A Prospective, One-arm and Open Clinical Study to Assess Efficacy and Safety of Avatrombopag in the Treatment of Pediatric Primary Immune Thrombocytopenia
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Overall efficacy response after AVA treatment within 12 weeks
研究概览
简要总结
To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.
详细描述
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.).
Eltrombopag is currently the only TPO receptor agonist with indications for pediatric immune thrombocytopenia. However, at present, the treatment response of pediatric ITP is not good, and a considerable number of patients need to switched to other TPO receptor agonist, such as avatrombopag, because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.
Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of avatrombopag in the treatment of pediatric immune thrombocytopenia in patients who who have been treated with eltrombopag before and switched to avatrobopag because of different reasons.
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
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 6-18 years old (including both ends), male and female;
- •Diagnosed with primary immune thrombocytopenia (ITP);
- •Patients who had previously received eltrombopag treatment and then converted to avatrombopag treatment because of ineffectiveness (platelet count < 30×10^9/L after eltrombopag treatment, or platelet count increased less than 2 times of the basic value, or bleeding) or large platelet fluctuation or due to patient preference, economic reasons and other reasons;
- •Cardiac function of the New York Society of Cardiac Function ≤ 2;
- •Understand the study procedure and voluntarily sign the informed consent.
排除标准
- •Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
- •Subjects with primary disease of important organs (liver, kidney, heart, etc.), or with immune system diseases;
- •Subjects who are known to be allergic to avatrombopag or any of its excipients;
- •Subjects who had used rituximab within the last 3 months;
- •Subjects who underwent splenectomy within the last 3 months；
- •Subjects with a history of abnormal platelet aggregation that may affect the reliability of platelet count measurements；
- •Any medical history or condition that the investigator deems unsuitable for participation in the study.
研究组 & 干预措施
Treatment group: Avatrombopag
Sixty subjects will be enrolled with the indicated treatment dose of avatrombopag
干预措施: Avatrombopag (Drug)
结局指标
主要结局
Overall efficacy response after AVA treatment within 12 weeks
时间窗: 12 weeks
Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding at the meanwhile within 12 weeks after initial administration in absence of rescue therapy.
次要结局
- Treatment response-1(12 weeks)
- Persistent response(12 weeks)
- Reduction of concomitant drug(12 weeks)
- Health-related quality of life survey of subjects(HRQoL)-1(12 weeks)
- Treatment response-2(12 weeks)
- Time to Response(12 weeks)
- Emergency treatment(12 weeks)
- Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.(12 weeks)
- Number of participants with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP.(12 weeks)
- Health-related quality of life survey of subjects(HRQoL)-2(12 weeks)
- Health-related quality of life survey of subjects(HRQoL)-3(12 weeks)
- Health-related quality of life survey of subjects(HRQoL)-4(12 weeks)