Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Number of Participants With Hematologic Response
研究概览
简要总结
RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib followed by stem cell transplant works in treating patients with primary systemic amyloidosis.
详细描述
OBJECTIVES:
- To determine if hematologic responses to high-dose melphalan and autologous stem cell transplantation increase with addition of bortezomib in the conditioning regimen in patients with primary systemic amyloidosis.
OUTLINE:
- Autologous stem cell mobilization and collection: Patients receive filgrastim to mobilize stem cells, which are then collected.
- Conditioning regimen: Patients receive bortezomib intravenously on days -6, -3, 1, and 4 and oral high-dose melphalan on days -2 and -1.
- Stem cell transplantation: Patients undergo autologous stem cell transplantation on day 0.
After completion of study therapy, patients are followed every 6 months for 1 year and 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
- 未提供
研究组 & 干预措施
Stem Cell Transplant with Bortezomib and Melphalan
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
干预措施: filgrastim (Biological)
Stem Cell Transplant with Bortezomib and Melphalan
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
干预措施: bortezomib (Drug)
Stem Cell Transplant with Bortezomib and Melphalan
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
干预措施: melphalan (Drug)
Stem Cell Transplant with Bortezomib and Melphalan
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
干预措施: Stem Cell Infusion (Procedure)
结局指标
主要结局
Number of Participants With Hematologic Response
时间窗: one year
complete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy \<5% plasma cells with no clonal predominance of kappa or lambda isotype. Partial response: any one of the following 1. For patients with detectable and quantifiable marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. 2. For patients with a detectable monoclonal peak on serum protein electropheresis or urine protein electropheresis, a reduction in the peak height of 50% or more. 3. For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume).
次要结局
- Number of Participants Surviving at 100 Days From Transplant(100 Days from transplant date)
- Number of Participants Surviving at 1 Year(one year from transplant)
- Number of Participants Surviving at 2 Years(2 years from transplant)
研究者
Vaishali Sanchorawala
Principal Investigator
Boston Medical Center