AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 23
- 试验地点
- 22
- 主要终点
- Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
研究概览
简要总结
Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia. Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study. The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.
详细描述
Regulatory Commitment
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- — 至 6 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
- •No prior exposure to factor products or any blood products.
排除标准
- •Presence of any bleeding disorder in addition to hemophilia A.
- •Treatment with any investigational agent or device within the past 30 days.
- •Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).
研究组 & 干预措施
1
The investigator treats subjects with ReFacto AF in the usual care setting.
干预措施: Laboratory Tests (Procedure)
结局指标
主要结局
Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
时间窗: 2 years
Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.
次要结局
- Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF(2 years)
- Annualized Bleeding Rate (ABR)(2 years)
- Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion(Within 48 hours after infusion, up to 2 years treatment duration)
- Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF(2 years)
- Consumption of Total International Units of Factor VIII(2 years)
- Consumption of Total International Units of Factor VIII Per Year(2 years)
- Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight(2 years)
- Mean of Total Number of Infusions of Study Drug Received(2 years)
- Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting(2 years)
- Mean Dose (IU) of Study Drug Consumed Per Infusion(2 years)
- Consumption of Total International Units of Factor VIII by Weight(2 years)
- Consumption of Total International Units of Factor VIII Per Year by Weight(2 years)
- Mean of Total Number of Days Participants Exposed to Study Drug(2 years)
- Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study(2 years)
- Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting(2 years)
- Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting(2 years)