A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 125
- 试验地点
- 114
- 主要终点
- Myasthenia Gravis Activities of Daily Living Profile (MG-ADL): Change From Baseline in MG-ADL Total Score at Week 26 by Worst-Rank Analysis of Covariance (ANCOVA)
研究概览
简要总结
The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients ≥18 years old
- •Diagnosis of MG made by the following tests:
- •Positive serologic test for anti-AChR Abs as confirmed at screening, and
- •One of the following:
- •History of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography (SFEMG) or repetitive nerve stimulation, or
- •History of positive anticholinesterase test, e.g. edrophonium chloride test, or
- •Subject has demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician.
- •MGFA Clinical Classification Class II to IV at screening.
- •MG-ADL total score must be ≥6 at screening and Randomization (Day 1).
- •Subjects who have:
- •Failed treatment with at least two immunosuppressive agents. Or,
- •Failed treatment with at least one immunosuppressive agent and require chronic plasma exchange or IVIg
排除标准
- •History of thymoma or other neoplasms of the thymus
- •History of thymectomy within 12 months prior to screening
- •MGFA Class I or MG crisis at screening (MGFA Class V)
- •Use of rituximab within 6 months prior to screening
- •Use of IVIg or PE within 4 weeks prior to Randomization (Day 1)
研究组 & 干预措施
Eculizumab
Biological/Vaccine: Eculizumab; Induction phase: 3 vials of study drug (equivalent to 900 mg of eculizumab) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (equivalent to 1200 mg of eculizumab) 1 week later for the fifth dose (Week 4); Maintenance phase: 4 vials of study drug (equivalent to 1200 mg of eculizumab) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).
干预措施: Eculizumab (Biological)
Placebo
Placebo contains the same buffer components without the active ingredient; Induction phase: 3 vials of study drug (placebo) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (placebo) 1 week later for the fifth dose (Week 4); Maintenance phase: 4 vials of study drug (placebo) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).
干预措施: Placebo (Drug)
结局指标
主要结局
Myasthenia Gravis Activities of Daily Living Profile (MG-ADL): Change From Baseline in MG-ADL Total Score at Week 26 by Worst-Rank Analysis of Covariance (ANCOVA)
时间窗: End of study (Week 26)
In the Worst-Rank analysis, the 125 total patients were ranked from best outcome (rank/score of 1) to worst outcome (rank/score of 125).
次要结局
未报告次要终点