A Phase II Trial of the Immunomodulatory Drug CC-5013 for Patients With AL Amyloidosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 82
- 试验地点
- 1
- 主要终点
- Number of Patients Removed From Study Treatment Due to Toxicities
研究概览
简要总结
RATIONALE: Drugs such as CC-5013 and dexamethasone may be effective in treating primary systemic amyloidosis.
PURPOSE: This phase II trial is studying CC-5013 to see how well it works with or without dexamethasone in treating patients with primary systemic amyloidosis.
详细描述
OBJECTIVES:
Primary
- Determine the tolerability of CC-5013 in patients with primary systemic (AL) amyloidosis.
- Determine the objective hematologic response rate in patients treated with this drug.
- Determine amyloid organ disease response in patients treated with this drug.
Secondary
- Determine hematologic and amyloid organ disease response in patients who do not achieve a response to CC-5013 alone and are subsequently treated with CC-5013 and dexamethasone.
- Determine the toxicity of CC-5013 in combination with dexamethasone in these patients.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DISEASE CHARACTERISTICS:
- •Histologically confirmed primary systemic (AL) amyloidosis
- •Tissue amyloid deposits or positive fat aspirate
- •Meets 1 of the following criteria for AL type disease:
- •Serum or urine monoclonal protein by immunofixation electrophoresis
- •Plasmacytosis of bone marrow by monoclonal staining for kappa- or lambda-light chain isotype
- •PATIENT CHARACTERISTICS:
- •18 and over
- •Performance status
- •Life expectancy
- •Not specified
- •Hematopoietic
- •White blood count> 3,000/mm^3
- •Hemoglobin > 8 g/dL
- •Platelet count > 100,000/mm^3
- •Absolute neutrophil count > 1,000/mm^3
- •Bilirubin ≤ 2 times upper limit of normal (ULN)
- •aspartate aminotransferase (AST) and Alanine Aminotransferase (ALT) ≤ 2 times ULN
- •PRIOR CONCURRENT THERAPY:
- •Biologic therapy
- •Prior thalidomide for AL amyloidosis allowed
- •Chemotherapy
- •More than 4 weeks since prior cytotoxic chemotherapy
- •Endocrine therapy
- •Prior steroids for AL amyloidosis allowed
- •Radiotherapy
- •More than 4 weeks since prior radiotherapy
- •Prior surgery allowed
- •Recovered from all prior therapy
排除标准
- •No secondary or familial amyloidosis
- •No multiple myeloma, defined as ≥ 30% plasma cells in bone marrow biopsy specimen OR lytic bone lesions
- •No prior CC-5013
- •No dialysis
- •Cardiovascular
- •No symptomatic cardiac arrhythmia
- •No oxygen-dependent restrictive cardiomyopathy
- •No untreated or uncontrolled infection
- •No other malignancy except basal cell skin cancer or carcinoma in situ of the cervix or breast
- •No other serious medical illness that would preclude study participation
- •No history of hypersensitivity reaction to thalidomide
- •HIV negative
- •Not pregnant or nursing
- •Negative pregnancy test
- •Fertile patients must use effective contraception
研究组 & 干预措施
revlimid
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg BID) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
干预措施: dexamethasone (Drug)
revlimid
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg BID) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
干预措施: lenalidomide (Drug)
结局指标
主要结局
Number of Patients Removed From Study Treatment Due to Toxicities
时间窗: 1 year
Number of Patients With Hematologic Response With Single-agent CC-5013
时间窗: 3 months
Complete response = Absence of detectable monoclonal protein in serum or urine by immunofixation electrophoresis, less than 5% plasma cells on bone marrow biopsy without clonal dominance of kappa or lambda isotype, and normal serum free light chain assay. Partial response= For patients with detectable and quantifiable monoclonal marrow plasmacytosis= a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. For patients with a detectable monoclonal peak on serum or urine protein electrophoresis= a reduction in the peak height of 50% or more. For patients with quantifiable urinary kappa or lambda chain concentration= a 50% reduction in daily light chain excretion in 24 hour urine. For patients with an elevated serum free light chain assay, a reduction of 50% or more.
次要结局
- Number of Patients Who Received Both CC-5013 and Dexamethasone and Had a Hematologic Response(1 year)
研究者
Vaishali Sanchorawala
Principal Investigator
Boston Medical Center