NCT02234310已完成3 期
An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B
Bioverativ, a Sanofi company29 个研究点 分布在 11 个国家目标入组 33 人开始时间: 2014年11月13日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 33
- 试验地点
- 29
- 主要终点
- Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
研究概览
简要总结
The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B. Secondary objectives were to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in PUPs, and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in PUPs.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 17 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Weight >=3.5 kilogram at the time of informed consent.
- •Severe hemophilia B was defined as less than or equal to (<=)2 International Units per deciliter (IU/dL) (<=2 percent [%]) endogenous FIX documented in the medical record or as tested during the Screening Period.
排除标准
- •History of positive inhibitor testing. A prior history of inhibitors was defined based on a participant's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (that is equal to or above lower limit of detection).
- •History of hypersensitivity reactions associated with any rFIXFc administration.
- •Exposure to blood components or injection with a coagulation factor IX (FIX) concentrate (including plasma derived) other than rFIXFc.
- •Injection with commercially available rFIXFc more than 28 days prior to Screening.
- •More than 3 injections of commercially available rFIXFc prior to confirmation of eligibility.
- •Other coagulation disorders in addition to hemophilia B.
- •Any concurrent clinically significant major disease that, in the opinion of the Investigator, would have made the participant unsuitable for enrollment (example HIV infection with cluster of differentiation 4 (CD4) lymphocyte count less than (<)200 cells/microliter (mcL) or a viral load greater than (>)200 particles/mcL, or any other known congenital or acquired immunodeficiency).
- •Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of steroids for treatment of asthma or management of acute allergic episodes or otherwise life-threatening episodes was allowed. Treatment in these circumstances should not have exceeded a 14-day duration.
- •Participation within the past 30 days in any other clinical study involving investigational treatment.
- •Current enrollment in any other clinical study involving investigational treatment.
- •Inability to comply with study requirements.
- •Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, would have made the participant unsuitable for enrollment.
- •NOTE: Other protocol-defined inclusion/exclusion criteria may apply.
结局指标
主要结局
Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
时间窗: Up to 3 years
Development of an inhibitor was defined as an inhibitor test result of \>= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of \>=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay.
次要结局
- Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])(Up to 3 years)
- Number of Injections of rFIXFc Required to Resolve a Bleeding Episode(Up to 3 years)
- Change From Baseline in rFIXFc Incremental Recovery (IR)(Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144)
- Total Number of Exposure Days (EDs)(Up to 3 years)
- Annualized Number of Spontaneous Joint Bleeding Episodes(Up to 3 years)
- Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale(Up to 3 years)
- Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes(Up to 3 years)
- Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode(Up to 3 years)
研究者
研究点 (29)
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