A pilot Randomised Control Trial comparing the efficacy and safety of Standard Dose and Low dose Emicizumab prophylaxis with Extended Half Life Factor VIII concentrate in children with severe Hemophilia A without Inhibitors
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- To assess and compare Annualized Bleeding Rate ( spontaneous, Traumatic, Joint bleeds) between various treatment groups
研究概览
简要总结
This is a pilot randomized controlled open label trial comparing the efficacy and safety of prophylaxis with extended half life recombinant factor VIII and two dosing regimens of emicizumab in patients with hemophilia A without inhibitors. A total of 30 participants will be enrolled, with 10 participants in each group. Randomization will be done using random number tables, and allocation concealment will be maintained using sequentially numbered sealed envelopes. Each participant will receive the assigned prophylactic regimen for six months. The primary outcome will be the annualized bleeding rate. Secondary outcomes will include Hemophilia Joint Health Score, Haemo QoL score assessed at end of 6 months and adverse events recorded at any time during the study period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 盲法
- None
入排标准
- 年龄范围
- 2.00 Year(s) 至 18.00 Year(s)（—）
- 性别
- Male
入选标准
- •Severe Hemophilia A without Inhibitors and has not been on prophylactic therapy with either Extended Half Life Factor VIII or Emicizumab.
排除标准
- •Presence of FVIII inhibitors Known case of coagulopathies and bleeding disorders other than Hemophilia A.
结局指标
主要结局
To assess and compare Annualized Bleeding Rate ( spontaneous, Traumatic, Joint bleeds) between various treatment groups
时间窗: At baseline and end of 6 months of prophylaxis
次要结局
- Hemophilia Joint Health Score (HJHS)(At baseline and and of 6 months)
- Haemo-QoL score(At baseline and end of 6 months)
- Adverse events monitoring(Any time during the study)
研究者
Aswathy V Nair
Shyam Shah Medical College and Associated SGMH, GMH