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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)

2020年12月16日 更新者：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.

研究概览

地位

完全的

条件

干预/治疗

详细说明

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)

研究类型

介入性

注册 (实际的)

120

阶段

  • 第三阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 中国
    • Beijingshì
      • Beijing、Beijingshì、中国、100005
        • Research Site
    • Guangdongsheng
      • Guangzhou、Guangdongsheng、中国、510515
        • Research Site
    • Shànghaishì
      • Shanghai、Shànghaishì、中国、200025
        • Research Site
    • Tianjinshì
      • Tianjing、Tianjinshì、中国、300020
        • Research Site
  • 加拿大
    • Ontario
      • Toronto、Ontario、加拿大、M5B 1W8
        • Research Site
    • Quebec
      • Montreal、Quebec、加拿大、H3T 1C5
        • Research Site
  • 南非
    • Gauteng
      • Johannesburg、Gauteng、南非、2193
        • Research Site
    • Western Cape
      • Cape Town、Western Cape、南非、7925
        • Research Site
  • 印度
    • Karnataka
      • Bangalore、Karnataka、印度、560034
        • Research Site
    • Maharashtra
      • Pune、Maharashtra、印度、411004
        • Research Site
    • Tamil Nadu
      • Vellore、Tamil Nadu、印度、632004
        • Research Site
  • 巴西
    • Sao Paulo
      • Campinas、Sao Paulo、巴西、13083-878
        • Research Site
  • 德国
    • North Rhine-westphalia
      • Bonn、North Rhine-westphalia、德国、53127
        • Research Site
  • 意大利
      • Florence、意大利、50134
        • Research Site
      • Milano、意大利、20122
        • Research Site
  • 日本
    • Aichi-Ken
      • Nagoya-Shi、Aichi-Ken、日本、466-8550
        • Research Site
    • Fukuoka-Ken
      • Kitakyushu、Fukuoka-Ken、日本、807-8555
        • Research Site
    • Kanagawa-Ken
      • Kawasaki、Kanagawa-Ken、日本、216-8511
        • Research Site
    • Nara-Ken
      • Kashihara-shi、Nara-Ken、日本、634-8522
        • Research Site
    • Tokyo-To
      • Shinjuku-ku、Tokyo-To、日本、160-0023
        • Research Site
      • Tokyo、Tokyo-To、日本、167-8515
        • Research Site
  • 比利时
      • Bruxelles、比利时、1200
        • Research Site
      • Leuven、比利时、3000
        • Research Site
  • 法国
    • Bouches-Du-Rhône
      • Marseille、Bouches-Du-Rhône、法国、13385
        • Research Site
  • 波兰
      • Lodz、波兰、93-510
        • Research Site
  • 澳大利亚
    • South Australia
      • Adelaide、South Australia、澳大利亚、5000
        • Research Site
    • Victoria
      • Parkville、Victoria、澳大利亚、3052
        • Research Site
    • Western Australia
      • Murdoch、Western Australia、澳大利亚、6150
        • Research Site
      • Perth、Western Australia、澳大利亚、6008
        • Research Site
  • 爱尔兰
      • Dublin、爱尔兰、D12 N512
        • Research Site
  • 瑞典
      • Malmö、瑞典、20502
        • Research Site
      • Stockholm、瑞典、17176
        • Research Site
  • 美国
    • Arizona
      • Phoenix、Arizona、美国、85016
        • Research Site
    • California
      • Sacramento、California、美国、95817
        • Research Site
    • Colorado
      • Aurora、Colorado、美国、80045
        • Research Site
    • Georgia
      • Atlanta、Georgia、美国、30322
        • Research Site
    • Hawaii
      • Honolulu、Hawaii、美国、96826
        • Research Site
    • Indiana
      • Indianapolis、Indiana、美国、46260
        • Research Site
    • Louisiana
      • New Orleans、Louisiana、美国、70112
        • Research Site
    • Michigan
      • East Lansing、Michigan、美国、48823
        • Research Site
    • Pennsylvania
      • Pittsburgh、Pennsylvania、美国、15213
        • Research Site
    • Washington
      • Seattle、Washington、美国、98104
        • Research Site
  • 英国
    • Cambridgeshire
      • Cambridge、Cambridgeshire、英国、CB2 0QQ
        • Research Site
    • Greater London
      • London、Greater London、英国、E1 1BB
        • Research Site
      • London、Greater London、英国、SE1 7EH
        • Research Site
    • Hampshire
      • Basingstoke、Hampshire、英国、RG24 9NA
        • Research Site
  • 荷兰
      • Utrecht、荷兰、3584 CX
        • Research Site
  • 香港
      • Hong Kong、香港
        • Research Site
    • New Territories
      • Hong Kong、New Territories、香港
        • Research Site

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 孩子
  • 成人
  • 年长者

接受健康志愿者

不

有资格学习的性别

男性

描述

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.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：非随机化
  • 介入模型：并行分配
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：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.
生物：rFIXFc
Administered as specified in the treatment arm.
其他名称：
  • 艾普力司
  • BIIB029
  • 凝血因子 IX（重组）Fc 融合蛋白
实验性的：Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
生物：rFIXFc
Administered as specified in the treatment arm.
其他名称：
  • 艾普力司
  • BIIB029
  • 凝血因子 IX（重组）Fc 融合蛋白

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Number of Participants With Any Positive Inhibitor Development
大体时间：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

次要结果测量

结果测量
措施说明
大体时间
Annualized Bleeding Rate (ABR)
大体时间：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
大体时间：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)
大体时间：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])
大体时间：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
大体时间：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
大体时间：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

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Bioverativ Therapeutics Inc.

调查人员

  • 研究主任：Medical Director、Bioverativ Therapeutics Inc.

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

一般刊物

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2011年12月8日

初级完成 (实际的)

2017年10月1日

研究完成 (实际的)

2017年10月1日

研究注册日期

首次提交

2011年8月19日

首先提交符合 QC 标准的

2011年8月29日

首次发布 (估计)

2011年8月30日

研究记录更新

最后更新发布 (实际的)

2020年12月19日

上次提交的符合 QC 标准的更新

2020年12月16日

最后验证

2018年11月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • 9HB01EXT
  • 2011-003075-11

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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    美国
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    Golcadomide和Rituximab作为桥接疗法，用于复发或难治性侵袭性B细胞非霍奇金淋巴瘤
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    复发性弥漫性大 B 细胞淋巴瘤 | 难治性弥漫性大 B 细胞淋巴瘤 | 伴有 MYC、BCL2 和 BCL6 重排的复发性高级别 B 细胞淋巴瘤 | 具有 MYC、BCL2 和 BCL6 重排的难治性高级 B 细胞淋巴瘤 | 复发性弥漫性大 B 细胞淋巴瘤，未另行说明 | 伴有 MYC 和 BCL2 或 BCL6 重排的复发性高级别 B 细胞淋巴瘤 | 难治性弥漫性大 B 细胞淋巴瘤，未另行说明 | 具有 MYC 和 BCL2 或 BCL6 重排的难治性高级 B 细胞淋巴瘤 | 复发性 T 细胞/富含组织细胞的大 B 细胞淋巴瘤 | 难治性 T 细胞/富含组织细胞的大 B 细胞淋巴瘤 | 复发性高级 B 细胞淋巴瘤，未另行说明 | 难治性高级... 及其他条件
    美国

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