NCT06155955招募中2 期
Comparison of Outcomes Between Low Dose Emicizumab and Extended Half-life Factor VIII With Pharmacokinetic-guided Prophylaxis in Clinically Severe Hemophilia A
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- treated spontaneous bleeds, treated joint bleeds
研究概览
简要总结
- To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration
- To study pharmacokinetic, side effect of low dose Emicizumab
详细描述
- To compare outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration using ABR, AJBR and HJHS, HaemoQoL
- To study pharmacokinetic, side effect and effectiveness of low dose Emicizumab
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 30 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Severe or moderate Hemophilia A with baseline FVIII:C of <3 IU/dl or severe bleeding phenotype
- •Receiving low dose FVIII prophylaxis for at least 6 months
排除标准
- •Detectable FVIII inhibitor at screening
- •Detectable FVIII inhibitor at screening Having other underlying diseases: Juvenile rheumatoid arthritis, metabolic bone diseases, or other conditions mimicking or causing joint diseases
研究组 & 干预措施
low dose emicizumab
Experimental
2 mg per kilograms per dose in first month, loading every 2 weeks then every 4 weeks
干预措施: "Emicizumab", "HEMLIBRA®" (Drug)
结局指标
主要结局
treated spontaneous bleeds, treated joint bleeds
时间窗: 6 months after start low dose emicizumab as secondary prophylaxis treatment
Using "Annualized joint bleeding rate" (AJBR)
spontaneous and traumatic bleeding rate
时间窗: 6 months after start low dose emicizumab as secondary prophylaxis treatment
Using "Annualized bleeding rate" (ABR)
The function of the knee, elbow, and ankle joints
时间窗: 6 months after start low dose emicizumab as secondary prophylaxis treatment
Using "Hemophilia Joint Health Score" (HJHS)
次要结局
未报告次要终点
研究者
Nuchanun Kessakorn, MD
MD
Chulalongkorn University
研究点 (1)
Loading locations...
相似试验
招募中
不适用
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia AHemophilia ANCT04131036Washington Institute for Coagulation40
尚未招募
不适用
Prophylactic Therapy In Hemophilia ACTRI/2020/07/026880Maulana Azad Medical College
已完成
不适用
Comparative Trial of Standard Versus Low-dose Peg-Interferon Plus Ribavirin in the Treatment of Chronic Hepatitis CHepatitis C, ChronicNCT00216775Kyoto University50
已完成
3 期
Clinical Trial of Efficacy and Safety of the Combination of Reduced Duration Prophylaxis Followed by Immuno-guided Prophylaxis in Lung Transplant Recipients.Transplantation InfectionCytomegalovirus InfectionsNCT03699254Maimónides Biomedical Research Institute of Córdoba150
已完成
不适用
Comparison of the influence of low-dose supplementation with potassium citrate or potassium chloride on blood pressure, plasma and erythrocyte electrolyte concentrations and bone health biomarkersHypertensionISRCTN52748737King's College London (UK)120