Individualizing Hemophilia Prophylaxis Using Thromboelastography
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by estimated proportion of patients whose dose is modified.
研究概览
简要总结
Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day. One of the issues with weight-based dosing is the possible over-treatment. This is likely due to the fact that laboratory tests are not sensitive enough at the low levels to support decision-making. The Thromboelastograph (TEG®) and Thromboelastometry (ROTEM®) are coagulation devices, which assess the dynamics of clot formation over time and have several characteristics which suggest they may provide important information for individualized prophylaxis treatment for our patients.
详细描述
This is a pilot study in children and adults with severe hemophilia A that utilizes the TEG/ROTEM to personalize their prophylaxis treatment.
Primary objectives:
• Assess the feasibility of TEG/ROTEM-guided modification of the current prophylaxis regimen for individuals with severe hemophilia A. Specifically, to:
- Test and refine the operational protocol for using TEG/ROTEM to guide factor dosing
- Estimate the proportion of hemophilia A patients whose dose will be modified based on TEG/ROTEM results
- Monitor short term safety of patients whose dose has been modified.
Secondary objectives
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males, 5-70 years, inclusive
- •Plasma FVIII activity <1% documented (Laboratory result or MD documentation of Severe Hemophilia A diagnosis)
- •Currently prescribed prophylaxis treatment regimen infusing ≥3 times a week
- •Willing to alter their prophylaxis treatment regimen per study protocol
排除标准
- •Bleeding disorder(s) other than Hemophilia A
- •Current inhibitor (>0.6BU)
- •Thrombocytopenia (platelet count <100,000K/µL since it can alter TEG®/ROTEM® results)
- •Creatinine >2x the upper limit of normal (indicating potential platelet dysfunction)
- •Prothrombin time >3 seconds above the upper limit of normal (indicating potential liver dysfunction)
- •Any concurrent clinically significant major disease, frequent bleeding pattern or history of non-compliance that, in the opinion of the investigator, would make the subject unsuitable for enrollment
- •Participation within the past 30 days in any other clinical study involving investigational drugs
- •Planned major surgery within 30 days prior to screening or during the study period
- •Current use of any medication known to have effects on the coagulation system
结局指标
主要结局
Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by estimated proportion of patients whose dose is modified.
时间窗: Ongoing while patients are on study (~ 6 months)
A total of 60 patients will be enrolled on this study. It is expected that less than 25% of these patients will be eligible for dose/schedule modification based on TEG/ROTEM.
Monitoring short-term safety of patients whose dose has been modified, assessed by monitoring number, type, and severity of bleeds.
时间窗: Ongoing while patients are on study (~ 6 months)
The primary endpoint for subject safety will be the occurrence of two serious spontaneous bleeding episodes within 28 days of each other.
Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by testing and refining operational protocol for using TEG/ROTEM to guide factor dosing.
时间窗: Ongoing while patients are on study (~ 6 months)
次要结局
- Thrombin generation assay will be performed to provide additional evidence supporting the TEG/ROTEM-guided dosing.(6 months)
- Assessment of direct costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation.(1 year)
- Assessment of indirect costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation.(6 months)
- Improvement of subject/family burden as assessed by the Health-Related Quality of Life.(6 months)
研究者
Guy Young
Guy Young, MD
Children's Hospital Los Angeles