Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction
研究概览
简要总结
OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation.
II. Determine the toxicity of this regimen in these patients.
详细描述
PROTOCOL OUTLINE: Patients may receive induction chemotherapy before study entry. Patients then receive filgrastim (G-CSF) or another growth factor for 4-6 days as peripheral blood stem cell (PBSC) mobilization. PBSC (or bone marrow) is harvested over 2-3 days.
Patients receive high-dose melphalan IV over 30 minutes twice daily on days -2 and -1. PBSC and/or bone marrow is reinfused on day 0. Patients receive G-CSF beginning on day 0 and continuing until blood counts recover. This course may be repeated 4-12 weeks later.
Patients are followed every 3 months for 1 year and then annually for 5 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •PROTOCOL ENTRY CRITERIA:
- •Disease Characteristics
- •Histologically confirmed primary amyloidosis
- •Ineligible for other high priority national or international study
- •Prior/Concurrent Therapy
- •Biologic therapy: Concurrent participation in gene therapy trials allowed
- •Chemotherapy: Prior chemotherapy allowed No other concurrent chemotherapy
- •Endocrine therapy: No concurrent steroids unless given with amphotericin B, for adrenal failure, or for septic shock No concurrent hormones except for non-disease-related conditions (e.g., insulin for diabetes)
- •Other: No concurrent barbiturates or acetaminophen Concurrent participation in supportive care trials allowed
- •Patient Characteristics
- •Performance status: ECOG 0-3
- •Hepatic: Bilirubin less than 2 times normal
- •Renal: Creatinine less than 2.5 mg/dL OR On stable hemodialysis
- •Pulmonary: DLCO at least 60% predicted OR Clearance by pulmonologist
- •Other: HIV negative
排除标准
- 未提供
结局指标
主要结局
Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction
次要结局
- Toxicity of high dose chemotherapy regimen