A Phase 3, Multicenter ,Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Biossil Inc.
- 入组人数
- 345
- 试验地点
- 138
- 主要终点
- Time to Readiness for Discharge From Hospital
研究概览
简要总结
This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At least 6 years of age.
- •Documented diagnosis of sickle cell disease.
- •Diagnosis of vaso-occlusive crisis necessitating admission to the hospital with treatment including IV opioids.
- •Able to receive the first dose of study drug within 24 hours from the administration of IV opioids.
排除标准
- •Serious systemic infection
- •Acute Chest Syndrome
- •Serious concomitant medical problems (for example, stroke)
- •SCD pain atypical of VOC
- •Severe renal or hepatic impairment
- •Chronic pain rather than a presentation of acute VOC
研究组 & 干预措施
Rivipansel Treatment Arm
干预措施: Rivipansel (Drug)
Placebo Treatment Arm
干预措施: Placebo (Other)
结局指标
主要结局
Time to Readiness for Discharge From Hospital
时间窗: Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8)
Time to readiness-for-discharge from hospital was defined as the difference (in hours) between the time and date when all criteria for readiness-for-discharge were met and the start time and date of the first infusion (loading dose) of study drug. Criteria for readiness-for-discharge were met when all of the applicable 6 criteria (in relation to treatment of VOC and complications related to the VOC) were documented to have occurred. The six criteria were: 1) only oral pain medication was required, 2) acute complications related to the VOC (such as acute chest syndrome, stroke, priapism) had resolved to the extent that management could be in an outpatient setting, 3) IV opioids had been discontinued, 4) IV hydration had been discontinued, 5) IV antibiotics had been discontinued and 6) red blood cell (RBC) transfusion was no longer required for treatment of this VOC.
次要结局
- Time to Discharge From Hospital(Day 1 up to the latest day when the order of hospital discharge was issued by a qualified healthcare provider (up to an average of Day 8))
- Cumulative Intravenous (IV) Opioids Consumption From Time of Loading Dose of Study Drug to Discharge From Hospital(Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8))
- Time to Discontinuation of Intravenous (IV) Opioids(Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8))
- Cumulative Intravenous (IV) Opioids Consumption Within First 24 Hours Post-Loading Dose of Study Drug(24 hours post first IV infusion of loading dose of study drug on Day 1)
- Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 3 Days of Discharge From Hospital(Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8)