Thalidomide, Cyclophosphamide and Prednisone in Newly Diagnosed Multicentric Castleman's Disease: a Prospective, Single-center, Single-arm, Phase-II Pilot Trial
试验速览
- 阶段
- 2 期
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Number of Patients With Durable Tumor and Symptomatic Response
研究概览
简要总结
To explore the effectiveness and safety of thalidomide, cyclophosphamide and prednisone (TCP regimen) in newly diagnosed Multicentric Castleman's disease (MCD) patients.
详细描述
This is a single center, open-labeled , single arm, phase-II pilot study which aims to evaluate the efficacy and safety of thalidomide, cyclophosphamide and prednisone (TCP regimen) in newly diagnosed Multicentric Castleman's disease (MCD) patients.There would be two phases of the study. The treatment and the response evaluation phase will last from the time of enrollment up to 24 months (evaluation will be carried out every 3 months). The follow-up phase to assess for progression of disease will last from 24 months (2 years) to 4 years 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
盲法说明
open-labeled
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
- •≥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 MCD 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);
- •Eastern Cooperative Oncology Group (ECOG) Performance Status ≤2;
- •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;
- •ECOG (eastern cooperative oncology group) status above 2;
- •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
TCP regimen
Thalidomide, cyclophosphamide and prednisone （TCP regimen）would be used for newly-diagnosed symptomatic MCD patients
干预措施: Thalidomide, cyclophosphamide and prednisone (Drug)
结局指标
主要结局
Number of Patients With Durable Tumor and Symptomatic Response
时间窗: From baseline to the time point when a patient achieves treatment response for 24 weeks.
Durable tumor and symptomatic response is complete response (CR) + partial response (PR). CR: complete disappearance of all measurable and evaluable disease (eg, pleural effusion) and resolution of baseline symptoms attributed to multicentric Castleman's disease, sustained for at least 18 weeks. PR: \>=50 percent decrease in sum of the product of the diameters of indicator lesion(s), with at least stable disease in all other evaluable disease in the absence of treatment failure sustained for at least 6 months.
次要结局
- Progression-free Survival(From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months)
- Overall Survival(From date of randomization until the date of death from any cause, assessed up to 36 months.)
- Change in SF-36 Score(From baseline to 24 weeks after treatment.)
- Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 ( ≥1 Grade)(From initiation of TCP regimen to 3 months after the end of treatment or to time point of the initiation of second line therapy.)
- Number of Participants With Treatment-related Serious Adverse Events as Assessed by CTCAE v4.0 ( ≥3 Grade)(From initiation of TCP regimen to 3 months after the end of treatment or to time point of the initiation of second line therapy.)
研究者
Jian Li
Associated professor in hematology
Peking Union Medical College Hospital