A Pilot Study of AMD3100 as a Sensitizing Agent in Myeloablative Allogeneic Blood and Marrow Transplantation for Chemotherapy Resistant Pediatric Acute Leukemia
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- AMD3100 safety
研究概览
简要总结
This study is for patients 2-21 years old who have acute leukemia that has not responded well to chemotherapy and will have a bone marrow transplant. This is a pilot (phase 1) study of AMD3100(also called Plerixafor, Mozobil). AMD3100 is given in combination with a standard pre-transplant conditioning regimen (total body irradiation, etoposide and cyclophosphamide). The conditioning regimen is the treatment that is given just before the transplant. This treatment kills leukemia cells as well as healthy bone marrow and immune cells. Researchers want to learn more about how AMD3100 affects acute leukemia cells. Blood and bone marrow samples from study participants will be collected to find out if AMD3100 is making patients' cells more sensitive to the conditioning regimen and to find out how it does this.
The first six patients receive three daily doses (240 mcg/kg via IV). If it appears that three doses do not significantly increase the side effects of transplant conditioning, the investigators will give a second group of six patients five daily doses.
详细描述
The first six patients will receive three daily doses of AMD3100 (240 mcg/kg via IV). If it appears that three doses do not significantly increase the side effects of transplant conditioning, the investigators will give a second group of six patients five daily doses.
AMD3100 is given in combination with a standard pre-transplant conditioning regimen (total body irradiation, etoposide and cyclophosphamide.) AMD3100 causes healthy bone marrow cells to be released from the bone marrow into the blood so that they can be collected in patients who will have peripheral (blood stream) blood stem cell transplants. AMD3100 also pushes out leukemia cells from the bone marrow. Research in animals and in test tubes shows that the bone marrow partially protects leukemia cells from chemotherapy and radiation. AMD3100 could make leukemia treatments better by pushing out the leukemia cells from the bone marrow and making them more sensitive to treatment. Clinical trials combining AMD3100 with normal doses of chemotherapy are being done for relapsed acute leukemia. Researchers hope AMD3100 can be given with conditioning regimen safely without causing more side effects. Up to 12 participants will be enrolled and estimated accrual duration is 2 years.
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
- 2 Years 至 22 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have chemotherapy-resistant acute leukemia (primary refractory or relapsed and refractory AML, ALL, undifferentiated, bi-lineage or mixed lineage leukemia)
- •Participant must have a well HLA matched related, mismatched related or unrelated marrow donor with whom the patient is allele matched at at least 7 of 8 HLA loci or a single unrelated cord blood unit matched at at least 4 of 6 HLA loci with minimal dose of 4x10(7)NC/Kg
排除标准
- •Prior allogeneic or autologous hematopoietic stem cell transplantation
- •Prior exposure to AMD3100
- •Active central nervous system leukemia
- •Uncontrolled viral, bacterial, fungal, protozoal infection
- •HIV infection
- •Does not meet standard organ function for transplant
研究组 & 干预措施
bone marrow transplant
干预措施: AMD3100 (Drug)
结局指标
主要结局
AMD3100 safety
时间窗: day 42 after bone marrow transplant
Incidence of grade 3 and 4 regimen-related toxicity assessed per Bearman scale at day 42. Patients in dose level 1 get 3 days of AMD3100. If 2 cases of grade 4 toxicity or 3 cases of grade 3-4 toxicity occur, dose level 1 and study will be closed. Otherwise, after 6 patients have been assessed at day 42, the dose will be escalated to 5 days of AMD3100 (dose level 2). If 2 cases of grade 4 toxicity or 3 cases of grade 3-4 toxicity occur, the study will be closed. Otherwise, after 6 patients have been enrolled at this level the study will be closed to enrollment.
次要结局
- AMD3100 correlative biology analyses(2 years)
研究者
Kuang-Yueh Chiang
Associate Professor
Emory University