Individualizing Bypassing Agent Therapy Utilizing TEG in Hemophilia Patients With Inhibitors
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 主要终点
- Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent
研究概览
简要总结
This pilot study will investigate the use of thromboelastograph (TEG) to determine the primary bypassing agent for the management of bleeding in children and adults severe hemophilia A patients with inhibitors.
The study will evaluate the TEG profile for each of the available bypassing agents (FEIBA and rFVIIa) in each participating patient, which will then determine which agent provides the most robust clot formation as measured by the TEG. This study will consist of screening visit and 2-4 pharmacokinetic studies to determine the "best" bypassing agent based on the TEG results. Patients will then be assigned that bypassing agent and dose for the treatment of their bleeding episodes (Prophylaxis or On-demand). Each patient will be then followed for a period of 6 months to monitor short-term safety of those patients whose bypassing agent was modified.
详细描述
Currently, there are no laboratory assays that can assess the pharmaceutical activity of the available agents known collectively as bypassing agents, which negatively impacts the treating physician's ability to manage these patients, leaving decisions on the dosing regimen to trial and error. Both agents, FEIBA and recombinant activated factor VII (rFVIIa), are approved for the treatment of bleeding in inhibitor patients with recommended dosing regimens that are based on clinical trials. However, a significant amount of "experimenting" with other dosing regimens is used by physicians and patients.
The goal of this project is to demonstrate that TEG is an excellent biomarker for predicting, managing and individualizing the treatment with FEIBA and rFVIIa of this rare and difficult to treat patient population.
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
- 4 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males, 4-60 years old, inclusive on Prophylaxis or On-Demand treatment
- •Diagnosis of Hemophilia with active titer inhibitors (> 0.6 BU)
- •Willing to alter their treatment regimen per study protocol
排除标准
- •Bleeding disorder(s) other than hemophilia A with inhibitors
- •Thrombocytopenia (platelet count <100,000K/µL)
- •Any concurrent clinically significant major disease that, in the opinion of the investigator, would make the subject unsuitable for enrollment
- •Participation within the past 30 days in a clinical study involving investigational drugs
- •Planned major surgery within 30 days prior to screening or during the study period
研究组 & 干预措施
Severe Hemophilia A subjects w/ inhibitors
Males age 4-70 years diagnosed with severe hemophilia A with inhibitors who are currently treated with prophylaxis or on-demand treatment will be enrolled.
干预措施: Thromboelastography-Guided Treatment Regimen (Other)
结局指标
主要结局
Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent
时间窗: 6 months
This study will assess the feasibility of TEG-guided individualization of bypassing agent treatment of bleeding for severe hemophilia A patients with inhibitors.
次要结局
- Thrombin generation assay measuring the formation of thrombin during clot formation(6 months)
- Number of participants with treatment-related adverse events(1 year)