CHP 834 Unrelated and Partially Matched Related Donor Peripheral Stem Cell Transportation With the CliniMACs Device for T and B Cell Depletion
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 93
- 试验地点
- 1
- 主要终点
- Number of Participants With Successful Engraftment
研究概览
简要总结
This is a pilot study with 2 strata to evaluate engraftment and graft vs. host disease (GVHD) in patients receiving unrelated or partially matched related donor peripheral stem cells using the CliniMACS system to positively deplete T cells to prevent severe GVHD. Feasibility will be tested, focusing on engraftment, treatment-related mortality (with a specific focus on interstitial pneumonitis) and severe GVHD.
详细描述
PRIMARY HYPOTHESIS: T cell depletion utilizing the CliniMACS device will allow more precise, specific and controlled graft engineering of peripheral blood stem cells from unrelated and partially matched related donors without an increase in relapse or graft rejection and grade III or IV acute graft vs. host disease (GVHD).
SECONDARY HYPOTHESIS: Use of the CliniMACS device will allow defined levels of T cell depletion to reflect the risk of severe GVHD in the donor/recipient pair.
Thus, patients with a relatively lower risk of severe GVHD will be assigned to Stratum 1 and receive a graft with lesser T cell depletion and a defined level of reinfused T cells. Patients with higher risk of severe GVHD or for whom there is no perceived clinical benefit of GVHD will be assigned to Stratum 2 and receive a more T cell-depleted graft.
Conditioning of the patient (except immunodeficiencies) includes :
- Thiotepa 5 mg/kg days for 2 days
- Cyclophosphamide 60 mg/kg days for 2 days
- Total body irradiation 200 cGy given twice a day for 3 days
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 22 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •As of October 2014 this study closed enrollment to malignant diseases. This study remains open to:
- •Non-malignant diseases:
- •Bone marrow failure, including severe aplastic anemia
- •Immunodeficiencies
排除标准
- •Patients who have had prior stem cell transplant (SCT) and bone marrow transplant (BMT) are excluded for study enrollment.
研究组 & 干预措施
1. CliniMACS CD3+/CD19+ depletion
- 6/6 or 8/8 matched (fully matched)
- 1 antigen or allele mismatched (mismatch at A or B or DRB1)
- 2 antigen or allele mismatched (mismatch ONLY at A and B but NOT at DRB1 plus either A or B).
Patients will receive grafts that have undergone CD3+ and CD19+ depletion. The CD3(-) fraction will be infused.
干预措施: CliniMACs (Device)
2. CliniMACS CD3+/CD19+ depletion
Stratum 2. CliniMACS CD3+/CD19+ depletion:
- Haploidentical match
- 2 antigen and/or allele mismatched where one of the mismatches includes DRB1
For patients in Stratum 2 we will perform CD3+ (T cell) and CD19+ (B cell) depletion. There will be no T cell add back in this stratum.
干预措施: CliniMACs (Device)
结局指标
主要结局
Number of Participants With Successful Engraftment
时间窗: 100 days Post Transplant
Successful engraftment will be whether subjects have achieved an absolute neutrophil count (ANC) \>500 and platelet count \>20 x 109/l by 100 days after transplant
次要结局
未报告次要终点
研究者
Nancy Bunin
BMT Medical Director
Children's Hospital of Philadelphia