A phase II, open-label, single arm study to evaluate the efficacy of Luspatercept in Erythropoiesis-stimulating agent naive lower-risk MDS patients with or without ring sideroblasts who do not require RBC transfusions (LENNON)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 24
- 主要终点
- The primary efficacy endpoint is erythroid response (HI-E) according to IWG 2018 criteria and defined as an increase in hemoglobin levels by at least 1.5 g/dL, persistent for at least 8 weeks over the baseline hemoglobin level (mean over 16 weeks prior to inclusion) and will be determined after 24 weeks of LUS treatment.
研究概览
简要总结
To evaluate the erythroid response (hematologic improvement erythroid, HI-E) rate of luspatercept (LUS) for the treatment of anemia due to very low-, low-, or intermediate-risk MDS in subjects who require no RBC transfusions.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of MDS according to WHO classification
- •Very low-, low-, or intermediate-risk disease with up to 3.5 score points according to revised International Prognostic Scoring System (IPSS-R) classification
- •Non-transfusion dependence (NTD) according to IWG 2018
- •Symptomatic anemia: mean baseline Hb < 10 g/dL
排除标准
- •Secondary MDS
- •Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
- •Prior allogeneic or autologous stem cell transplant
- •Prior ESA treatment
结局指标
主要结局
The primary efficacy endpoint is erythroid response (HI-E) according to IWG 2018 criteria and defined as an increase in hemoglobin levels by at least 1.5 g/dL, persistent for at least 8 weeks over the baseline hemoglobin level (mean over 16 weeks prior to inclusion) and will be determined after 24 weeks of LUS treatment.
The primary efficacy endpoint is erythroid response (HI-E) according to IWG 2018 criteria and defined as an increase in hemoglobin levels by at least 1.5 g/dL, persistent for at least 8 weeks over the baseline hemoglobin level (mean over 16 weeks prior to inclusion) and will be determined after 24 weeks of LUS treatment.
次要结局
- To evaluate HI-E response duration on a time horizon of 18 months after response
研究者
Susanne Melzer
Scientific
Universitaet Leipzig