An Open-Label Study To Evaluate The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age In Usual Care Settings
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 1
- 主要终点
- Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
研究概览
简要总结
This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old. Annualized bleeding rates and physician / caregiver assessments of responses to treatment will be characterized. FVIII inhibitor levels will be assessed throughout the study.
详细描述
The study was terminated on 22 Sept 2009 due to competition with another Wyeth study for a similar patient population. The decision to terminate the trial was not based on any safety issues.
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
- 0 Years 至 5 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male patients less than 6 years of age with moderately severe to severe hemophilia A (FVIII less than or equal to 2%).
- •Treatment history of less than 50 exposure days to prior recombinant or plasma-derived FVIII replacement products.
- •Not receiving treatment for HIV or hepatitis infection, or the patient is on a stable antiviral regimen at the time of enrollment in the study.
排除标准
- •Presence of any bleeding disorder in addition to hemophilia A.
- •Inhibitor titer of greater than or equal to 5 Bethesda Units (BU) at screening.
- •Treated with immunomodulatory therapy during the screening period
- •Treatment history of more than 5 exposure days (ED) to Xyntha.
- •Known hypersensitivity to hamster protein.
结局指标
主要结局
Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
时间窗: Baseline to 24 months or early withdrawal.
Incidence of inhibitor development was defined as any result determined positive at a central laboratory (Bethesda inhibitor titer of \>=0.6 BU/mL) using Nijmegen modification of the Bethesda assay.
Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting
时间窗: Baseline to 24 months or early withdrawal.
LETE in the on-demand setting was based on the response to the treatment of a bleeding episode. LETE in the on-demand setting occurred if the participant recorded 2 successive "No Response" ratings (indicated there was no improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsened) after 2 successive Xyntha infusions, respectively. The infusions was to be administered within 24 hours (=\<24 hours) of each other for the treatment of the same bleeding event in the absence of confounding factor.
Percentage of Participants With LETE in the Prophylaxis Setting
时间窗: Baseline to 24 months or early withdrawal.
The LETE in the prophylaxis setting was the occurrence of a bleed. LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (=\<48 hours) after a regularly scheduled prophylactic dose of Xyntha (which was not used to treat a bleed) in the absence of confounding factors.
Percentage of Participants With Low Recovery LETE
时间窗: Baseline to 24 months or early withdrawal.
The LETE could be considered lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors.
次要结局
- Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver(Baseline to 24 months or early withdrawal)
- Mean Annualized Bleed Rate (ABR)(Baseline to 24 months or early withdrawal.)
- Number of Xyntha Infusions Needed to Treat Each New Bleed(Baseline to 24 months or early withdrawal.)
- Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds(Baseline to 24 months or early withdrawal.)