Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in the Treatment of Hemophagocytic Lymphohistiocytosis
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Overall response rate
研究概览
简要总结
This study aimed to investigate the efficacy and safety of venetoclax combined with dexamethasone and etoposide as a salvage therapy for hemophagocytic lymphohistiocytosis.
详细描述
Hemophagocytic lymphohistiocytosis (HLH) is a rare and aggressive disease with high mortality and poor prognosis. The HLH-94 and HLH-04 regimens are most commonly used, but approximately 30% of the patients remain unresponsive. Currently, there is no unified evidence-based salvage treatment. There is no unified salvage regimen. This study plans to enroll 20 patients with refractory and recurrent hemophagocytic lymphohistiocytosis. Subjects will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.The safety was evaluated once a week, and the efficacy was evaluated once every 2 weeks. Overall response rate will be evaluated at 8 weeks. Patients were followed up every 3 months thereafter.
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
- •age ≥18 years old, expected survival time more than 3 months;
- •met HLH-2004 diagnostic criteria；
- •ECOG score 0-2；
- •ECG QTcF interval: male ≤450ms, female ≤470ms;
- •AST and ALT ≤3.0 ULN, TB ≤1.5×ULN；serum creatinine≤1.5×ULN，or CrCL ≥ 50mL/min；INR、APTT、PT ≤1.5×ULN；
- •without pregnancy or lactation, and agree to contraception during and for at least 6 months after the study；
- •signed informed consent.
排除标准
- •patients with malignancies unrelated to HLH, except for fully recovered non-melanoma skin cancer and carcinoma in situ;
- •patients participated in other clinical trials within 4 weeks；
- •previously treated with Bcl-2 inhibitors；
- •unable to take oral medication；
- •history of substance abuse or patients with mental illness；
- •severe infection；
- •cardiovascular disease，NYHA II-IV；
- •allergic to venetoclax or etoposide.
研究组 & 干预措施
Venetoclax Combined With Dexamethasone and Etoposide
Patients who met the inclusion criteria will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.
干预措施: Venetoclax (Drug)
Venetoclax Combined With Dexamethasone and Etoposide
Patients who met the inclusion criteria will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.
干预措施: Dexamethasone (Drug)
Venetoclax Combined With Dexamethasone and Etoposide
Patients who met the inclusion criteria will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.
干预措施: Etoposide (Drug)
结局指标
主要结局
Overall response rate
时间窗: 1 year
The percentage of cases with complete response (CR) and partial response (PR) after treatment in the total evaluable cases
Incidence and severity of adverse effects
时间窗: 1 years
The percentage of cases with adverse effects and its severity.
次要结局
- Overall survival(1 years)
研究者
Zhao Wang
Professor and Head of hematology
Beijing Friendship Hospital