A Prospective Open-Label Study of the Effectiveness of Epoetin Beta for Treating Anemic Patients With Low/Intermediate-1-Risk Myelodysplastic Syndrome (MDS)
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 100
- 试验地点
- 10
- 主要终点
- Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria
研究概览
简要总结
This is a Phase IV, prospective, multi-center, open-label study to assess the effectiveness and safety profile of epoetin beta (Recormon®) for treatment of symptomatic anemia in adult participants associated with low/intermediate-1-risk MDS. After screening, eligible participants will be treated with epoetin beta as recommended in the approved label and international guidelines for the use of epoetin in MDS participants and the dosage will be adjusted on the basis of erythroid response.
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
- •Adult participants with low or intermediate-1 risk MDS
- •No previous treatment with hematopoietic growth factors within 3 months prior to screening
- •Symptomatic anemia (hemoglobin <10 g/dL) as determined by investigator
- •Serum erythropoietin <500 milliunits/milliliter (mU/mL) within 14 days prior to the first dose of study treatment
- •Require no red blood cell transfusion or dependent on <4 units within 8 weeks prior to screening
- •Clinically stable for at least 1 month prior to entry into the study
- •For female participants of childbearing potential and male participants with partners of childbearing potential, agreement (by participants and/or partner) to use highly effective form(s) of contraception
排除标准
- •Contraindications and/or known hypersensitivity to the active substance and/or any of the excipients of epoetin beta treatment
- •Poorly controlled hypertension as assessed by the investigator
- •History of Acute Myeloid Leukemia (AML) or high risk for AML
- •Administration of another investigational drug within 1 month before screening or planned during the study period
- •Previously documented evidence of Pure Red Cell Aplasia (PRCA)
研究组 & 干预措施
Epoetin Beta
Participants will receive epoetin beta at an initial dose of 30,000 International Units (IU) per week administered subcutaneously (SC). Response will be firstly evaluated at Week 4 and the subsequent dose will be based on the response: if hemoglobin level reaches greater than or equal to (>/=)12 grams per deciliter (g/dL) at any time, epoetin beta will be discontinued until hemoglobin levels are less than or equal to (</=) 10 g/dL; if the hemoglobin level increases less than (<) 1 g/dL from screening level and hemoglobin level ˂12 g/dL, a 60,000 IU per week epoetin beta will be administered SC until Week 12; if the hemoglobin level increases >/=1 g/dL from screening level and hemoglobin level ˂12 g/dL, a 30,000 IU per week epoetin beta will be continued until Week 12.
干预措施: Epoetin beta (Drug)
结局指标
主要结局
Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria
时间窗: Week 12
Erythroid response at Week 12 according to IWG 2006 criteria was defined as a hemoglobin (Hb) increase of \>/= 1.5 grams/deciliter (g/dL), and a reduction of units of red blood cell (RBC) transfusions by at least 4 transfusions/8 weeks compared with the pre-treatment transfusion number in the previous 8 weeks. Only RBC transfusions given for an Hb of \</= 9.0 g/dL pre-treatment were counted in the RBC transfusion response evaluation.
次要结局
- Percentage of Participants With Neutrophil Response (in Participants With Pre-Treatment Neutrophil <1.0*10^9 Per Liter) at Week 12 as Assessed by IWG 2006 Response Criteria(Week 12)
- Percentage of Participants With Adverse Events(From signing of informed consent up to 4 weeks after last dose (up to 18 weeks))
- Percentage of Participants With Platelet Response (in Participants With Pre-Treatment Platelets <100*10^9 Per Liter) at Week 12 as Assessed by IWG 2006 Response Criteria(Week 12)