Thalidomide for the Treatment of Cytopenias of Patients With Low Risk Myelodysplastic Syndromes
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 112
- 试验地点
- 20
- 主要终点
- Efficacy evaluated at week 12 according to the IWG criterias
研究概览
简要总结
The GFM previously conducted a dose-escalating phase II trial of thalidomide in MDS with a minimum dose of 200mg/d and a maximum dose 800mg/d. Responses were evaluated according to IWG criteria at week 16 and thalidomide continued up to week 56 in responders. 82% patients received at least 8 weeks of treatment and were evaluable. 59% had hematological improvement, mainly on the erythroid lineage (Increase of Hemoglobin). Most responses were observed at low doses and between 4 and 8 weeks.
The objectives of this trial (Thal-SMD-20) are to evaluate the efficacy and tolerance of lower doses thalidomide in low risk MDS patients with transfusion-dependant anemia.
详细描述
Thalidomide:
First part of the trial: 82 patients at 200mg/day given at bedtime x 12 weeks, decreased to 100mg/day if grade 1 or 2 side. Stopped temporally for 1 week if grade 3 or 4 side effects. Then reintroduced at the same dose. If side effects again, definitively stopped.
Responses evaluated at 12 weeks according to IWG criteria for the erythroid lineage
At week 12:
- If no Hematological improvement (HI): increased to 300mg/day for 8 weeks and then eventually to 400mg/day for 8 weeks more, if no HI.
- If Hematological improvement (HI): continued at the same dose.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients ≥18 years, with IPSS Low or Int-1 MDS
- •Transfusion dependant anemia above 2 packed red blood cells (PRBC)/month
- •ECOG index = 0, 1, 2
- •No peripheral neurological disease
排除标准
- •MDS patients with IPSS Int-2 or High
- •Patients with less than 2 packed red blood cells (PRBC)/month
- •Patients with previous history of venous thrombosis
- •Patient treated with EPO +/- G-CSF in the 2 months before inclusion in the protocol
- •Patient having received intensive chemotherapy in the 3 months before inclusion in the protocol
- •Patient having received Thalidomide in a previous protocol
- •Patient presenting an iron, B12 vitamin or folic acid uncorrected deficiency
- •Patient with peripheral neurological disease
- •Patient not being able to subject itself to a regular clinical and biological follow-up
- •Pregnant patient or patient in a period of lactation
- •Patient refusing to take a contraceptive treatment through out all the study
- •Patient receiving drugs able to interfere with the mechanism of action of Thalidomide
- •Patient refusing to sign the informed consent.
结局指标
主要结局
Efficacy evaluated at week 12 according to the IWG criterias
次要结局
- Safety