A Phase 3, Prospective, Multicenter, Double Blind, Randomized, Placebo Controlled Study to Evaluate the Efficacy and Safety of Eculizumab in Patients With Guillain-Barré Syndrome (GBS)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 57
- 试验地点
- 1
- 主要终点
- Time to First Reaching a Hughes Functional Grade (FG) Score <=1
研究概览
简要总结
This is a Phase 3, prospective, multicenter, placebo controlled, double blind, randomized study to investigate the efficacy and safety of eculizumab in participants with severe GBS, defined using the Hughes Functional Grade (FG) scale as progressively deteriorating FG3 or FG4/FG5 within 2 weeks from onset of weakness due to GBS.
This study will be conducted only at sites in Japan.
详细描述
Eligible participants will be randomized to receive intravenous (IV) infusion of eculizumab or placebo at a 2:1 ratio. All participants will be on concomitant IV immunoglobulin G (Ig) therapy as per standard of care.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants who meet the GBS criteria.
- •Participants who were able to run prior to onset of GBS symptoms.
- •Participants with onset of weakness due to GBS < 2 weeks before screening.
- •Participants unable to walk unaided for ≥ 5 meters (progressively deteriorating FG3 or FG4 to FG5).
- •Participants who are already on IVIg or deemed eligible for and who will start IVIg.
- •Participants who can start their first dose of study drug before the end of the IVIg treatment period.
排除标准
- •Participants who have previously received or are currently receiving treatment with complement modulators.
- •Participants who have been administered another investigational product within 30 days or 5 half-lives (whichever is longer) prior to providing consent or are currently participating in another interventional study.
- •Participants who have received rituximab within 12 weeks prior to screening.
- •Participants who are being considered for or are already on plasmapheresis.
- •Participants who have received immunosuppressive treatment during the 4 weeks prior to providing consent.
研究组 & 干预措施
Eculizumab
Participants will receive eculizumab.
干预措施: Eculizumab (Biological)
Placebo
Participants will receive placebo.
干预措施: Placebo (Drug)
结局指标
主要结局
Time to First Reaching a Hughes Functional Grade (FG) Score <=1
时间窗: Up to Week 24
The mobility of the participants was evaluated on a 7 point disability functional grade scale and described as Hughes FG score of 0 (Healthy, no signs or symptoms of Guillain-Barré syndrome); 1 (Minor signs or symptoms and able to run); 2 (Able to walk 5 metre (m) across an open space without assistance); 3 (Able to walk 5 m across an open space with the help of one person and waist-level walking-frame, stick, or sticks); 4 (Chairbound/bedbound: unable to walk as in 3); 5 (Requiring assisted ventilation \[for at least part of day or night\]) and 6 (Dead), where higher numbers indicate more severe impairment. The Kaplan-Meier estimate of time to event of FG\<=1 is reported. Time (days) to first event=Date of first event-Date of first dose+1. Participants who discontinued early without achieving FG \<= 1 were censored at the date of discontinuation. Participants who completed the study without achieving FG\<=1 were censored at the date of study completion.
次要结局
- Number of Participants With A Hughes Functional Grade (FG) Score <=1(Week 8, Week 24)
- Free Complement Component 5 in Serum(Week 24)
- Number of Participants Who Required Mechanical Ventilator Support(Up to Week 24)
- Number of Participants With A Hughes Functional Grade Score Improvement of >=3(Week 24)
- Hemolytic Complement Activity in Serum(Week 24)
- Length of Stay in the Hospital(Up to Week 24)
- Concentration of Eculizumab in Serum(Up to Week 24)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(Day 1 up to Week 24)
- Number of Participants With Positive Antidrug Antibodies(Up to Week 12)