A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of Romiplostim (AMG 531) Treatment of Subjects With Low or Intermediate Risk Myelodysplastic Syndrome (MDS) Receiving Hypomethylating Agents
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 69
- 主要终点
- Occurrence of a Clinically Significant Thrombocytopenic Event
研究概览
简要总结
The purpose of this study is to evaluate the effect of Romiplostim (AMG 531) on the incidence of clinically significant thrombocytopenic events (grade 3 or 4 and/or receipt of platelet transfusions) in subjects with low or intermediate risk Myelodysplastic Syndrome (MDS) receiving hypomethylating agents. It is hypothesized that Romiplostim administration, at the appropriate dose and schedule, will result in reduction in the incidence of clinically significant thrombocytopenic events in low or intermediate risk MDS subjects receiving hypomethylating agents.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Double (Participant, Investigator)
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
- •Diagnosis of MDS by bone marrow biopsy based on the World Health Organization (WHO) classification - Low, Intermediate-1 or Intermediate-2 risk category MDS using the IPSS (International Prognostic Scoring System) - Planned to receive either azacytidine 75 mg/m2 by subcutaneous administration each day for 7 days or decitabine 20 mg/m2 by intravenous administration each day for 5 days for at least 4 cycles
排除标准
- •Prior exposure to >3 cycles hypomethylating agents
- •Prior history of leukemia or aplastic anemia
- •Prior history of bone marrow transplantation
- •Prior malignancy (other than in situ cervical cancer or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for ³ 3 years before randomization
- •Active or uncontrolled infections
- •Unstable angina, congestive heart failure [NYHA (New York Heart Association) > class II], uncontrolled hypertension [diastolic > 100 mmHg], uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction
- •History of arterial thrombosis ( eg, stroke or transient ischemic attack) in the past year
- •History of venous thrombosis that currently requires anti-coagulation therapy
- •Received IL-11 within 4 weeks of screening
- •Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA approved for any indication
- •Have previously received any other thrombopoietic growth factor
研究组 & 干预措施
Dose level 1 750 AMG 531 (Part B - decitabine)
750 mcg AMG 531 weekly via subcutaneous injection + 20 mg/m2 decitabine for 4 cycles
干预措施: Decitabine (Drug)
Dose level 1 500 AMG 531 (Part A - azacitidine)
500 mcg AMG 531 weekly via subcutaneous injection + 75 mg/m2 azacitidine for 4 cycles
干预措施: AMG 531 (Romiplostim) (Biological)
Dose level 1 500 AMG 531 (Part A - azacitidine)
500 mcg AMG 531 weekly via subcutaneous injection + 75 mg/m2 azacitidine for 4 cycles
干预措施: Azacitidine (Drug)
Dose level 1 750 AMG 531 (Part B - decitabine)
750 mcg AMG 531 weekly via subcutaneous injection + 20 mg/m2 decitabine for 4 cycles
干预措施: AMG 531 (Romiplostim) (Biological)
Dose level 2 750 AMG 531 (Part A - azacitidine)
750 mcg AMG 531 weekly via subcutaneous injection + 75 mg/m2 azacitidine for 4 cycles
干预措施: AMG 531 (Romiplostim) (Biological)
Dose level 2 750 AMG 531 (Part A - azacitidine)
750 mcg AMG 531 weekly via subcutaneous injection + 75 mg/m2 azacitidine for 4 cycles
干预措施: Azacitidine (Drug)
Placebo (Part A - azacitidine)
Placebo weekly via subcutaneous injection + 75 mg/m2 azacitidine for 4 cycles
干预措施: Placebo (Drug)
Placebo (Part A - azacitidine)
Placebo weekly via subcutaneous injection + 75 mg/m2 azacitidine for 4 cycles
干预措施: Azacitidine (Drug)
Placebo (Part B - decitabine)
Placebo weekly via subcutaneous injection + 20 mg/m2 decitabine for 4 cycles
干预措施: Placebo (Drug)
Placebo (Part B - decitabine)
Placebo weekly via subcutaneous injection + 20 mg/m2 decitabine for 4 cycles
干预措施: Decitabine (Drug)
结局指标
主要结局
Occurrence of a Clinically Significant Thrombocytopenic Event
时间窗: Treatment period (up to 20 weeks)
Occurrence of a clinically significant thrombocytopenic event within the participant, defined as any platelet count obtained from day 15 of cycle 1 through the end of the interim follow-up visit that was less than 50 x 10\^9/L or receipt of platelet transfusions at any time through the interim follow-up visit.
次要结局
- Achieving an Overall Response (Complete or Partial Response, CR or PR) at the End of the Treatment Period(Treatment period (up to 20 weeks))
- Hypomethylating Agent Dose Reduction and Delay Due to Thrombocytopenia(Treatment period (up to 20 weeks))
- Platelet Transfusion(Study day 1 through the interim follow-up visit (up to 20 weeks))