A Multicenter, Open-label, Randomized, Controlled, Phase 2 Trial Comparing the Efficacy and Safety of Teriflunomide Plus Danazol in Patients With Steroid-resistant/Relapse ITP
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 124
- 试验地点
- 1
- 主要终点
- Sustained response
研究概览
简要总结
To compare the efficacy and safety of teriflunomide plus danazol versus danazol in patients with steroid-resistant/relapse ITP
详细描述
This is a prospective, multicenter, randomized, controlled trial of 124 adult patients with steroid-resistant/relapse ITP in China. Patients were randomized to receive either experimental teriflunomide plus danazol or active comparator danazol monotherapy. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
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
- •Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
- •Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid tapering or after its discontinuation;
- •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
- •Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, or patients with confirmed autoimmune disease);
- •Active or a history of malignancy;
- •Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than two times the upper limit of normal (ULN);
- •Pregnancy or lactation;
- •Current or recent (< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
- •Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
- •Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB;
- •Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled;
- •Patients who are deemed unsuitable for the study by the investigator.
研究组 & 干预措施
Teriflunomide plus danazol
Oral teriflunomide was given at a starting dose of 7 mg once daily and danazol was given at a dose of 200mg twice daily for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
干预措施: Teriflunomide (Drug)
Teriflunomide plus danazol
Oral teriflunomide was given at a starting dose of 7 mg once daily and danazol was given at a dose of 200mg twice daily for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
干预措施: Danazol (Drug)
Danazol
Danazol was given at a dose of 200 mg twice a day for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
干预措施: Danazol (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
- Overall response(From the start of study treatment (Day 1) to the end of week 24)
- Adverse events(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)
- 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)
- Initial response(From the start of study treatment (Day 1) up to week 4 of treatment)
- Bleeding events(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