Bortezomib, Cyclophosphamide and Dexamethasone (BCD) in Newly Diagnosed Idiopathic Multicentric Castleman's Disease (iMCD) : a Prospective, Single-center, Single-arm, Phase-II Pilot Trial
试验速览
- 阶段
- 2 期
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Overall response
研究概览
简要总结
To explore the effectiveness and safety of bortezomib, cyclophosphamide and dexamethasone (BCD regimen) in newly diagnosed idiopathic Multicentric Castleman's disease (iMCD) patients.
详细描述
This will be a single center, open-labeled, single arm, phase-II pilot study. The treatment and the response evaluation phase will last from the time of enrollment up to 21 months (evaluation will be carried out every 3 months in the first 9 months and every 6 months from Month 9 to Month 21). The maintenance and follow-up phase to assess for progression of disease will last from 21 months to 45 months after enrollment (evaluation will be carried out every 12 months). The total study duration will be 4 years after the last patient starts study medication.
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
- •Demography: ≥18 years, all race/ethnic groups in China;
- •Newly diagnosed and previously untreated (patients are allowed to have received oral prednisone for up to 1 week before enrollment) symptomatic iMCD patients (symptomatic disease is defined by the presence of clinical symptoms with the NCI-CTCAE grading ≥1 that are attributable to the disease, and for which treatment is indicated; iMCD diagnosis is based on the international consensus diagnostic criteria);
- •Clinical laboratory values meeting these criteria at screening: absolute neutrophil count ≥ 1·0 x 109/L, Platelets ≥ 50 x 109/L, Alanine aminotransferase (ALT) within 2·5 x upper limit of normal (ULN); total bilirubin within 2·5 x ULN; estimated glomerular filtration rate (according to MDRD formula) <15ml/min;
- •Women of childbearing potential must agree to use birth control measures during the study and for at least 3 months after receiving the last dose of study agent, and must have a negative pregnancy test at screening period. Men must agree to use birth control measures during the study and for at least 3 months after receiving the last dose of study agent;
- •Informed consent must be signed.
排除标准
- •age under 18 years;
- •Immunosuppressive or anti-neoplastic drugs within the last 3 months;
- •serious diseases including malignancy;
- •Plan to have babies within 1 year after enrollment (for women and men), or pregnancy / breast-feeding (for women);
- •Known hypersensitivity to study agents;
- •Active infection requiring systemic treatment;
- •Other severe concurrent disease (eg. uncontrolled diabetes, symptomatic coronary heart disease) that is likely to interfere with study procedures or results, or that in the opinion of the investigator would constitute a hazard for participating in this study;
- •Unwilling or unable to provide informed consent;
- •Unwilling to return for follow-up at PUMCH.
研究组 & 干预措施
BCD regimen
Bortezomib, cyclophosphamide and dexamethasone (the BCD regimen) would be utilized in newly diagnosed iMCD (idiopathic Multicentric Castleman's disease) patients
干预措施: Bortezomib (Drug)
BCD regimen
Bortezomib, cyclophosphamide and dexamethasone (the BCD regimen) would be utilized in newly diagnosed iMCD (idiopathic Multicentric Castleman's disease) patients
干预措施: Cyclophosphamide (Drug)
BCD regimen
Bortezomib, cyclophosphamide and dexamethasone (the BCD regimen) would be utilized in newly diagnosed iMCD (idiopathic Multicentric Castleman's disease) patients
干预措施: Dexamethason (Drug)
结局指标
主要结局
Overall response
时间窗: 12 months after the last patient begins study treatment.
Overall response is composed by biochemical, lymph node and symptom response, is the primary outcome of this study. According to the CDCN response criteria, an overall CR (complete response) requires a complete biochemical, lymph node, and symptomatic response; and overall PR (partial response) requires nothing less than a PR across all categories, but not meeting criteria for CR; an overall SD (stable disease) requires no PD (progression disease) in any of the categories and not meeting the criteria for CR or PR; an overall PD occurs when any category has a PD.
次要结局
- Time to initial response(12 months after the last patient begins study treatment.)
- Progression-free survival (PFS)(12 months after the last patient begins study treatment.)
- Time to best response(12 months after the last patient begins study treatment.)
- Overall survival (OS)(12 months after the last patient begins study treatment.)
- Change in PHQ-9 score(From Day 1 of the BCD treatment until 12 months after the treatment)
- Change in hemoglobin level(From baseline until 12 months after the treatment)
- Change in IL-6 (interleukin-6)(From baseline until 12 months after the treatment)
- Change in CRP(From baseline until 12 months after the treatment)
- Change in ESR(From baseline until 12 months after the treatment)
- Change in IgG level(From baseline until 12 months after the treatment)
- Change in MCD-related overall symptom score(From baseline until 12 months after the treatment)
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 ( ≥1 grade)(12 months after the last patient begins study treatment.)
- Number of participants with treatment-related serious adverse events as assessed by CTCAE v4.0 ( ≥3 grade)(12 months after the last patient begins study treatment)
研究者
Jian Li
Professor in hematology
Peking Union Medical College Hospital