NCT05567458进行中（未招募）2 期
A Phase 2, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Luspatercept (ACE-536) in Chinese Adult Subjects Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia
Bristol-Myers Squibb10 个研究点 分布在 1 个国家目标入组 94 人开始时间: 2022年10月17日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 94
- 试验地点
- 10
- 主要终点
- Proportion of participants with ≥ 33% reduction from baseline in red blood cell (RBC) transfusion burden over any consecutive 24 weeks
研究概览
简要总结
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of luspatercept plus best supportive care (BSC) versus placebo plus BSC in participants who require regular red blood cell transfusions due to β-thalassemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
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 is willing and able to adhere to the study visit schedule (for example, not scheduled to receive hematopoietic stem cell transplantation [HSCT]) and other protocol requirements.
- •Participant has documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia (β-thalassemia with mutation and/or multiplication of alpha (α) globin is allowed).
- •Participant is regularly transfused, defined as: 6-25 RBC units in the 24 weeks prior to randomization and no transfusion-free period for >42 days during that period.
- •Participant has Eastern Cooperative Oncology Group (ECOG) score of 0 or 1.
排除标准
- •Participant has a diagnosis of Hemoglobin S/β-thalassemia or α-thalassemia (for example, Hemoglobin H).
- •Participant has active hepatitis C virus (HCV) infection as demonstrated by a positive HCVribonucleic acid (RNA) test of sufficient sensitivity, or active infectious hepatitis B virus (HBV) as demonstrated by the presence of hepatitis B surface antigen (HBsAg) and/or HBVdeoxyribonucleic acid (DNA) positive, or known positive human immunodeficiency virus (HIV).
- •Participant has a history of deep venous thrombosis or stroke or thromboembolic events (venous or arterial) requiring medical intervention ≤24 weeks prior to randomization.
- •Participant uses chronic anticoagulant therapy, unless the treatment stopped at least 28 days prior to randomization. Anticoagulant therapies used for prophylaxis for surgery or high-risk procedures as well as low-molecular-weight heparin for superficial venous thrombosis and chronic aspirin are allowed.
- •Participant who has EMH complications requiring treatment to control the growth of EMH mass(es) during the screening period.
- •Participant used immunomodulatory imide drugs (IMiDs) ≤ 24 weeks prior to randomization
研究组 & 干预措施
Luspatercept
Experimental
干预措施: Luspatercept (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Proportion of participants with ≥ 33% reduction from baseline in red blood cell (RBC) transfusion burden over any consecutive 24 weeks
时间窗: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 - Week 48
次要结局
- Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 1-24(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 24)
- Proportion of subjects with ≥ 33% reduction from baseline in RBC transfusion burden during any rolling 24-week interval compared to the 24-week interval prior to start of IP for luspatercept plus BSC versus placebo plus BSC.(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 134)
- Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 134)
- Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 134)
- Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 37-48(24 weeks prior to Dose 1 Day 1 (inclusive); Week 37 to Week 48)
- Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 25-48(24 weeks prior to Dose 1 Day 1 (inclusive); Week 25 to Week 48)
- Frequency of Antidrug antibodies (ADA)(Up to 2 years)
- Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over any consecutive 24 weeks(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 134)
- Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 13-24(24 weeks prior to Dose 1 Day 1 (inclusive); Week 13-Week 24)
- Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 1-24(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 24)
- Change from baseline in Liver Iron Concentration (LIC) (mg/g dw) by magnetic resonance imaging (MRI)(Up to 96 weeks)
- Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 13-24(24 weeks prior to Dose 1 Day 1 (inclusive); Week 13-Week 24)
- Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by TranQoL(Up to 48 weeks)
- Proportion of participants who are transfusion independent for any consecutive ≥8 weeks during treatment(Week 1 to Week 134)
- Duration of RBC transfusion independence (TI)(Week 1 to Week 134)
- Change in spleen volume(Up to 96 weeks)
- Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 37-48(24 weeks prior to Dose 1 Day 1 (inclusive); Week 37 to Week 48)
- Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 25-48(24 weeks prior to Dose 1 Day 1 (inclusive); Week 25 to Week 48)
- Change from baseline in total RBC units transfused over Weeks 25-48(24 weeks prior to Dose 1 Day 1 (inclusive); Week 25 to Week 48)
- Change from baseline in myocardial iron by T2-star (T2*) MRI(Up to 96 weeks)
- Change from baseline in mean daily dose of iron chelation therapy (ICT)(12 weeks prior to Dose 1 Day 1 (inclusive); Week 37 to Week 48)
- Number of participants with Adverse Events (AEs)(Up to 4 years)
- Best change from baseline in total RBC units transfused in 24 weeks within the first 48-week treatment period(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 48)
- Change from baseline in total RBC units transfused over Weeks 1-24(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 24)
- Mean change from baseline in serum ferritin(12 weeks prior to Dose 1 Day 1 (inclusive); Week 37 to Week 48)
- Change from baseline in self-reported HRQoL assessed by SF-36(Up to 48 weeks)
- Proportion of participants who are transfusion independent for any consecutive ≥12 weeks during treatment(Week 1 to Week 134)
- Duration of reduction in transfusion burden(Week 1 to Week 134)
- Maximum plasma concentration (Cmax)(Up to 2 years)
- Area under the curve (AUC)(Up to 2 years)
- Proportion of subjects, without increase in transfusion burden and with an increase of ≥ 1.0 g/dL in pre-transfusion Hb level on at least 2 separate tests (at least 60 days apart) during any rolling 24-week interval, compared to baseline(24 weeks prior to Dose 1 Day 1 (inclusive); Dose 1 Day 2 through completion of 48-week treatment for last subject)
- Time to response(Week 1 to Week 134)
- Least number of transfusion events in 24 weeks within the first 48-week treatment period(24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 48)
研究者
研究点 (10)
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