The Combination of Teriflunomide and High-dose Dexamethasone vs High-dose Dexamethasone Alone as First-line Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia (ITP): A Prospective, Multicenter, Randomized Trial
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 132
- 试验地点
- 10
- 主要终点
- Sustained response
研究概览
简要总结
A randomized, open-label, multicenter study to compare the efficacy and safety of teriflunomide plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with newly diagnosed primary immune thrombocytopenia (ITP).
详细描述
This is a parallel-group, multicenter, randomized controlled trial of 132 adults with ITP in China. Patients were randomized to teriflunomide plus high-dose dexamethasone and high-dose dexamethasone monotherapy group. Patients who do not respond to dexamethasone may receive another cycle of high-dose dexamethasone therapy within 2 weeks. 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
- None
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
- •Newly diagnosed, treatment naïve ITP patients
- •Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with bleeding manifestations at the enrollment;
- •Willing and able to sign written informed consent.
排除标准
- •Received first-line and second-line ITP-modifying therapy (any previous dose of corticosteroids or other immune-suppressive agents);
- •Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- •Active or a history of malignancy;
- •Positive test result for hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
- •Pregnancy or lactation;
- •Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN);
- •Current or recent (<4 weeks before screening) clinically serious viral, bacterial, fungal, or parasitic infection;
- •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
Teriflunomide plus Dexamethasone
Oral Teriflunomide was given at a dose of 7 mg once daily for 24 weeks and dexamethasone was given at a dose of 40mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Nonresponsive participants with platelets less than 20 x10^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment.
干预措施: Teriflunomide (Drug)
Teriflunomide plus Dexamethasone
Oral Teriflunomide was given at a dose of 7 mg once daily for 24 weeks and dexamethasone was given at a dose of 40mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Nonresponsive participants with platelets less than 20 x10^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment.
干预措施: Dexamethasone (Drug)
Dexamethasone
Dexamethasone was given at a dose of 40mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Participants in this treatment arm who failed to achieve a sustained response and had a platelet count of less than 20 x 10^9/L or with active bleeding were also allowed to repeat the 4-day course of dexamethasone treatment.
干预措施: Dexamethasone (Drug)
结局指标
主要结局
Sustained response
时间窗: From the start of study treatment (Day 1) to the end of week 24
Platelet count over 30,000/μL and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy for at least four of the six visits between weeks 19 and 24.
次要结局
- Bleeding events(From the start of study treatment (Day 1) to the end of week 24)
- Overall response(From the start of study treatment (Day 1) to the end of week 24)
- Initial response(From the start of study treatment (Day 1) up to week 4 of treatment)
- Adverse events(From the start of study treatment (Day 1) to the end of follow-up)
- Time to response(From the start of study treatment (Day 1) to the end of week 24)
- Duration of response(From the start of study treatment (Day 1) to the end of week 24)
- Health-related quality of life (HRQoL)(From the start of study treatment (Day 1) to the end of week 24)
研究者
Xiao Hui Zhang
Vice president of Peking Univeristy Institute of Hematology
Peking University People's Hospital