A Prospective Study to Evaluate the Effect of rFVIII-FS in Different Prophylactic Regimens on Bleeding Events Frequency and Development of Arthropathy in Previously Treated and Minimally Treated Hemophilia A Pediatric Population.
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 32
- 主要终点
- Percentage of Participants With Less Than 2 Joint Bleeds During the 9-month Treatment Period
研究概览
简要总结
A prospective study to evaluate the effect of rFVIII-FS in different prophylactic regimens on bleeding events frequency and development of arthropathy in Previously Treated and Minimally treated Hemophilia A pediatric population.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Severe hemophilia A or moderate hemophilia A
- •1-12 years of age
- •Requiring treatment with FVIII
排除标准
- •Current or prior inhibitor or familial antecedents of inhibitor
- •Surgery required during the study (9 months)
- •Positive for HIV
研究组 & 干预措施
rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw
rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection once per week [qw] (weekly on Day 7 + 1 after previous injection) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 35 IU/kg twice a week or further escalation to 25 IU/kg three times a week)
干预措施: rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per week (Drug)
rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg)
rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection twice per week [biw] (30 IU/kg [day 1] + 40 IU/kg [day 4]) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 25 IU/kg three times a week)
干预措施: rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg) (Drug)
rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)
rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 75 IU/kg, dosing by injection three times per week [tiw] (3 x 25 IU/kg [day 1, 3, 5]) for 9 months. No escalation opportunity for participants in this group
干预措施: rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg) (Drug)
结局指标
主要结局
Percentage of Participants With Less Than 2 Joint Bleeds During the 9-month Treatment Period
时间窗: Up to 9 months
次要结局
- Number of Bleeds Per Participant During the 9-month Treatment Period(Up to 9 months)
- Number of Participants With Bleeding Events During the 9-month Treatment Period(Up to 9 months)
- Number of Participants With Joint Bleeds During the 9-month Treatment Period(Up to 9 months)
- Number of Participants in Each Group at the End of the Study(Up to 9 months)
- Actual Monthly rFVIII-FS Consumption(Up to 9 months)
- Change From Baseline in Stockholm Hemophilia Joint Score at 9 Months of Treatment(baseline and 9 months)
- Haemo-QoL Standardized Total Score at 9 Months of Treatment (Completed by Participants in the Total Group)(9 months)
- Haemo-QoL Standardized Total Score (Completed by Parents/Caregivers in the Total Group) at 9 Months of Treatment(9 months)