A Multi-center, Phase II, Single-arm Clinical Study of Venetoclax Combined With Azacytiside in the Treatment of Myelodysplastic/Myeloproliferative Neoplasms in Adults
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 33
- 试验地点
- 1
- 主要终点
- Overall Response Rate (ORR)
研究概览
简要总结
To explore the efficacy of venetoclax combined with azacytidine in Myelodysplastic / myeloproliferative neoplasms（MDS/MPN）, so as to improve the overall survival and treatment status of MDS/MPN patients.
详细描述
At present, there is no standardized treatment strategy for MDS/MPN. The purpose of our study is to explore the efficacy of venetoclax combined with azacytidine in the treatment of MDS/MPN, so as to improve the overall survival and treatment status of patients with MDS/MPN. After the participants were treated with four cycles of venetoclax combined with azacytidine, the efficacy was evaluated according to the 2015 adult MDS/MPN response criteria to determine the disease status. Participants with disease progression and intolerance withdrew from the study during treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 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
- •Male or female, Age (years) >= 18；
- •Patients newly diagnosed or previously treated with MDS/MPNs (CMML, MDS/MPN-U, aCML) according to 2016 WHO diagnostic criteria:
- •Initial diagnosis: CMML: CPSS-mol intermediate risk 2 and above; aCML; MDS/MPN-U.
- •Previous treatment: HMA treatment failed.
- •Eastern Cooperative Oncology Group (ECOG) Performance status of 0,1, 2 ;
- •Liver function: Total bilirubin ≤3 upper limit of normal (ULN); aspartate aminotransferase (AST) ≤3 ULN; alanine aminotransferase (ALT)≤3 ULN；
- •Renal function#Ccr ≥30 ml/min;
- •Patients who sign the informed consent must have the ability to understand and be willing to participate in the study and sign the informed consent.
排除标准
- •Acute myeloid leukemia
- •Myelodysplastic syndrome
- •Subjects who had previously been treated with Venetoclax
- •Subjects who are known to be allergic to ingredients of the study drug or their analogues
- •HIV infection
- •HBV-DNA or HCV-RNA positive
- •Subjects with grade 2 or above cardiac failure and those considered unsuitable for inclusion by the investigator
- •Subjects who are pregnant or breastfeeding
- •Subjects reject to participate in the study
研究组 & 干预措施
Treatment regime
On day 1 of each cycle, decitabine 75 mg/m2 will be given subcutaneously, and will continue for 5 days. Simultaneously the patient will start out with Venetoclax 100mg and progress to 400mg until the 14 day cycle is finished.
干预措施: venetoclax combined with azacitidine (Drug)
结局指标
主要结局
Overall Response Rate (ORR)
时间窗: Study start date to study end date, or death, whichever comes first, up to 4 years
ORR (equals the rates of complete remission \[CR\]+partial remission \[PR\]+complete cytogenetic remission \[CCyR\]+marrow response \[MR\[+clinical benefit \[CB\] )of venetoclax in combination with azacitidine. 1. CR and CCyR are shown in the secondary outcome measures below. 2. PR: Normalization of peripheral counts and hepatosplenomegaly with bone marrow blasts (and blast equivalents) reduced by 50%, but remaining\>5% of cellularity except in cases of MDS/MPN with≤5% bone marrow blasts at baseline. 3. MR: Optimal marrow response: Presence of all marrow criteria necessary for CR without normalization of peripheral blood indices. Partial marrow response: Bone marrow blasts (and blast equivalents) reduced by 50%, but remaining\>5% of cellularity, or reduction in grading of reticulin fibrosis from baseline on at least 2 bone marrow evaluations spaced at least 2 months apart. 4. CB: Hematology improvement, spleen response and symptom response.
次要结局
- Complete remission rate(Study start date to study end date, or death, whichever comes first, up to 4 years)
- Complete remission rate of bone marrow morphology(Study start date to study end date, or death, whichever comes first, up to 4 years)
- Hematology improvement (HI) rate(Study start date to study end date, or death, whichever comes first, up to 4 years)
- Spleen response rate(Study start date to study end date, or death, whichever comes first, up to 4 years)
- Symptom response rate(Study start date to study end date, or death, whichever comes first, up to 4 years)
- Complete cytogenetic remission rate(Study start date to study end date, or death, whichever comes first, up to 4 years)
- Incidence of severe infection (≥grade 3 )(Study start date to study end date, or death, whichever comes first, up to 4 years)
研究者
Chen Suning
Head of Hematology Laboratory
The First Affiliated Hospital of Soochow University