A Comparative Study of Bortezomib-Thalidomide-Dexamethason and Bortezomib-Cyclophosphamide-Dexamethason in the Treatment of Monoclonal Immunoglobulin Light Chain Amyloidosis: A Prospective Randomized Controlled Trial（BTD-CHINA-TRIAL）
试验速览
- 阶段
- 4 期
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- Hematologic Response
研究概览
简要总结
Research Objective:At present, there is no standard therapeutic regimen for monoclonal immunoglobulin light chain (AL) amyloidosis in the world. To compare the efficacy and safety of the regimens between bortezomib-thalidomide-dexamethasone (BTD) and bortezomib-cyclophosphamide-dexamethasone (BCD) in the treatment of AL amyloidosis, so as to provide more clinical evidence for the standard treatment for the disease.
Research Design:This study was designed as a prospective, randomized and controlled clinical study. Patients who meet the inclusion criteria of this study will be randomized to the BTD scheme group or BCD scheme group.
详细描述
Object and source:Patients meeting the inclusion criteria of AL amyloidosis are enrolled into the study. Screening failure rate ≤ 30%, follow-up dropout rate ≤ 10%.
Sample size evaluation: The complete remission rate (CR) and the very good partial remission rate (VGPR) were used as statistical indexes for hematology remission rate estimation. Referring to the previous literature, the CR and VGPR of bortezomib-thalidomide-dexamethasone (BTD) is 83%, and CR and VGPR of bortezomib-cyclophosphamide-dexamethasone (BCD) is 43%. The boundary value is 0.1, according to the level of significance level α = 0.05, test efficacy 1 - β = 0.8, using the method of effectiveness test, using pass11 software calculation to estimate the sample size to be included in the group, the expected dropout rate is 20%, the total sample size is 70, 35 in each group.
Observation index:Before and after treatment, physical examination should be carried out every month. The following test should be required. Blood pressure
, blood routine, liver function, electrolyte, 24h urine volume, 24h urine protein quantity, urine protein/creatinine ratio, blood uric acid, serum albumin, urea nitrogen, creatinine, eGFR(estimated Glomerular Filtration Rate), blood lipids, infection index, NT-proBNP (N terminal pro B type natriuretic peptide), TNT(Troponin-T), blood and urine free light chain should be detected respectively.
Quality assurance plan:The study shall be conducted in accordance with the current approved protocol and the July 1996 ICH drug clinical study management code (CPMP/ICH / 135/95) (ICH GCP), the Helsinki declaration, regulations and standard operating procedures.
研究设计
- 研究类型
- Interventional
- 分配方式
- 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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1.Signed the written informed consent; 2.18 years old ≤ age ≤ 80 years old, no restriction on gender; 3.AL amyloidosis was confirmed by pathological biopsy in the accumulated system or organ (kidney, heart, liver, skin), and excluded other secondary factors; 4.The proliferation of monoclonal plasma cells was confirmed by fixed electrophoresis of bone marrow or blood/urine.
排除标准
- •Pathological biopsy showed non-AL amyloidosis;
- •Abnormal proliferation of plasma cells reached the standard of multiple myeloma；
- •Other hematological system tumors;
- •Cushing's syndrome;
- •Active hepatitis;
- •5.Pregnant or lactating women;
研究组 & 干预措施
Group1
Group1 is the AL amyloidosis patients who have bortezomib-thalidomide-dexamethason-based regimens for their treatment.
干预措施: Thalidomide (Drug)
Group2
Group2 is the AL amyloidosis patients who have bortezomib-cyclophosphamide-dexamethason-based regimens for their treatment.
干预措施: Cyclophosphamide (Drug)
结局指标
主要结局
Hematologic Response
时间窗: 1 year
According to the criteria of hematologic response of AL amyloidosis.
次要结局
- Organ Response(1 year)