Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC): A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial of MST-188 (Purified Poloxamer 188) Injection in Subjects With Sickle Cell Disease Experiencing Vaso Occlusive Crisis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 388
- 试验地点
- 57
- 主要终点
- Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease.
研究概览
简要总结
The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether MST-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Additionally, this study will compare the development of acute chest syndrome during VOC in subjects who receive MST-188 to those who do not receive MST-188.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 4 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 4 through 65 years
- •Subject has a confirmed diagnosis of HbSS, HbSC, HbSβ+thal, or HbSβ0thal
- •Subject is experiencing acute pain typical of vaso-occlusive crisis requiring treatment with parenteral analgesia
- •Subject requires hospitalization
排除标准
- •Subject has acute chest syndrome
- •Subject's laboratory results indicate inadequate organ function
- •Subject is pregnant or nursing an infant
- •Subject had a painful crisis requiring hospitalization within the preceding 14 days or has experienced > 5 hospitalizations for VOC in the prior 6 months
- •Subject has been transfused within the past 14 days
- •Subject is hospitalized for a condition other than VOC
- •Subject has complications related to SCD
研究组 & 干预措施
MST-188
MST-188 injection administered as a continuous infusion 100 mg/kg for 1 hour followed by 30 mg/kg/hr for up to 48 hours.
干预措施: MST-188 (Drug)
Saline
Saline administered as a continuous infusion for up to 49 hours
干预措施: Saline (Drug)
结局指标
主要结局
Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease.
时间窗: Study participants will be followed for the duration of hospital stay, an expected average of 4 days
次要结局
- Re-hospitalization rate for VOC(Hospital discharge to 14 days post-discharge)
- Occurence of acute chest syndrome(Randomization to 120 hours after randomization)