A Study of All-trans Retinoic Acid in the Treatment of Immune Thrombocytopenia
试验速览
- 阶段
- 2 期
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- Sustained response
研究概览
简要总结
An open-label, multicenter study to compare the efficacy and safety of ATRA for the treatment of adults with primary immune thrombocytopenia (ITP)
详细描述
The investigators are undertaking an open-label, single-arm, multicenter trial of adults patients with ITP in China. Patients with treatmant naive ITP were given ATRA treatment. 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
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed newly-diagnosed, treatment-naive ITP;
- •Platelet counts <30×109/L ;
- •Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- •Willing and able to sign written informed consent.
排除标准
- •Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 3 months before the screening visit;
- •Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) within 3 months before the screening visit;
- •Received high-dose steroids or IVIG in the 3 weeks prior to the start of the study.
- •Current HIV infection or hepatitis B virus or hepatitis C virus infections;
- •Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
- •Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period;
- •Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
- •Patients who are deemed unsuitable for the study by the investigator.
研究组 & 干预措施
ATRA treatment group
ATRA is given at a daily dose of 10 mg twice daily orally for 12 weeks
干预措施: all trans retinoic acid (Drug)
结局指标
主要结局
Sustained response
时间窗: 6 months
The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up
次要结局
- Number of patients with bleeding(6 months)
- Number of patients with adverse events(6 months)
- Time to response(6 months)
- Duration of response (DOR)(6 months)
- loss of response(6 months)
- complete response (CR)(day 14)
- Response (R)(day 14)
研究者
Xiao Hui Zhang
Vice president of Peking Univeristy Institute of Hematology
Peking University People's Hospital