Capital Characteristic Application: Pharmacokinetic（PK） Research on Chinese Children of Hemophilia
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 155
- 试验地点
- 1
- 主要终点
- Ultrasound evaluation
研究概览
简要总结
The study start on June 30, 2018. The Severe(F Ⅷ<1%) hemophilia A children without F Ⅷ inhibitor combining were recruited to Test the concentration of the drug in the blood to provide better treatment.
详细描述
Through the integration of two technologies, pharmacokinetics and comprehensive evaluation system, we can improve the individualized prevention and treatment of hemophilia in Chinese children and achieve precise customization of treatment plans. Rational use of medical resources, but also fully achieve the prevention and treatment goals.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 14 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •1-14years old,
- •There have been at least one history of bleeding in any joint of the knee, elbow, or ankle.
- •A blood product containing FVIII is applied.
- •There were no inhibitors at the time of enrollment, and there was no inhibitor-positive history and family history.
- •Clinical visits are available on a regular basis and data is available, and preventive treatment is available prior to enrollment.
- •The child was enrolled in the group and the guardian agreed.
排除标准
- •Combining other disease researchers believes that it is not suitable for enrollment.
- •FVIII inhibitor was found.
- •Refuse to participate in research
研究组 & 干预措施
PK research
Interventional studies were performed in the 1-7 year old subgroup, and the children received a comprehensive assessment and PK test every 3 months. After the 72-hour washout period, 50 IU/kg of concentrated FVIII was administered in a single dose, and blood was taken within half an hour before the infusion and 1 h, 9 h, 24 h, and 48 h after the infusion, and the samples were centrifuged. If the assessment considers that the treatment is inadequate, then the valley concentration target is upgraded.
干预措施: concentrated FVIII (Drug)
结局指标
主要结局
Ultrasound evaluation
时间窗: 3 years
the sonographer will use ultrasound to assess whether the patient's joint lesions become more severe than when they were enrolled.
Imaging evaluation
时间窗: 3 years
the imaging specialist will use MRI to assess whether the patient's joint lesions are more severe than when they were enrolled.
次要结局
- Annualized Joint Bleeding Rate(3 years)
- Annualized Bleeding Rate(3 years)
研究者
Runhui WU
Capital Medical Univercity
Beijing Children's Hospital