An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With Xyntha (B Domain Deleted Recombinant Factor VIII, Albumin Free) In Chinese Subjects With Hemophilia A
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 53
- 试验地点
- 1
- 主要终点
- Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion
研究概览
简要总结
This study will evaluate the safety and efficacy of on-demand treatment with Xyntha in Chinese hemophilia A subjects.
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia A (FVIII activity: more than 5%, 1-5%, or less than 1%, respectively)
- •Subjects with previous exposure to FVIII replacement therapy
- •If human immunodeficiency virus (HIV) positive, documented cluster of differentiation (CD4) count more than 200/µL within 6 months of study entry
排除标准
- •Diagnosed with any bleeding disorder in addition to hemophilia A
- •Current FVIII inhibitor or history of FVIII inhibitor (defined as positive result of the reporting laboratory)
- •Subject has no history of exposure to FVIII products (previously untreated patient [PUP])
- •Subject is currently utilizing primary FVIII prophylaxis
- •Subjects anticipating elective surgery that may be planned to occur in the 6 months following study entry
- •Treated with immunomodulatory therapy within 30 days prior to study entry or planned use for the duration of their study participation
- •Participated in another investigational drug or device study within 30 days prior to study entry or planned participation for the duration of their study participation
- •Subjects with a known hypersensitivity to hamster protein
- •Significant hepatic or renal impairment (alanine aminotransferase [ALT] and aspartate aminotransferase [AST] >5 x upper limit of normal [ULN], bilirubin >2 mg/dL or serum creatinine >1.25 x ULN)
- •Prothrombin Time >1.5 x ULN
- •Platelet count <80,000 / µL
- •Pregnant or breastfeeding women
- •Unwilling or unable to follow the terms of the protocol
- •Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation, in the opinion of the Investigator or Sponsor
研究组 & 干预措施
Xyntha
This trial was an open-label and included assessments of safety, clinical efficacy, and Factor VIII (FVIII) recovery in Chinese subjects with hemophilia A. Subjects received on-demand treatments with Xyntha over a 6-month (calendar day) period.
干预措施: Xyntha (Biological)
结局指标
主要结局
Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion
时间窗: 8 hours post infusion
The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion
时间窗: 24 hours post infusion
The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Number of Participants With Factor VIII (FVIII) Inhibitor Development
时间窗: Day 1 and Month 6 or Early Termination Visit
Incidence of FVIII inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory. Incidence was stratified by participant exposure history: Minimally Treated Patients (MTPs): those who had received at least 1 prior FVIII infusion, and \<= 100 documented Exposure Days (EDs), while Previously Treated Patients (PTPs): those who had received \>100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.
次要结局
- Number of Participants With Thrombosis(Baseline up to 6 months)
- FVIII Recovery : Change From Baseline in FVIII Concentration(Day 1 and Month 6 or Early Termination Visit)
- Number of Participants With Less Than Expected Therapeutic Effect (LETE)(24 hours after each of 2 successive infusion, up to 6 months)
- Number of Participants With Thrombosis Allergic-Type Reactions(Baseline up to 6 months)