królestwo
    • Cambridgeshire
      • Cambridge, Cambridgeshire, Zjednoczone Królestwo, CB2 0QQ
        • Research Site
    • Greater London
      • London, Greater London, Zjednoczone Królestwo, E1 1BB
        • Research Site
      • London, Greater London, Zjednoczone Królestwo, SE1 7EH
        • Research Site
    • Hampshire
      • Basingstoke, Hampshire, Zjednoczone Królestwo, RG24 9NA
        • Research Site

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

  • Dziecko
  • Dorosły
  • Starszy dorosły

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Męski

Opis

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 studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Nielosowe
  • Model interwencyjny: Przydział równoległy
  • Maskowanie: Brak (otwarta etykieta)

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: 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.
Biologiczny: rFIXFc
Administered as specified in the treatment arm.
Inne nazwy:
  • Alproliks
  • BIIB029
  • czynnik krzepnięcia IX (rekombinowane) białko fuzyjne Fc
Eksperymentalny: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biologiczny: rFIXFc
Administered as specified in the treatment arm.
Inne nazwy:
  • Alproliks
  • BIIB029
  • czynnik krzepnięcia IX (rekombinowane) białko fuzyjne Fc

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Number of Participants With Any Positive Inhibitor Development
Ramy czasowe: 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

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Annualized Bleeding Rate (ABR)
Ramy czasowe: 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
Ramy czasowe: 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)
Ramy czasowe: 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])
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Bioverativ Therapeutics Inc.

Śledczy

  • Dyrektor Studium: Medical Director, Bioverativ Therapeutics Inc.

Publikacje i pomocne linki

Osoba odpowiedzialna za wprowadzenie informacji o badaniu dobrowolnie udostępnia te publikacje. Mogą one dotyczyć wszystkiego, co jest związane z badaniem.

Publikacje ogólne

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

8 grudnia 2011

Zakończenie podstawowe (Rzeczywisty)

1 października 2017

Ukończenie studiów (Rzeczywisty)

1 października 2017

Daty rejestracji na studia

Pierwszy przesłany

19 sierpnia 2011

Pierwszy przesłany, który spełnia kryteria kontroli jakości

29 sierpnia 2011

Pierwszy wysłany (Oszacować)

30 sierpnia 2011

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

19 grudnia 2020

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

16 grudnia 2020

Ostatnia weryfikacja

1 listopada 2018

Więcej informacji

Terminy związane z tym badaniem

Słowa kluczowe

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • 9HB01EXT
  • 2011-003075-11

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

Badania kliniczne na Ciężka hemofilia B

Badania kliniczne na rFIXFc

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