Phase II Study of Thalidomide in the Treatment of Myelodysplastic Syndromes in Adults: A Clinical and Biologic Study
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 29
- 试验地点
- 1
- 主要终点
- Confirmed response defined as complete hematologic response (CHR) or partial response (PR) or hematological improvement (HI) on 2 consecutive evaluations in terms of proportion of successes measured using criteria reported by Cheson et al
研究概览
简要总结
Phase II trial to study the effectiveness of thalidomide in treating patients who have myelodysplastic syndrome. Thalidomide may improve the immune system's ability to fight myelodysplastic syndrome
详细描述
OBJECTIVES:
I. Determine whether thalidomide improves cytopenias in patients with myelodysplastic syndromes.
II. Determine the toxicity of this regimen in these patients. III. Determine whether this regimen down regulates the peripheral blood levels of tumor necrosis factor alpha, interferon gamma, and interleukin-12 and whether these changes correlate with clinical response in these patients.
IV. Determine whether this regimen alters the peripheral blood T-cell subset distribution and whether these changes correlate with clinical response in these patients.
V. Determine the effect of this regimen on bone marrow microvessel density and whether these effects correlate with clinical response in these patients.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pre-transfusion hemoglobin =< 10 g/dL
- •Pre-transfusion platelet count =< 50,000/μL
- •Absolute neutrophil count < 1000/μL
- •Total bilirubin ≤ 1.5 x UNL
- •Alkaline phosphatase ≤ 3 x UNL
- •AST ≤ 3 x UNL
- •Creatinine ≤ 1.5 x UNL
- •A diagnosis of MDS as demonstrated in the bone marrow; any subtypes are eligible including:
- •Refractory anemia (cytopenia)
- •Refractory anemia with ringed sideroblasts
- •Chronic myelomonocytic leukemia
- •Refractory anemia with excess blasts
- •Refractory anemia with excess blasts in transformation
- •Unclassified MDS
- •Patients with refractory anemia with excess blasts in transformation who are not candidates for (or who decline) induction chemotherapy are eligible; those patients who were candidates for (and accepted) induction chemotherapy should have failed at least 1 chemotherapy regimen prior to entry
- •Patients who are candidates for marrow transplantation should have this option discussed prior to study entry
排除标准
- •Any of the following as this regimen may be harmful to a developing fetus or nursing child:
- •Pregnant women
- •Nursing women
- •Women of childbearing potential or their sexual partners who are unwilling to employ 2 adequate methods of contraception (condoms, diaphragm, birth control pills, injections, intrauterine device [IUD], surgical sterilization, subcutaneous implants, or abstinence, etc.)
- •Peripheral neuropathy (by history or clinical exam)
- •Concomitant therapy ≤ 30 days for myelodysplastic syndrome with any specific agent including chemotherapy, corticosteroids and/or growth factors (i.e. erythropoietin, G-CSF, GM-CSF, thrombopoietic agent); patients on chronic low-dose corticosteriods (< 20 mg/d) for reasons other than MDS are allowed
- •Uncontrolled infections
研究组 & 干预措施
Treatment (thalidomide)
Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.
干预措施: thalidomide (Drug)
Treatment (thalidomide)
Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.
干预措施: laboratory biomarker analysis (Other)
结局指标
主要结局
Confirmed response defined as complete hematologic response (CHR) or partial response (PR) or hematological improvement (HI) on 2 consecutive evaluations in terms of proportion of successes measured using criteria reported by Cheson et al
时间窗: Up to 3 months
Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
Incidence and severity of toxicities, graded according to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0
时间窗: Up to 5 years
次要结局
- Survival time(Time from registration to death due to any cause, assessed up to 5 years)
- Time to disease progression(Time from registration to documentation of disease progression, assessed up to 5 years)
- Duration of response measured using criteria reported by Cheson et al(Date at which the patient's objective status is first noted to be either a CHR or PR to the date progression is documented, assessed up to 5 years)
- Time to treatment failure(Time from the date of registration to the date at which the patient is removed from treatment due to progression, toxicity, or refusal, assessed up to 5 years)