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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 bijgewerkt door: 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.

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

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

Ingrijpend

Inschrijving (Werkelijk)

120

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • Australië
    • South Australia
      • Adelaide, South Australia, Australië, 5000
        • Research Site
    • Victoria
      • Parkville, Victoria, Australië, 3052
        • Research Site
    • Western Australia
      • Murdoch, Western Australia, Australië, 6150
        • Research Site
      • Perth, Western Australia, Australië, 6008
        • Research Site
  • België
      • Bruxelles, België, 1200
        • Research Site
      • Leuven, België, 3000
        • Research Site
  • Brazilië
    • Sao Paulo
      • Campinas, Sao Paulo, Brazilië, 13083-878
        • Research Site
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5B 1W8
        • Research Site
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • Research Site
  • China
    • Beijingshì
      • Beijing, Beijingshì, China, 100005
        • Research Site
    • Guangdongsheng
      • Guangzhou, Guangdongsheng, China, 510515
        • Research Site
    • Shànghaishì
      • Shanghai, Shànghaishì, China, 200025
        • Research Site
    • Tianjinshì
      • Tianjing, Tianjinshì, China, 300020
        • Research Site
  • Duitsland
    • North Rhine-westphalia
      • Bonn, North Rhine-westphalia, Duitsland, 53127
        • Research Site
  • Frankrijk
    • Bouches-Du-Rhône
      • Marseille, Bouches-Du-Rhône, Frankrijk, 13385
        • Research Site
  • Hongkong
      • Hong Kong, Hongkong
        • Research Site
    • New Territories
      • Hong Kong, New Territories, Hongkong
        • Research Site
  • Ierland
      • Dublin, Ierland, D12 N512
        • Research Site
  • Indië
    • Karnataka
      • Bangalore, Karnataka, Indië, 560034
        • Research Site
    • Maharashtra
      • Pune, Maharashtra, Indië, 411004
        • Research Site
    • Tamil Nadu
      • Vellore, Tamil Nadu, Indië, 632004
        • Research Site
  • Italië
      • Florence, Italië, 50134
        • Research Site
      • Milano, Italië, 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
  • Nederland
      • Utrecht, Nederland, 3584 CX
        • Research Site
  • Polen
      • Lodz, Polen, 93-510
        • Research Site
  • Verenigd Koninkrijk
    • Cambridgeshire
      • Cambridge, Cambridgeshire, Verenigd Koninkrijk, CB2 0QQ
        • Research Site
    • Greater London
      • London, Greater London, Verenigd Koninkrijk, E1 1BB
        • Research Site
      • London, Greater London, Verenigd Koninkrijk, SE1 7EH
        • Research Site
    • Hampshire
      • Basingstoke, Hampshire, Verenigd Koninkrijk, RG24 9NA
        • Research Site
  • Verenigde Staten
    • Arizona
      • Phoenix, Arizona, Verenigde Staten, 85016
        • Research Site
    • California
      • Sacramento, California, Verenigde Staten, 95817
        • Research Site
    • Colorado
      • Aurora, Colorado, Verenigde Staten, 80045
        • Research Site
    • Georgia
      • Atlanta, Georgia, Verenigde Staten, 30322
        • Research Site
    • Hawaii
      • Honolulu, Hawaii, Verenigde Staten, 96826
        • Research Site
    • Indiana
      • Indianapolis, Indiana, Verenigde Staten, 46260
        • Research Site
    • Louisiana
      • New Orleans, Louisiana, Verenigde Staten, 70112
        • Research Site
    • Michigan
      • East Lansing, Michigan, Verenigde Staten, 48823
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, Verenigde Staten, 15213
        • Research Site
    • Washington
      • Seattle, Washington, Verenigde Staten, 98104
        • Research Site
  • Zuid-Afrika
    • Gauteng
      • Johannesburg, Gauteng, Zuid-Afrika, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, Zuid-Afrika, 7925
        • Research Site
  • Zweden
      • Malmö, Zweden, 20502
        • Research Site
      • Stockholm, Zweden, 17176
        • Research Site

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Kind
  • Volwassen
  • Oudere volwassene

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Mannelijk

Beschrijving

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: 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.
Biologisch: rFIXFc
Administered as specified in the treatment arm.
Andere namen:
  • Alprolix
  • BIIB029
  • stollingsfactor IX (recombinant) Fc-fusie-eiwit
Experimenteel: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biologisch: rFIXFc
Administered as specified in the treatment arm.
Andere namen:
  • Alprolix
  • BIIB029
  • stollingsfactor IX (recombinant) Fc-fusie-eiwit

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Number of Participants With Any Positive Inhibitor Development
Tijdsspanne: 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

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Annualized Bleeding Rate (ABR)
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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])
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Bioverativ Therapeutics Inc.

Onderzoekers

  • Studie directeur: Medical Director, Bioverativ Therapeutics Inc.

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

8 december 2011

Primaire voltooiing (Werkelijk)

1 oktober 2017

Studie voltooiing (Werkelijk)

1 oktober 2017

Studieregistratiedata

Eerst ingediend

19 augustus 2011

Eerst ingediend dat voldeed aan de QC-criteria

29 augustus 2011

Eerst geplaatst (Schatting)

30 augustus 2011

Updates van studierecords

Laatste update geplaatst (Werkelijk)

19 december 2020

Laatste update ingediend die voldeed aan QC-criteria

16 december 2020

Laatst geverifieerd

1 november 2018

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • 9HB01EXT
  • 2011-003075-11

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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