A Phase 2, Open-Label, Single Arm, Multi-Center Study to Assess the Efficacy and Safety of BST-236 as a Single Agent in Adults Unfit for Intensive Chemotherapy With Relapsed or Refractory AML or Higher-Risk Myelodysplastic Syndromes
试验速览
- 阶段
- 2 期
- 入组人数
- 40
- 试验地点
- 4
- 主要终点
- Overall response rate (ORR)
研究概览
简要总结
An open label multi center study to assess the safety and efficacy of BST-236 as single agent in adult patients unfit for standard therapy with Acute Myeloid Leukemia (AML) or higher-risk (HR) Myelodysplastic Syndromes (MDS) who fail to respond or relapsed following first line therapy.
Approximately 20 adult patients with relapsed and/or refractory AML and approximately 20 adult patients with relapsed and/or refractory HR MDS, will be enrolled into the study.
Patients will be treated with 1-2 induction courses and 2-4 maintenance courses. All patients will be followed for 1 year in the study and additional 1 year post study 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
- 未提供
排除标准
- •MDS or AML evolving from a pre-existing myeloproliferative neoplasm (MPN)
- •MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (CML), juvenile myelomonocytic leukemia (JMML) and unclassifiable MDS/MPN
- •Acute promyelocytic leukemia
- •Previous treatment for AML or MDS with drugs other than HMA or LDAC or combinations of venetoclax with either HMA or LDAC
- •Previous allogeneic hematopoietic stem cell transplantation (HSCT) or solid organ transplantation
- •Participation in a previous clinical trial involving use of an investigational drug within 30 days or at least 5 half-lives of tested drug (whichever is shorter) of study day 1
- •Peripheral White Blood Cell (WBC) count >30,000 /µL in the 48 hours prior to first BST-236 dose administration. Hydroxyurea administration or leukapheresis is permitted to meet this criterion
- •Administration of HMA, LDAC, or venetoclax within 14 days prior to Study Day 1
- •Previous treatment with cytarabine at a dose higher than 20 mg/ m2/d
- •Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment)
- •Any medical or surgical condition, presence of laboratory abnormalities or psychiatric illness that may preclude safe and complete study participation based on the Investigator's judgment
- •Diagnosis of malignant disease (other than AML) within the previous 12 months (excluding basal cell carcinoma of the skin without complications, "in-situ" carcinoma, or other local malignancy excised or irradiated with a high probability of cure and not treated with systemic or topical chemotherapy)
- •Surgical procedure, excluding central venous catheter placement or other minor procedures (e.g. skin biopsy) in the 14 days prior to first BST-236 dose administration
- •History of allergic reactions attributed to compounds of similar chemical composition as BST-236 and/or cytarabine
- •Life expectancy shorter than 3 months attributed to any known medical condition other than AML/MDS
- •In 12 leads ECG, corrected QT interval (QTc)>480msec or history of QT prolongation or Torsades de pointes
研究组 & 干预措施
BST-236
BST-236 Intravenous, 4.5 g/m^2/d or 2.3 g/m^2/d, for 6 days
干预措施: BST-236 (Drug)
结局指标
主要结局
Overall response rate (ORR)
时间窗: To be assessed 5 months after the last patient was enrolled to the study
In MDS patients -Overall response rate (ORR), defined as the proportion of patients who achieve a CR or PR per proposal for modification of the International Working Group (IWG) criteria for MDS, 2006
CR rate
时间窗: To be assessed 5 months after the last patient was enrolled to the study
In AML patients -The proportion of patients who achieve a CR per the IWG 2006 Criteria
次要结局
未报告次要终点