An Open-label, Single-arm, Post-authorization Pragmatic Clinical Trial On The Safety And Efficacy Of Benefix (Nonacog Alfa, Recombinant Factor Ix) In Subjects With Hemophilia B In Usual Care Settings In China
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 70
- 试验地点
- 16
- 主要终点
- Percentage of Participants Who Developed Factor IX Inhibitor
研究概览
简要总结
The purpose of this post-approval study is to evaluate the safety and efficacy of Benefix in subjects with hemophilia B in usual care settings in China.
详细描述
The purpose of this post-approval study is to provide supplementary information relating to the use of BeneFIX in Chinese subjects with hemophilia B, especially on the safety and efficacy in different populations of Chinese hemophilia B patients, in particular in pediatric patients <6 years of age, pediatric patients ≥6 to ≤12 years of age, Previously Untreated Patients (PUPs) , subjects receiving prophylaxis treatment after enrollment in the study, and severe patients (FIX activity <1%).
研究设计
- 研究类型
- Interventional
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and/or female subjects with hemophilia B.
- •Subjects/parents/legal representatives must be able to comply with study procedures (informed consent/assent process, clinical visits, reporting of infusion and bleed data, reporting of adverse events, etc)
排除标准
- •Presence of any other bleeding disorder in addition to hemophilia B. Subjects with a past history of, or current factor IX inhibitor. For laboratory-based assessments, any Bethesda inhibitor titer greater than the laboratory's normal range or ≥0.6 Bethesda Unit (BU)/mL.
- •Subjects with known hypersensitivity to the active substance or to any of the excipients of BeneFIX.
- •Subjects with a known hypersensitivity to Chinese Hamster Ovary cell proteins.
研究组 & 干预措施
Benefix
This is a single arm study. Subjects will be treated with Benefix by the investigator according to usual care in China and in accord with the China BeneFIX Package Insert.
干预措施: Benefix (Drug)
结局指标
主要结局
Percentage of Participants Who Developed Factor IX Inhibitor
时间窗: Up to 6 months
Factor IX (FIX) inhibitor development was defined as any Bethesda inhibitor titer greater than the laboratory's normal range or Bethesda inhibitor titer \>=0.6 Bethesda Unit (BU)/mL.
Number of Participants With Allergic Reactions
时间窗: Up to 6 months
FIX product allergy was defined as a hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgments.
Number of Participants With Thrombotic Events
时间窗: Up to 6 months
Thrombotic event was defined as any event associated with formation of a blood clot including catheter-associated thrombi and thrombotic complications.
次要结局
- Average Infusion Dose and Total Factor IX Consumption in Recovery Setting(Up to 6 months or 50 exposure days whichever occurred first)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)(Up to 7 months (28 calendar days after end of 6-month or 50-exposure day study treatment))
- Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response(Up to 6 months or 50 exposure days whichever occurred first)
- Number of BeneFIX Infusions to Treat Each New Bleed(Up to 6 months or 50 exposure days whichever occurred first)
- Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in Prophylaxis Setting(Up to 6 months or 50 exposure days whichever occurred first)
- Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period(Up to 6 months or 50 exposure days whichever occurred first)
- Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX(Up to 6 months or 50 exposure days whichever occurred first)
- Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period(Up to 6 months or 50 exposure days whichever occurred first)
- Average Infusion Dose and Total Factor IX Consumption in Prophylaxis Setting(Up to 6 months or 50 exposure days whichever occurred first)
- Average Infusion Dose and Total Factor IX Consumption in On-demand Setting(Up to 6 months or 50 exposure days whichever occurred first)
- Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in On-demand Setting(Up to 6 months or 50 exposure days whichever occurred first)