Comparison of Low Dose Emicizumab and Low Dose Factor VIII as a Prophylaxis in Hemophilia A Patients- a Randomized Control Trial'
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Mean of Annualized bleeding rate (ABR) among the study groups will be compared.
研究概览
简要总结
Low dose factor VIII prophylaxis is practiced around the world. Role of standard dose Emicizumab prophylaxis is well established. Emicizumab is an expensive drug. Standard dose Emicizumab prophylaxis is very expensive for hemophilia A patients and troublesome for government to ensure continuous supply. This study intends to compare low dose Factor VIII prophylaxis with low dose Emicizumab prophylaxis and compare whether low dose Emicizumab is as effective as low dose Factor VIII prophylaxis. So, it is possible to continue prophylaxis program in hemophilia A patients with a cost-effective way in our country without risking the patient health. Moreover, Emicizumab prophylaxis reduces the chance of developing inhibitor to Factor VIII and it is convenient for administration due to less frequent and subcutaneous administration. 20 severe hemophilia A patients will be selected randomly from interested patients for this study. 6 patients with inhibitor will be randomized in Low dose Emicizumab with Inhibitor group (I) and rest 14 will be randomized by block randomization in Low dose Emicizumab (without inhibitor-WI) and low dose Factor VIII group at 4:10 ratio. Initial loading dose will be given and the participants will be followed up for 6 months. At the end of the study Annualized bleeding rate (ABR), Annualized joint bleeding rate (AJBR), Annualized spontaneous bleeding rate (ASBR), APTT will be compared among the groups. Inhibitor to factor VIII will also be evaluated after six months in Factor VIII group.
详细描述
Selection criteria for the study- Severe hemophilia A patients with or without inhibitor to factor VIII will be eligible to participate in the study. Patients with inhibitor will not be included in factor VII group.
Sample Size: Sample size is determined by availability of drugs rather than any scientific formula. The investigators can provide Emicizumab to 10 patients for 28 weeks as prophylaxis. So, the investigators intend to include 10 patients in each group.
Sampling technique: The investigators will select 20 severe hemophilia A patients randomly from interested patients. The investigators will randomize 6 patients with inhibitor in Low dose Emicizumab with Inhibitor group (I) and rest 14 will be randomized by block randomization in Low dose Emicizumab (without inhibitor-WI) and low dose Factor VIII group at 4:10 ratio Variables to be studied- A. Independent variable
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Socio-demographic variables- Age, Educational status, Weight, Occupation
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Variables related to Hemophilia- Presence of Inhibitor B. Dependent variable
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Main outcome variables- Annualized bleeding rate (ABR), Annualized joint bleeding rate (AJBR), Annualized spontaneous bleeding rate (ASBR), APTT 2. Confounding variables- Development of new inhibitor to factor VIII Patients and/or parents will be thoroughly informed about the study, drugs to be used, risk & benefits and follow up plan. Their consent for this study will be taken and they will be enrolled in the study. For Participants under 18 years, consent will be obtained from parents or legal guardian. Participants ≥18 years will give his/her own consent. Randomization will be generated by computer.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Severe hemophilia A patient with or without inhibitor to factor VIII.
排除标准
- •Severe hemophilia A patient not willing to participate.
研究组 & 干预措施
Emicizumab Group- WI
Severe hemophilia A Patients without inhibitor (Emicizumab Group):
干预措施: Emicizumab Injection (Drug)
Factor VIII Group
Severe hemophilia A Patients without inhibitor.
干预措施: Factor VIII (FVIII) (Drug)
Emicizumab Group- I
Severe hemophilia A patients with inhibitor
干预措施: Emicizumab Injection (Drug)
结局指标
主要结局
Mean of Annualized bleeding rate (ABR) among the study groups will be compared.
时间窗: 06 months
ABR will be calculated as the number of reported bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant total bleeding event are 12 in 6 months, ABR of this patients is 12/6\*12=24
Mean of Annualized joint bleeding rate (AJBR) among the study groups will be compared.
时间窗: 06 months
AJBR will be calculated as the number of reported joint bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant Joint Bleeding event is 6 in 6 months than AJBR will be 6/6\*12=12.
Mean of Annualized spontaneous bleeding rate (ASBR) among the study groups will be compared.
时间窗: 06 months
ASBR will be calculated as the number of reported spontaneous bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant has total 4 spontaneous bleeding event in six months than ASBR will be 4/6\*12= 7.99
Mean of APTT among the groups will be compared.
时间窗: 06 months
APTT
次要结局
- Inhibitor to Factor VIII-(06 months)
研究者
Akhil Ranjon Biswas
Professor and Head of the Department of Hematology.
Dhaka Medical College