[Translation] 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 β-thalassemia
主要目的:
确定在任何连续的24周间隔内(从治疗开始至末例受试者完成48周治疗),接受罗特西普 + BSC与安慰剂 + BSC治疗相比,输血负荷(单位RBC/次)相对于基线降低≥33%的受试者比例。
次要目的:
评价罗特西普+ BSC与安慰剂+ BSC对以下指标的影响:
- RBC输注负荷降低(在任何连续的12周间隔和规定的固定周期内,输血负荷相对于基线降低≥33%/≥50%的受试者比例;在任何连续的24周间隔内,输血负荷相对于基线降低≥50%的受试者比例)
- 输血负荷相对于基线的平均变化
- TI(不依赖输血≥8/≥12周的受试者比例)
- 输血负荷减少或TI的持续时间
- 至输血减少的时间
- 输血事件频率
- 铁参数(血清铁蛋白、肝脏铁浓度、心肌铁)和铁螯合治疗的使用
- HRQoL评估,包括TranQoL和SF-36
- 脾脏体积
在中国β-地中海贫血受试者中评价罗特西普与安慰剂相比的安全性
评价罗特西普在中国β-地中海贫血受试者中的免疫原性
评价罗特西普在中国β-地中海贫血受试者中的PK
探索性目的:
评价罗特西普对医疗资源利用的影响
[Translation] Primary objective:
To determine the proportion of subjects who achieved a ≥33% reduction in transfusion burden (RBC/transfusion) from baseline in any consecutive 24-week interval (from the start of treatment until the last subject completed 48 weeks of treatment) treated with Rotacept + BSC compared with placebo + BSC.
Secondary objectives:
To evaluate the effects of Rotecept + BSC vs. placebo + BSC on the following indicators:
- Reduction in RBC transfusion burden (proportion of subjects with a ≥33%/≥50% reduction in transfusion burden from baseline in any consecutive 12-week interval and defined fixed period; proportion of subjects with a ≥50% reduction in transfusion burden from baseline in any consecutive 24-week interval)
- Mean change in transfusion burden from baseline
- TI (proportion of subjects transfusion-independent for ≥8/≥12 weeks)
- Duration of transfusion burden reduction or TI
- Time to transfusion reduction
- Frequency of transfusion events
- Iron parameters (serum ferritin, liver iron concentration, myocardial iron) and use of iron chelation therapy
- HRQoL assessments, including TranQoL and SF-36
- Spleen volume
To evaluate the safety of Rotecept compared with placebo in Chinese subjects with β-thalassemia
To evaluate the immunogenicity of Rotecept in Chinese subjects with β-thalassemia
To evaluate the PK of Rotecept in Chinese subjects with β-thalassemia
Exploratory objective:
To evaluate the impact of Rotecept on the utilization of medical resources