A Phase 2, Randomized, Open-Label Study of Azacitidine (Vidaza) vs MGCD0103 vs Azacitidine in Combination With MGCD0103 for the Treatment of Elderly Subjects With Newly Diagnosed AML or Intermediate-2 or High-Risk MDS
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 6
- 试验地点
- 8
- 主要终点
- Overall response rate as assessed using IWG criteria for AML and MDS
研究概览
简要总结
The purpose of the study is to determine how effective azacitidine, MGCD0103, and the combination of azacitidine and MGCD0103 are in treating AML or MDS in people over 60 years of age.
详细描述
This randomized, 3-arm Phase 2 study will compare the safety and efficacy of single-agent azacitidine (currently 1 of 3 approved treatments for myelodysplastic syndrome [MDS]) to that of single-agent MGCD0103 and to that of combination therapy with MGCD0103 and azacitidine in elderly patients with acute myelogenous leukemia (AML) or intermediate-2 (Int-2) or high-risk MDS, for whom no standard of care exists. The goal of the study is to determine which of the 3 treatment arms are worthy of further investigation in a subsequent Phase 3 study of elderly subjects with AML or Int-2 or high-risk MDS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 60 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Able to provide written informed consent, and be willing and able to comply with all the study procedures
- •Must be 60 years of age or older
- •Must have a pathologic confirmation of newly diagnosed (de novo or untreated secondary) AML or newly diagnosed Int-2 or high-risk MDS (IPSS classification) according to WHO criteria
- •Must have a Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- •Must have adequate organ function, including total bilirubin ≤ 1.5 x upper limit of normal (ULN); AST & ALT ≤ 2.5 x ULN; and serum creatinine ≤ 2.0 x ULN.
排除标准
- •Considered fit for intensive chemotherapy and opt to be treated with intensive chemotherapy
- •Prior transplantation or any prior anticancer therapy (standard or investigational, including chemotherapy, treatment with HDAC inhibitors, or combination HDAC and azacitidine) administered to treat AML or MDS.
- •Clinical evidence of central nervous system (CNS) involvement by leukemia
- •A diagnosis of promyelocytic leukemia
- •Previous or concurrent malignancy except adequately treated basal cell or squamous cell skin cancer; in situ carcinoma of the cervix, or other solid tumor treated curatively, and without evidence of recurrence for at least 3 years prior to study entry
- •Active and uncontrolled clinically significant infection
- •Known positive serology for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV Ab) or human immunodeficiency virus (HIV)
- •Less than 4 weeks elapsed since any major surgery
- •Any prior or active disease that may interfere with the procedures or evaluations to be conducted in the study
研究组 & 干预措施
A
Azacitidine
干预措施: Azacitidine (Drug)
B
MGCD0103
干预措施: MGCD0103 (Drug)
C
Azacitidine + MGCD0103
干预措施: Azacitidine (Drug)
C
Azacitidine + MGCD0103
干预措施: MGCD0103 (Drug)
结局指标
主要结局
Overall response rate as assessed using IWG criteria for AML and MDS
时间窗: After 45, 90, 135, and 180 subjects are enrolled and evaluated for response to treatment
次要结局
- Duration of response; Time to progression; Progression-free survival; RBC transfusion independence; Hematologic improvement; Quality of life; Safety profile; and Pharmacokinetics of azacitidine and MGCD0103(After 45, 90, 135, and 180 subjects are enrolled and evaluated for response to treatment)