NCT04477850已完成2 期
A Phase 2, Multicenter, Single-Arm Bridging Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes(MDS) in Chinese and Japanese Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Celgene
- 入组人数
- 30
- 试验地点
- 40
- 主要终点
- Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of luspatercept (ACE-536) for the treatment of anemia due to Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate risk myelodysplastic syndromes (MDS) in Chinese and Japanese participants with ring sideroblasts who require Red Blood Cells (RBC) transfusions.
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
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Refractory or intolerant to, or ineligible for, prior Erythropoiesis stimulating agent (ESA) treatment as defined by any one of the following: Refractory to prior ESA treatment, Intolerant to prior ESA treatment, or ESA ineligible.
- •previously treated with an ESA or granulocyte colony-stimulating factor, granulocyte-macrophage colony-stimulating factor, both agents must have been discontinued ≥ 4 weeks prior to date of luspatercept treatment
- •Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2
排除标准
- •Prior therapy with disease modifying agents for underlying MDS disease
- •Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
- •Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≥ 3.0 x upper limit of normal (ULN)
- •Other protocol-defined inclusion/exclusion criteria apply
研究组 & 干预措施
Luspatercept Administration
Experimental
干预措施: Luspatercept (Drug)
结局指标
主要结局
Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks
时间窗: Week 1 through Week 24
次要结局
- Duration of RBC-TI(Week 1 through Week 24)
- Progression to acute myeloid leukemia (AML)(Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose)
- Incidence of frequency of AEs(Screening through 42 days post last dose)
- Reduction in Red Blood Cell (RBC) units transfused over 16 weeks compared to baseline(Week 9 through Week 24)
- Incidence of seriousness of AEs(Screening through 42 days post last dose)
- Mean decrease in serum ferritin compared to baseline(Week 9 through Week 24)
- Overall survival (OS)(Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose)
- Mean hemoglobin increase ≥ 1.0 g/dL(Week 1 through Week 24)
- Incidence of severity of AEs(Screening through 42 days post last dose)
- Frequency of Anti-drug antibodies (ADA)(Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose)
- RBC-TI ≥ 12 weeks(Week 1 through Week 24)
- Modified hematologic improvement - erythroid (mHI-E) per International Working Group (IWG)(Week 1 through Week 24)
- Mean decrease in iron chelation therapy (ICT) use compared to baseline(Week 9 through Week 24)
- Time to RBC-TI(Week 1 through Week 24)
- Incidence of relationship of AEs to study treatment(Screening through 42 days post last dose)
- Incidence of type of adverse events (AEs)(Screening through 42 days post last dose)
- Pharmacokinetics - Area under the curve (AUC)(Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose)
- Pharmacokinetics - Maximum plasma concentration of the drug (Cmax)(Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose)
研究者
研究点 (40)
Loading locations...
相似试验
进行中（未招募）
3 期
Assessment of Effectiveness and Safety of Luspatercept in Patients Suffering From Lower-risk Myelodysplastic Syndrome.Myelodysplastic SyndromesNCT05181592GWT-TUD GmbH70
进行中（未招募）
3 期
A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA NaïveMyelodysplastic SyndromesNCT03682536Celgene363
进行中（未招募）
3 期
A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome ParticipantsMyelodysplastic SyndromesNCT06045689Bristol-Myers Squibb106
进行中（未招募）
2 期
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.Beta-thalassemiaNCT05567458Bristol-Myers Squibb94
已完成
2 期
A Safety and Efficacy Study to Evaluate Luspatercept in Subjects With Myeloproliferative Neoplasm-associated Myelofibrosis Who Have Anemia With and Without Red Blood Cell-transfusion DependenceAnemiaPrimary MyelofibrosisNCT03194542Celgene95