Autologous Purged Hematopoietic Stem Cell Transplantation for Chronic Myelogenous Leukemia (CML)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Time to absolute neutrophil count (ANC) recovery to 500
研究概览
简要总结
Primary Objective:
- To study ex-vivo purging of autologous hematopoietic stem cells that will be used to support high-dose chemotherapy in patients with chronic myelogenous leukemia (CML). Major endpoints are neutrophil engraftment and survival.
Secondary Objectives:
- To evaluate the toxicity of ex-vivo purged autologous cells when used to support high-dose chemotherapy.
- To evaluate the rate and duration of cytogenetic remissions achieved with this strategy.
- To determine the time to platelet recovery to 20,000/mm3.
- To determine the one-year survival rate.
详细描述
Before treatment starts, you will have a complete physical exam. You will have about 4 tablespoons of blood drawn for tests to check on the status of the disease, to check organ functions, and to check for infectious diseases (hepatitis, HIV, etc.). You will have a sample of bone marrow collected. To collect a bone marrow sample, an area of the hip or chest bone is numbed with anaesthetic and a small amount of bone marrow is withdrawn through a large needle. You will also have heart (cardiac ejection fraction) and lung function tests. All of these tests are being done to make sure you are eligible for this treatment.
In order to access the blood in the circulation, a silicone catheter (small plastic tube) will be placed in the collar bone area. You will be under local anesthesia during this procedure. If you are found to be eligible to take part in this study, this catheter will be used for the collection of stem cells, infusion of chemotherapy, fluids, electrolytes, other medications, and also for blood sampling for lab tests. It will remain in your body for the length of the treatment (between 2-5 months).
Normally, there are very few stem cells in the blood. The majority of them are in the bone marrow. To help move or "mobilize" the cells needed from your bone marrow to your blood, you will be given chemotherapy followed by injections under the skin once a day of a drug called G-CSF and another drug called GM-CSF. These injections will be given over 7-21 days. The injections may be given by a nurse in the hospital, in the outpatient setting, or you may learn how to give them to yourself.
The chemotherapy you will receive to help mobilize the stem cells is cyclophosphamide. It will be given by vein over a few hours in one day. This is the first step toward collecting the stem cells necessary for transplantation later on. It is separate from the high-dose chemotherapy that you get after the transplant.
Blood samples (about 4 teaspoons each) will be drawn once a day after the cyclophosphamide to check for the numbers of stem cells in your blood and to monitor for possible side effects. If the number of stem cells in the blood is high enough, you will go to the UTMDACC Apheresis Unit and have your peripheral blood progenitor cells (PBPC, or stem cells) collected.
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
- 21 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with Philadelphia (Ph) chromosome positive CML < age 65 and older than 21 years.
- •Ph positive CML that is either in:
- •late 1st chronic phase (> 2 years from diagnosis)
- •early chronic phase who did not achieve complete cytogenetic remission after one year on imatinib
- •beyond first chronic phase
- •accelerated phase
- •blastic phase that has responded to therapy (characterized by the presence of < 10% bone marrow and/or circulating blasts at consent signing)
- •chronic phase, developing imatinib resistance (loss of molecular remission defined as at least a 1 log increase in the BCR-ABL/ABL ratio, in 2 time points at least 1 month apart, or loss of cytogenetic remission)
- •Patients must have a Zubrod PS <
- •Creatinine < 1.8 mg/dl
- •Serum bilirubin </= 1.5 mg/dl
- •Serum glutamate pyruvate transaminase (SGPT) < 3 x normal values
- •Patients with an HLA identical sibling are eligible if they refuse allogeneic transplantation, or if they are ineligible for allogeneic transplantation due to age.
- •DLCO >/= 50% of predicted
- •Cardiac Ejection fraction >/= 40%
排除标准
- •Uncontrolled life-threatening infections or comorbid condition that could impair tolerance to the regimen.
- •HIV positivity.
- •Pregnant or lactating women.
- •CML in blastic phase that has not responded to therapy given prior to enrollment in this study (characterized by the presence of more than 9% bone marrow and/or peripheral blood blasts at the time of consent signing)
- •Hepatitis B or C virus infection. Hepatitis B infection defined by positive DNA test, positive E and / or surface antigen.
- •CML in first molecular remission.
研究组 & 干预措施
Busulfan + Cyclophosphamide + G-CSF + GM-CSF
干预措施: Busulfan (Drug)
Busulfan + Cyclophosphamide + G-CSF + GM-CSF
干预措施: Cyclophosphamide (Drug)
Busulfan + Cyclophosphamide + G-CSF + GM-CSF
干预措施: G-CSF (Drug)
Busulfan + Cyclophosphamide + G-CSF + GM-CSF
干预措施: GM-CSF (Drug)
结局指标
主要结局
Time to absolute neutrophil count (ANC) recovery to 500
时间窗: 30 Days
Survival Time
时间窗: 30 Days (Success Rate + ANC Recovery to 500)
次要结局
未报告次要终点