Phase II Trial of Second Autologous Transplantation in AL Amyloidosis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Feasibility and Tolerability
研究概览
简要总结
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly.
PURPOSE: This phase II trial is studying how well autologous stem cell transplant works in treating patients with persistent or recurrent primary systemic (AL) amyloidosis.
详细描述
OBJECTIVES:
- Determine the feasibility and tolerability of second autologous stem cell transplantation in patients with persistent or recurrent AL amyloidosis.
- Determine the response rate and durability of response in patients treated with this regimen.
- Determine immune reconstitution in patients treated with this regimen.
OUTLINE:
- Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily beginning before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection.
- Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2.
- Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.
Patients are followed at 6 months, 1 year, and then annually thereafter.
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •No myelodysplastic syndromes
- •No abnormal bone marrow cytogenetics
- •Not pregnant or nursing
- •Fertile patients must use effective contraception
- •Acceptable toxicity from first transplantation, confirmed by the transplant team
- •HIV negative
- •No other concurrent malignancy except treated skin cancer
研究组 & 干预措施
2nd Stem Cell Transplant
Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion
干预措施: filgrastim (Biological)
2nd Stem Cell Transplant
Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion
干预措施: melphalan (Drug)
2nd Stem Cell Transplant
Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion
干预措施: autologous stem cell transplantation (Procedure)
2nd Stem Cell Transplant
Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion
干预措施: stem cell infusion (Procedure)
结局指标
主要结局
Feasibility and Tolerability
时间窗: 3 months after treatment and annually
Feasibility and tolerability will be evaluated based on participants completing second transplant with tolerable adverse events
Response and Durability of Response
时间窗: 3 months after treatment and annually
Response and durability of response will be based on hematologic Complete Response or Partial Response and date of relapse or death
Evaluate Immune Reconstitution
时间窗: 3 months after treatment and annually
Evaluate immune reconstitution based on time to engraftment
次要结局
未报告次要终点
研究者
Karen Quillen
Medical Director, Blood Bank
Boston Medical Center