Phase II Study of Azacytidine Followed by GM-CSF in Patients With Low- or Intermediate-1- Risk Myelodysplastic Syndrome (MDS)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Overall Response Rate (ORR)
研究概览
简要总结
The goal of this clinical research study is to learn if the combination of azacitidine and GM-CSF can help to control MDS. The safety of these drugs will also be studied.
Azacitidine is designed to block certain proteins that stop the function of tumor-fighting genes. By blocking the "bad" proteins, the tumor-fighting genes may be able to work better.
Granulocyte-macrophage colony-stimulating factor (GM-CSF) is designed to help produce white blood cells. This may help to fight infections.
详细描述
Study Drug Administration:
If you are found to be eligible to take part in this study, on Days 1-4 of every cycle, you will receive azacitidine by vein over 15-30 minutes.
You may receive drugs to help prevent nausea and vomiting before you receive your dose of azacitidine.
On Days 5-7 of every cycle, you will receive GM-CSF by vein over about 15 minutes or by injection.
Each study cycle will be about 4-6 weeks, depending on the study doctor's decision.
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
- •Patients with low- or intermediate-1-risk MDS according to the International Prognostic Scoring System (IPSS) classification
- •Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UT MD Anderson Cancer Center.
- •Age >/= 18 years old.
- •Prior therapy with growth factor support, lenalidomide, or other investigational agents is allowed.
- •Previously untreated patients are eligible for this study.
排除标准
- •Any previous adverse reaction (>/= Common Terminology Criteria for Adverse Events (CTCAE) grade 2) to GM-CSF.
- •Prior treatment with azacytidine or decitabine.
- •Unresolved diarrhea >/= CTCAE grade 2.
研究组 & 干预措施
Azacytidine + GM-CSF
Azacytidine administered intravenously (IV) or subcutaneously (SQ) at starting dose of 40 mg/m^2, daily for 4 days.
GM-CSF administered IV or subcutaneously at 250 mcg/m^2 one day (the next day) after completion of azacytidine treatment, for 3 consecutive days.
Each treatment cycle will last at least 4 weeks
干预措施: Azacytidine (Drug)
Azacytidine + GM-CSF
Azacytidine administered intravenously (IV) or subcutaneously (SQ) at starting dose of 40 mg/m^2, daily for 4 days.
GM-CSF administered IV or subcutaneously at 250 mcg/m^2 one day (the next day) after completion of azacytidine treatment, for 3 consecutive days.
Each treatment cycle will last at least 4 weeks
干预措施: GM-CSF (Drug)
结局指标
主要结局
Overall Response Rate (ORR)
时间窗: Baseline up to 2 treatment cycles (8 weeks)
ORR is percentage total participants with overall response (Complete Response (CR) or Partial Response (PR)) within two treatment cycles. Response based on modified International Working Group (IWG) criteria: Complete response - Bone marrow: 5% myeloblasts with normal maturation of all cell lines, Persistent dysplasia noted, Peripheral blood Hgb 11 g/dL, Platelets 100x109/L, Neutrophils 1.0x109/L, Blasts 0%. Partial response: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by 50% over pretreatment but still \> 5% , Cellularity and morphology not relevant; Stable disease - Failure to achieve at least PR, but no evidence of progression for \> 8 weeks; No Response or Failure - Death during treatment or disease progression characterized by worsening of cytopenias, increase in percentage of bone marrow blasts, or progression to a more advanced MDS French-American-British (FAB) classification subtype than pretreatme
次要结局
未报告次要终点