A Multicenter Randomized Trial of Second Line Treatment for Corticosteroid-Resistant or Relapsed Immune Thrombocytopenia: Combined Terbutaline and Danazol Versus Danazol Monotherapy
试验速览
- 阶段
- 2 期
- 入组人数
- 228
- 主要终点
- Overall response
研究概览
简要总结
A randomized, open-label, multicenter study to compare the efficacy and safety of terbutaline plus danazol compared to danazol monotherapy for the second-line treatment of adults with corticosteroid-resistant or relapsed primary immune thrombocytopenia (ITP).
详细描述
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 228 adults with ITP in China. Patients were randomized to terbutaline plus danazol compared to danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients >18 years old with corticosteroid-resistant or relapsed ITP who had either a platelet count of <30×10^9/L or a platelet count of <50×10^9/L and clinically significant bleeding.
- •Not achieving a sustained response to therapy with full-dose corticosteroids for a duration of at least 4 weeks or relapsed during the process of corticosteroid tapering or discontinuation;
- •Platelet counts <30×10^9/L or platelet counts < 50×10^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- •Willing and able to sign written informed consent.
排除标准
- •Pregnant or lactating women;
- •Secondary ITP (have a known diagnosis of connective tissue diseases, malignancy, active infection, HIV infections or hepatitis B virus or hepatitis C virus infections);
- •Received drugs affecting the platelet counts within 6 months before the screening visit (e.g., chemotherapy, anticoagulants, etc);
- •Severe medical condition (lung, heart, hepatic or renal disorder);
- •Patients who are deemed unsuitable for the study by the investigator.
- •Patients who had hypertension, diabetes mellitus, hyperthyroidism or coronary heart disease.
研究组 & 干预措施
Terbutaline plus danazol
Terbutaline: A dose of 2.5 mg three times daily for 16 weeks Danazol: A dose of 200 mg twice daily for 16 weeks
干预措施: Terbutaline (Drug)
Terbutaline plus danazol
Terbutaline: A dose of 2.5 mg three times daily for 16 weeks Danazol: A dose of 200 mg twice daily for 16 weeks
干预措施: Danazol (Drug)
Danazol monotherapy
Danazol: A dose of 200 mg twice daily for 16 weeks
干预措施: Danazol (Drug)
结局指标
主要结局
Overall response
时间窗: From date of randomization until 1 years or the end of follow-up
Achieving a platelet count ≥ 30 × 10\^9/L confirmed on at least two separate occasions (at least 7 days apart), at least a doubling of the baseline platelet count without any other ITP-specific treatment and the absence of bleeding.
次要结局
- Sustained response(From date of randomization until 1 years or the end of follow-up)
- Time to response(From date of randomization until 1 years or the end of follow-up)
- Duration of response(From date of randomization until 1 years or the end of follow-up)
- Rescue therapy(From date of randomization until 1 years or the end of follow-up)
- Associated factors of treatment failure, OR, SR and remission(From date of randomization until 1 years or the end of follow-up)
- Relapse-free survival(From date of randomization until 1 years or the end of follow-up)
- Complete response(From date of randomization until 1 years or the end of follow-up)
- Number of patients with side effects(From date of randomization until 1 years or the end of follow-up)
- Relapse(From date of randomization until 1 years or the end of follow-up)
- Remission(at 12-month follow-up)
- Number of patients with bleeding(From date of randomization until 1 years or the end of follow-up)
研究者
Xiao Hui Zhang
Vice President of Peking University Institute of Hematology
Peking University People's Hospital