Phase III Trial of Stem Cell Transplantation Compared to Parenteral Melphalan and Oral Dexamethasone in the Treatment of Primary Systemic Amyloidosis (AL)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 89
- 试验地点
- 1
- 主要终点
- Hematologic Response Rate
研究概览
简要总结
RATIONALE: Drugs used in chemotherapy, such as melphalan and dexamethasone, work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having an autologous 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. It is not yet known whether combination chemotherapy is more effective than chemotherapy followed by an autologous stem cell transplant in treating primary systemic amyloidosis.
PURPOSE: This randomized phase III trial is studying the side effects and how well giving low-dose melphalan together with dexamethasone works compared with high-dose melphalan followed by an autologous stem cell transplant in treating patients with primary systemic amyloidosis.
详细描述
OBJECTIVES:
Primary
- Compare hematologic response rate in patients with primary systemic amyloidosis treated with conventional chemotherapy comprising low-dose melphalan and dexamethasone vs high-dose melphalan followed by autologous stem cell transplantation.
- Compare the toxicity of these regimens in these patients.
Secondary
- Compare the overall and progression-free survival of patients treated with these regimens.
- Compare the regression of organ involvement in patients treated with these regimens.
- Compare the duration of response in patients treated with these regimens.
- Correlate clonal burden and time to in vitro amyloid formation with clinical outcomes in patients treated with these regimens.
- Compare quality of life of patients treated with these regimens.
- Compare the information-seeking behavior in patients treated with these regimens.
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
- 18 Years 至 —（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
Arm B
Patients receive filgrastim (G-CSF) on days -7 to -3 and undergo autologous hematopoietic stem cell (HSC) collection. Patients receive high-dose melphalan IV over 1 hour on days -2 and -1 and undergo autologous HSC transplantation on day 0.
干预措施: melphalan (Drug)
Arm A
Patients receive low-dose melphalan IV over 15-30 minutes on day
1 or orally once daily on days 1-7 and oral dexamethasone on days 1-4 and 22-25. Treatment repeats every 6 weeks for 10 courses.
Study treatment beyond one year is not allowed.
干预措施: dexamethasone (Drug)
Arm A
Patients receive low-dose melphalan IV over 15-30 minutes on day
1 or orally once daily on days 1-7 and oral dexamethasone on days 1-4 and 22-25. Treatment repeats every 6 weeks for 10 courses.
Study treatment beyond one year is not allowed.
干预措施: melphalan (Drug)
Arm B
Patients receive filgrastim (G-CSF) on days -7 to -3 and undergo autologous hematopoietic stem cell (HSC) collection. Patients receive high-dose melphalan IV over 1 hour on days -2 and -1 and undergo autologous HSC transplantation on day 0.
干预措施: filgrastim (Biological)
Arm B
Patients receive filgrastim (G-CSF) on days -7 to -3 and undergo autologous hematopoietic stem cell (HSC) collection. Patients receive high-dose melphalan IV over 1 hour on days -2 and -1 and undergo autologous HSC transplantation on day 0.
干预措施: autologous hematopoietic stem cell transplantation (Procedure)
结局指标
主要结局
Hematologic Response Rate
时间窗: 10 years
Response that was confirmed on 2 consecutive evaluations during treatment. A hematologic response consisted of a Complete response, Very Good Partial Response or Partial Response. * Complete Response (CR): Complete disappearance of M-protein from serum and urine on immunofixation, normalization of Free Light Chain (FLC) ratio and \<5% plasma cells in bone marrow. * Very Good Partial Response (VGPR): \>=90% reduction in serum M-component; Urine M-Component \<=100 mg per 24 hours. * Partial Response (PR): \>=50% reduction in serum M-component and/or Urine M-Component \>=90% reduction or \<200 mg per 24 hours; or \>=50% decrease in difference between involved and uninvolved FLC levels.
次要结局
- 3 Year Overall Survival(3 years)
- Organ Response to Treatment(10 years)