A Phase III Randomized Trial of G-CSF Stimulated Bone Marrow vs. Conventional Bone Marrow as a Stem Cell Source In Matched Sibling Donor Transplantation
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 21
- 主要终点
- Estimated Two-year Event-free Survival (EFS)
研究概览
简要总结
This randomized phase III trial is studying donor bone marrow transplant with or without G-CSF to compare how well they work in treating young patients with hematologic cancer or other diseases. Giving chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer or abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving methotrexate and tacrolimus or cyclosporine before and after transplant may stop this from happening. It is not yet known whether donor bone marrow transplant is more effective with or without G-CSF in treating hematologic cancer or other diseases.
详细描述
PRIMARY OBJECTIVE:
I. Compare improvement in event-free survival of patients with hematologic cancer or other diseases undergoing filgrastim (G-CSF)-stimulated bone marrow transplantation (BMT) vs conventional BMT.
SECONDARY OBJECTIVES:
I. Compare the incidence and time to engraftment in patients treated with these regimens.
II. Compare rates of acute and chronic graft-vs-host disease (GVHD) in patients treated with these regimens.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of hematologic cancer or other disease, including any of the following:
- •Chronic myelogenous leukemia in first or second chronic phase
- •Acute lymphoblastic leukemia (ALL), meeting any of the following criteria:
- •Relapsed ALL enrolled on a Children's Oncology Group (COG) relapse clinical trial OR received ≥ 1 round of reinduction therapy (4-6 weeks) and 1 round of intensive consolidation chemotherapy (3-6 weeks)
- •ALL in second complete remission (CR)* after a bone marrow, extramedullary, or combined bone marrow and extramedullary relapse
- •Very high-risk ALL in first CR, defined as any of the following:
- •Philadelphia chromosome-positive ALL
- •Hypodiploidy (< 44 chromosomes)
- •Mixed lineage leukemia rearrangement
- •Induction failure
- •Acute myeloid leukemia in first or second CR
- •Induction therapy must be completed
- •Juvenile myelomonocytic leukemia
- •Myelodysplastic syndromes
- •No clinically evident CNS or extramedullary disease
- •No blasts seen on cerebrospinal fluid cytospin
- •Post-relapse reinduction therapy must be completed
- •Not planning to receive reduced-intensity conditioning regimen
- •Not planning to receive a graft that has undergone T-cell depletion
- •No Down syndrome
- •Matched sibling donor must be available and must be enrolled on ASCT0631D companion study
- •Karnofsky performance status (PS) 60-100% (patients > 16 years of age) OR Lansky PS 60-100% (patients ≤ 16 years of age)
- •AST or ALT < 5 times upper limit of normal for age
- •Bilirubin < 2.5 mg/dL (unless due to Gilbert's syndrome)
- •Creatinine clearance or radioisotope glomerular filtration rate ≥ 70 mL/min OR serum creatinine base on age and/or gender as follows:
- •0.4 mg/dL (1 month to < 6 months of age)
- •0.5 mg/dL (6 months to < 1 year of age)
- •0.6 mg/dL (1 to 2 years of age)
- •0.8 mg/dL (2 to < 6 years of age)
- •1.0 mg/dL (6 to < 10 years of age)
- •1.2 mg/dL (10 to < 13 years of age)
- •1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to < 16 years of age)
- •1.7 mg/dL (male) or 1.4 mg/dL (female) (≥ 16 years of age)
- •Shortening fraction ≥ 27% by echocardiogram OR LVEF ≥ 50% by radionuclide angiogram
- •FEV_1, FVC, and DLCO ≥ 60% OR meets the following criteria (for patients unable to cooperate for pulmonary function tests):
- •No evidence of dyspnea at rest
- •No exercise intolerance
- •No requirement for supplemental oxygen therapy
- •Not pregnant or nursing
- •No known HIV
- •No known uncontrolled fungal, bacterial, or viral infections
- •Patients acquiring fungal disease during induction therapy may proceed if they have a significant response to antifungal therapy with no or minimal evidence of disease remaining by CT scan
- •No prior allogeneic or autologous stem cell transplantation
排除标准
- 未提供
研究组 & 干预措施
Arm I
Patients undergo filgrastim (G-CSF)-stimulated allogeneic bone marrow transplantation on day 0.
干预措施: filgrastim (Biological)
Arm I
Patients undergo filgrastim (G-CSF)-stimulated allogeneic bone marrow transplantation on day 0.
干预措施: allogeneic bone marrow transplantation (Procedure)
Arm I
Patients undergo filgrastim (G-CSF)-stimulated allogeneic bone marrow transplantation on day 0.
干预措施: laboratory biomarker analysis (Other)
Arm II
Patients undergo conventional allogeneic bone marrow transplantation on day 0.
干预措施: allogeneic bone marrow transplantation (Procedure)
Arm II
Patients undergo conventional allogeneic bone marrow transplantation on day 0.
干预措施: laboratory biomarker analysis (Other)
结局指标
主要结局
Estimated Two-year Event-free Survival (EFS)
时间窗: at 2 years
EFS is defined as relapse or treatment-related mortality (TRM). relapse is defined by either morphological or cytogenetic evidence of ALL consistent with pre-transplant features.
次要结局
- Estimated Graft Failure Rate(Up to 10 years)
- Estimated Percentage of Chronic Graft-versus-host Disease (cGVHD)(18 months post-transplant)
- Estimated Incidence of Grade III-IV Acute Graft-versus-host Disease (aGVHD)(Up to 3 months)
- Estimated Median Time to Neutrophil Engraftment(Up to 10 years)
- Estimated 100-day Transplant Related Mortality (TRM) Percentage(100 days)
- Estimated Median Length of Initial Hospitalization(Up to 10 years)