A Phase II Study of the Use of 5-Azacytidine as Pre-Transplant Cytoreduction Prior to Allogeneic Stem Cell Transplantation for High Risk Myelodysplastic Syndromes
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
研究概览
简要总结
The purpose of this study is to examine the feasibility and efficacy of using the demethylating agent 5-Azacytidine prior to allogeneic stem cell transplantation in patients with high risk myelodysplastic syndrome (MDS).
详细描述
The study drug, 5-azacytidine, is given daily intravenously for 7 days. After every 2 cycles study participants will have a bone marrow test to evaluate the effect of the 5-azacytidine on the Myelodysplastic Syndrome (MDS). Participants continue to get cycles of 5-Azacytidine until 2 bone marrow tests show the MDS has stopped responding to the treatment. At that time they will undergo a transplant if a donor is available.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients fulfilling the following criteria will be eligible for study entry:
- •Diagnosis of MDS according to WHO criteria
- •Intermediate-2 or high risk by IPSS score
- •Clinically able to receive 5-Azacytidine
- •Serum bilirubin levels </=1.5 times the upper limit of the normal range for the laboratory (ULN). Higher levels are acceptable if these can be attributed to active hemolysis or ineffective erythropoiesis
- •Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic transaminase (SGPT) levels </=2 x ULN
- •Serum creatinine levels </=1.5 x ULN
- •Negative serum pregnancy test prior to 5-Azacytidine treatment for women of childbearing potential
- •Women and men of childbearing potential agree to use contraception while receiving treatment with 5-Azacytidine
- •Potentially eligible for allogeneic transplantation
- •No prior allogeneic transplant
- •Age 18 to 70, inclusive.
排除标准
- •Known or suspected hypersensitivity to 5-azacytidine or mannitol
- •Patients previously treated with 5-azacytidine or deoxyazacytidine
- •Pregnant or breast feeding
- •Patients with advanced malignant hepatic tumors
研究组 & 干预措施
Arm A: 5-azacytidine
5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes.
干预措施: 5-azacytidine (Drug)
结局指标
主要结局
One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
时间窗: 1 year
Percentage of patients alive one year after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated one year survival rate from this curve is 50%, while the estimated two year survival rate is 50%.
Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
时间窗: 2 years
Percentage of patients alive two years after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated two year survival rate is 50%, the same as one year survival rate.
One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
时间窗: 1 year
Percentage of participants that received allogeneic transplant and had event free survival, as estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated one-year event-free survival rate is the same as overall survival, 50%.
Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
时间窗: 2 years
Percentage of participants that received allogeneic transplant and had event free survival. The percentage of patients was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated two-year event-free survival rate is the same as overall survival, 50%.
次要结局
- One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts(1 year)
- Two-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts.(2 years)
- One-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts(1 year)
- Two-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts(2 years)