NCT06045689进行中（未招募）3 期
A Phase 3b, Open-label Study Evaluating the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) Initiated at Maximum Approved Dose in LR-MDS With IPSS-R Very Low-, Low-, or Intermediate-risk Who Require RBC Transfusions (MAXILUS)
Bristol-Myers Squibb52 个研究点 分布在 9 个国家目标入组 106 人开始时间: 2023年10月5日最近更新:
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 106
- 试验地点
- 52
- 主要终点
- Number of participants who achieve red blood cell transfusion independence (RBC-TI) for 8 weeks with a simultaneous mean hemoglobin (Hb) increase of ≥ 1 g/dL from Week 1 to Week 24
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of Luspatercept when administered at the maximum approved dose in low-risk Myelodysplastic Syndrome participants who require red blood cell transfusions.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 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
- •Participant had documented diagnosis of MDS according to World Health Organization (WHO) classification that met Revised International Prognostic Scoring System (IPSS-R) classification of very low-, low-, or intermediate-risk disease.
- •Participant has an Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or
- •Participant must have red blood cell transfusions according to study criteria.
排除标准
- •Participant has known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding.
- •Participant has had a prior allogeneic or autologous stem cell transplant.
- •Participant has known history or diagnosis of AML.
- •Participant has uncontrolled hypertension.
- •Other protocol-defined inclusion/exclusion criteria apply
研究组 & 干预措施
Cohort 1: erythropoiesis-stimulating agents (ESA) naïve
Experimental
干预措施: Luspatercept (Drug)
Cohort 2: ESA relapsed or refractory
Experimental
干预措施: Luspatercept (Drug)
结局指标
主要结局
Number of participants who achieve red blood cell transfusion independence (RBC-TI) for 8 weeks with a simultaneous mean hemoglobin (Hb) increase of ≥ 1 g/dL from Week 1 to Week 24
时间窗: Up to week 24
次要结局
- Number of participants who have a time from first dose to first onset of RBC-TI ≥ 8-, 12-, and 16-weeks from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT(Up to 2 years)
- Number of participants with an increase from baseline in Hb values of ≥ 1.0 g/dL over any consecutive 16-week period in absence of RBC transfusions from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT(Up to 2 years)
- Number of participants with a mean change in total RBC units transfused over a fixed 16-week period from Week 9 to Week 24 and from Week 33 to Week 48(Up to week 48)
- Number of participants with adverse events (AEs)(Up to 2 years)
- Number of participants with a change in subscale scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) from Week 1 to Week 48 and from baseline through EOT(Up to 2 years)
- Number of participants who achieve RBC-TI over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 to EOT(Up to 2 years)
- Number of participants who achieve RBC-TI over any consecutive 12-, 16-, and 24-week periods from Week 1 to Week 24, from Week 1 to Week 48 and from Week 1 through EOT(Up to 2 years)
- Number of participants with change in serum ferritin (SF) over a 16-week period from Week 9 to Week 24 and from Week 33 to Week 48(Up to week 48)
- Number of participants with a maximum duration of RBC-TI for participants who achieve RBC TI ≥ 8- and 16-week period from Week 1 to Week 24 and from Week 1 to EOT(Up to 2 years)
- Number of participants with an increase from baseline in mean hemoglobin (Hb) values of ≥ 1.0 g/dL over any consecutive 8-week period in absence of RBC transfusions from Week 1 to Week 48 and from Week 1 through EOT(Up to 2 years)
- Number of participants with an increase from baseline in Hb values of ≥ 1.5 g/dL over any consecutive 8-, and 16-week periods in absence of RBC transfusions from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT(Up to 2 years)
- Number of participants who achieve Hematological Improvement Erythroid (mHI-E) per International Working Group-2018 (IWG-2018) over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT(Up to 2 years)
- Number of participants who achieve Hematological Improvement - Platelets (HI-P) per IWG-2018 over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT(Up to 2 years)
- Number of participants with change in mean daily dose of iron chelation therapy (ICT) over a 16-week period from Week 9 to Week 24 and from Week 33 to Week 48(Up to week 48)
- Number of participants with acute myeloid leukemia (AML) progression(Up to 4 years)
- Time to AML progression(Up to 4 years)
- Time from treatment start date to death due to any cause(Up to 4 years)
- Number of participants who achieve Hematological Improvement - Neutrophils (HI-N) per IWG-2018 over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT(Up to 2 years)
研究者
研究点 (52)
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相关资讯
Luspatercept Demonstrates Durable Transfusion Independence in Lower-Risk MDS Across Two Pivotal Trials- In the phase 3b MAXILUS trial, 81.5% of ESA-naive patients with lower-risk MDS achieved RBC transfusion independence for ≥8 weeks plus concurrent Hb increase ≥1 g/dL.
- Long-term COMMANDS data show luspatercept nearly doubled the duration of transfusion independence compared with epoetin alfa, with some "super-responders" remaining transfusion-free for ≥2.5 years.
- MAXILUS results affirm the importance of early treatment initiation with luspatercept at the maximum approved dose in both ESA-naive and ESA-relapsed/refractory populations.
- Both trials reinforce luspatercept's role as a frontline standard in lower-risk MDS, raising expectations for durable transfusion independence.3 months agoMAXILUS Trial Investigates Higher Luspatercept Dose in Lower-Risk MDS- The phase 3b MAXILUS trial (NCT06045689) is evaluating the safety and efficacy of initiating treatment with luspatercept at the maximum approved dose in patients with lower-risk myelodysplastic syndromes (MDS).
- The study aims to determine if starting luspatercept at 1.75 mg/kg, instead of the standard 1 mg/kg, can improve outcomes for both ESA-naive and ESA-refractory/relapsed patients.
- MAXILUS is an open-label, nonrandomized study that will enroll approximately 100 adult patients with very low-, low-, or intermediate-risk MDS, with an estimated primary completion date of January 2026.last year