A Single-Arm, Prospective Clinical Study of Venetoclax Combined With Azacitidine Followed by Bridging Transplantation in Patients With High-Risk Myelodysplastic Neoplasms With Increased Blasts 2 (MDS-IB2)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 46
- 试验地点
- 1
- 主要终点
- 2-Year Relapse-Free Survival(2-RFS)
研究概览
简要总结
A Single-Arm, Prospective Clinical Study of Venetoclax Combined with Azacitidine Followed by Bridging Transplantation in Patients with High-Risk Myelodysplastic Neoplasms with Increased Blasts 2 (MDS-IB2)
详细描述
This is a single-center, single-arm, prospective study investigating pre-transplant bridging therapy in patients aged ≥18 years with higher-risk MDS-IB2. The study plans to enroll 46 eligible patients. Participants will receive Venetoclax combined with Azacitidine, administered in 28-day cycles for 1 to 2 cycles. Treatment response will be assessed according to the IWG 2023 HR-MDS response criteria.Patients achieving modified Composite Complete Remission (mCRc: CR or CR-equivalent + CRL + CRh) after Cycle 1 will proceed directly to transplantation. Patients not achieving mCRc will receive a second cycle of therapy. All patients will undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) within 3 months after completing Cycle 2, regardless of mCRc status. Patients unable to proceed to transplantation will receive standard institutional care and undergo follow-up.
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
- •Newly diagnosed MDS confirmed by morphological and immunophenotypic analysis of bone marrow；
- •Age ≥18 years, any gender；
- •Bone marrow blasts ≥10%；
- •IPSS-R score >4.5；
- •ECOG performance status 0-2；
- •Scheduled for allogeneic hematopoietic stem cell transplantation (allo-HSCT)；
- •Adequate major organ function：
- •Cardiac: LVEF ≥50%
- •Hepatic: Bilirubin ≤1.5×ULN
- •AST/ALT ≤2.5×ULN
- •Renal: Creatinine clearance ≥60 mL/min；
- •Written informed consent provided by the patient or legally authorized representative.
排除标准
- •Extramedullary disease involvement；
- •Hypersensitivity to any study drugs；
- •Clinically significant hepatic/renal dysfunction exceeding inclusion thresholds；
- •Severe cardiac disease, including congestive heart failure, myocardial infarction, and cardiac insufficiency;
- •Concurrent malignant tumors of other organs, which can be enrolled if previously cured;
- •Active tuberculosis or HIV infection;
- •Concomitant hematologic disorders;
- •Pregnancy or lactation;
- •Inability to comply with protocol requirements;
- •Concurrently participating in other clinical studies.
研究组 & 干预措施
venetoclax + Azacitidine
干预措施: VEN (Venetoclax) (Drug)
结局指标
主要结局
2-Year Relapse-Free Survival(2-RFS)
时间窗: From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months.
Defined as the time from enrollment in this study to the occurrence of relapse. For patients without relapse, the time is calculated until death from any cause or the time of the last follow-up
次要结局
- Overall Survival (OS)(From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 48 months.)
- Cumulative Relapse Rate (CIR)(rom date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months.)
- Composite Complete Response Rate（mCRc）(Through study completion, an average of 1 year)
研究者
Yehui Tan
Professor
The First Hospital of Jilin University