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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 décembre 2020 mis à jour par: 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.

Aperçu de l'étude

Statut

Complété

Les conditions

Intervention / Traitement

Description détaillée

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)

Type d'étude

Interventionnel

Inscription (Réel)

120

Phase

  • Phase 3

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Enfant
  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Homme

La description

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 d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Non randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: 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.
Biologique: rFIXFc
Administered as specified in the treatment arm.
Autres noms:
  • Alprolix
  • BIIB029
  • facteur de coagulation IX (recombinant) protéine de fusion Fc
Expérimental: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biologique: rFIXFc
Administered as specified in the treatment arm.
Autres noms:
  • Alprolix
  • BIIB029
  • facteur de coagulation IX (recombinant) protéine de fusion Fc

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Number of Participants With Any Positive Inhibitor Development
Délai: 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

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Annualized Bleeding Rate (ABR)
Délai: 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
Délai: 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)
Délai: 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])
Délai: 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
Délai: 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
Délai: 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

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Bioverativ Therapeutics Inc.

Les enquêteurs

  • Directeur d'études: Medical Director, Bioverativ Therapeutics Inc.

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Publications générales

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

8 décembre 2011

Achèvement primaire (Réel)

1 octobre 2017

Achèvement de l'étude (Réel)

1 octobre 2017

Dates d'inscription aux études

Première soumission

19 août 2011

Première soumission répondant aux critères de contrôle qualité

29 août 2011

Première publication (Estimation)

30 août 2011

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

19 décembre 2020

Dernière mise à jour soumise répondant aux critères de contrôle qualité

16 décembre 2020

Dernière vérification

1 novembre 2018

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • 9HB01EXT
  • 2011-003075-11

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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