NCT07752121尚未招募不适用
A Prospective, Multicenter, Non-Interventional Study Assessing First-line Luspatercept in Anemic Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Neoplasms Requiring Red Blood Cell Transfusions
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 190
- 试验地点
- 1
- 主要终点
- Percentage of participants achieving red blood cell transfusion independence (RBC-TI) for at least 8 consecutive weeks
研究概览
简要总结
This study will observe adults with lower-risk myelodysplastic neoplasms (MDS) who have anemia requiring regular red blood cell transfusions and who are prescribed first-line luspatercept as part of routine medical care. The study will follow participants for up to 2 years to understand how often treatment leads to periods without transfusions, changes in hemoglobin levels, health-related quality of life, and safety outcomes. Information on treatment use and outcomes in routine clinical practice in Germany will also be collected.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Males and females ≥18 years of age at enrollment.
- •Documented diagnosis of myelodysplastic neoplasms according to World Health Organisation (WHO) 2022 or WHO 2016 classification meeting International Prognostic Scoring System-Revised (IPSS-R) criteria for very low-, low-, or intermediate-risk disease.
- •Documented red blood cell transfusion dependence of ≥2 units of red blood cells within the 8 weeks preceding Day 1 treatment initiation.
- •First-line treatment based on the approved luspatercept label and decision for treatment with luspatercept as assessed by the treating physician prior to study participation
- •Provision of written informed consent.
排除标准
- •Contraindication according to the Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC).
- •Parallel participation in an interventional clinical trial (except follow-up phase as specified in protocol).
- •Patients who have completed their participation in an interventional clinical trial or who are not receiving any study drug anymore and who are only in the follow-up phase can be enrolled. For blinded studies, the study drug administered needs to be known at the time of enrolment.
- •Concurrent malignancy requiring treatment.
研究组 & 干预措施
First-Line Luspatercept
Adult participants with red blood cell transfusion-dependent lower-risk myelodysplastic neoplasms receiving first-line luspatercept in routine clinical practice in Germany.
干预措施: Luspatercept (Drug)
结局指标
主要结局
Percentage of participants achieving red blood cell transfusion independence (RBC-TI) for at least 8 consecutive weeks
时间窗: Up to Week 24
次要结局
- Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 112 consecutive days.(Up to 2-years)
- Percentage of participants achieving red blood cell transfusion independence for at least 12 consecutive weeks(Up to Week 48)
- Percentage of participants achieving red blood cell transfusion independence for at least 16 consecutive weeks(Up to Week 48)
- Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 84 consecutive days.(Up to 2-years)
- Mean change from baseline in hemoglobin concentration(Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years))
- Percentage of participants with hemoglobin increase of at least 1.5 g/dl from baseline(Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years))
- Percentage of participants achieving >50% reduction in transfusion burden compared with baseline(Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years))
- Time from first luspatercept administration to first on-treatment red blood cell transfusion.(Up to 2-years)
- Time to red blood cell transfusion independence for at least 8 consecutive weeks(Up to Week 24)
- Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 56 consecutive days.(Up to 2-years)
- Percentage of participants achieving hematologic improvement-erythroid response according to International Working Group (IWG) 2006 criteria(Up to Week 48)
- Number of participants with adverse events(Up to 2-years)
- Change from baseline in health-related quality of life assessed by European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30)(Day 1, Week 6, Week 12, Week 18, Week 24, Week 36, Week 48, and End of Treatment (up to 2-years))
- Change from baseline in health-related quality of life assessed by Quality of Life in Myelodysplasia Scale (QUALMS)(Day 1, Week 6, Week 12, Week 18, Week 24, Week 36, Week 48, and End of Treatment (up to 2-years))
研究者
研究点 (1)
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