A Single-arm, Open-label Phase II Study to Determine the Safety and Efficacy of Teriflunomide in Patients With Steroid-resistant/Relapse Thrombocytopenia
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Sustained response
研究概览
简要总结
Single-arm, open-label, single-center study to evaluate the efficacy and safety of teriflunomide for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
详细描述
This is a prospective, single-arm, open-label study of teriflunomide in approximately 40 patients with ITP who have relapsed or have an insufficient response to prior therapies. Teriflunomide is administered as 7 mg orally once daily with dose adjustments for 24 weeks. Efficacy and safety outcomes are assessed on scheduled study visits.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of primary immune thrombocytopenia (ITP) by excluding other supervened causes of thrombocytopenia
- •Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with bleeding manifestations at the enrollment;
- •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;
- •Willing and able to sign written informed consent.
排除标准
- •Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- •Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, malignancy, or patients with confirmed autoimmune disease);
- •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 prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
- •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;
- •Patients who are deemed unsuitable for the study by the investigator.
研究组 & 干预措施
Teriflunomide
Oral Teriflunomide is given at a dose of 7 mg once daily with dose adjustments for 24 weeks. Treatment was discontinued if a dose-limiting toxic effect occurred, rescue medication was used, or concomitant medication for immune thrombocytopenia was changed beyond the 10% level as defined above.
干预措施: Teriflunomide (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)
- Initial response(From the start of study treatment (Day 1) up to week 4 of treatment)
- Time to response(From the start of study treatment (Day 1) to the end of week 24)
- Bleeding events(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)
研究者
Xiao Hui Zhang
Vice president of Peking Univeristy Institute of Hematology
Peking University People's Hospital