NCT02170402已完成4 期
Study to Evaluate Efficacy and Safety of ADVATE in the Treatment of Previously Treated Patients With Hemophilia A
Baxalta now part of Shire12 个研究点 分布在 1 个国家目标入组 82 人开始时间: 2014年6月26日最近更新:
适应症
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 82
- 试验地点
- 12
- 主要终点
- Percentage of reduction in annualized bleed rate (ABR) during prophylactic treatment compared to ABR during on demand treatment
研究概览
简要总结
The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs)
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Main Inclusion Criteria:
- •Ethnic Chinese
- •is of any age
- •has a documented diagnosis of severe or moderately severe hemophilia A (congenital FVIII deficiency: baseline Factor VIII (FVIII) ≤ 2%)
- •has documented and verified >50 exposure days (EDs) to FVIII (recombinant or plasma derived)
- •is receiving on-demand treatment with FVIII at the time of enrolment in this study
- •has negative history of inhibitor development
- •is HIV negative or HIV positive with stable disease and CD4+ count ≥ 200 cells per mm^3
- •is negative for Hepatitis C virus (HCV); Or participant is HCV positive with chronic stable hepatitis as assessed by investigator
排除标准
- •has prior history of hypersensitivity or anaphylaxis associated with receipt of FVIII
- •is diagnosed with other bleeding disorder(s) other than hemophilia A, including but not limited to thrombocytopenia (platelet count < 100000 /mL)
- •has been exposed to an investigational product (IP) within 30 days prior to the screening visit or is scheduled to participate in another clinical study involving an IP or investigational device during participation in the study
- •is planned, or likely to have surgery during the study period
- •has end-stage renal failure or evidence of a severe or uncontrolled systemic disease as judged by the investigator
- •has active hepatic disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 5 times the upper limit of normal)
- •has clinical or laboratory evidence of severe liver impairment including (but not limited to) a recent & persistent international normalized ratio (INR) >1.4, and/or the presence of splenomegaly and/or significant spider angioma on physical exam, and/or a history of esophageal hemorrhage or documented esophageal varices
- •is a family member of the investigator or site staff
结局指标
主要结局
Percentage of reduction in annualized bleed rate (ABR) during prophylactic treatment compared to ABR during on demand treatment
时间窗: 12 months
Computed as: {\[median ABR on-demand - median ABR prophylaxis\]÷\[median ABR on-demand\]}\*100% The ABR, will be assumed to have a negative binomial distribution. The 2 treatment regimens (on-demand and prophylaxis) will be compared in terms of mean ABR within a generalized linear model framework (with a logarithmic link function which is the default for the negative binomial distribution), accounting for the fixed effect of study arm and the follow-up time (in years) as an offset. Ratios between treatment means (95% CI) will be estimated within this model.
次要结局
- Overall evaluation of efficacy on a four-point scale (Excellent-Good-Fair-Poor)(12 months)
- Clearance (CL)(Within 30 minutes prior to the start of the infusion through 48 hours post-infusion)
- Number of units per kg body weight of ADVATE required to resolve a bleeding episode (BE)(12 months)
- Annualized bleeding episode rates (ABR) according to bleed type and bleed etiology summarized by treatment regimen(12 months)
- Inhibitor incidence(13 months)
- Adverse events according to relatedness, seriousness, and severity(13 months)
- Area under the plasma concentration/time curve from time 0 to infinity(Within 30 minutes prior to the start of the infusion through 48 hours post-infusion)
- Elimination phase half-life(Within 30 minutes prior to the start of the infusion through 48 hours post-infusion)
- Number of infusions of ADVATE required to resolve a bleeding episode (BE)(12 months)
- Mean Residence Time (MRT)(Within 30 minutes prior to the start of the infusion through 48 hours post-infusion)
- Incremental Recovery (IR) at Cmax(Within 30 minutes prior to the start of the infusion, and within 1 hour post-infusion)
- Volume of distribution at steady state (Vss)(Within 30 minutes prior to the start of the infusion through 48 hours post-infusion)
研究者
研究点 (12)
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