NCT00727194终止2 期
A Randomized, Double-Blind, Placebo-Controlled, Cross-Over, Multi-Center Study of Eculizumab in Patients With Generalized Myasthenia Gravis (gMG) Who Have Moderate to Severe Muscle Weakness Despite Treatment With Immunosuppressants
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 14
- 试验地点
- 25
- 主要终点
- Quantitative Myasthenia Gravis (QMG): The Primary Efficacy Endpoint in This Study Was the Percentage of Patients With a 3-point Reduction From Baseline in the QMG Total Score for Disease Severity.
研究概览
简要总结
The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of patients with generalized myasthenia gravis despite treatment with various immunosuppressants, such as prednisone, methotrexate, Cellcept, cyclosporine, and cyclophosphamide, that are currently available.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Generalized MG
- •MGFA Clinical Classification Class II, III or IVa.
- •QMG total score ≥12
- •Minimum score of two (2) in four (4) or more test items in the QMG
- •Able to give informed consent.
- •Have failed at least two immunosuppressants after one year of treatment
- •A positive serologic test for binding anti-acetylcholine receptor Abs at Screening and one of the following a) history of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation, or b) history of positive anticholinesterase test, eg, edrophonium chloride test, or c) patient has demonstrated improvement in MG signs on acetylcholinesterase inhibitors as assessed by treating physician.
排除标准
- •History of thymoma or other neoplasms of the thymus.
- •History of thymectomy within 12 months prior to screening.
- •Pregnancy or lactation
- •Current or chronic use of plasmapheresis/plasma exchange
- •IVIG treatment within 8 weeks prior to screening.
- •Use of etanercept within 2 months prior to screening.
- •Use of rituximab (RITUXAN®) within 6 months prior to screening.
- •MGFA Class I, IVb, and V
- •Crisis or impending crisis
研究组 & 干预措施
1
Experimental
eculizumab
干预措施: eculizumab (Drug)
2
Placebo Comparator
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Quantitative Myasthenia Gravis (QMG): The Primary Efficacy Endpoint in This Study Was the Percentage of Patients With a 3-point Reduction From Baseline in the QMG Total Score for Disease Severity.
时间窗: 16 weeks
The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The MGFA task force has recommended that the QMG score be used in prospective studies of therapy for MG.
次要结局
- Mean Change From Baseline in QMG Total Score(16 weeks)
- Change From Baseline to the End of Treatment (16 Weeks) in the Two Most Affected QMG Items for Disease Severity (Individual Test Item: Ptosis)(16 weeks)
- Change From Baseline in the MGFA Post-Intervention Status (PIS)(16 weeks)
- Change From Baseline in the QoL Instrument, SF-36.(16 weeks)
- Change From Baseline in Respiratory Function Tests to Characterize the Degree of Involvement of Respiratory Muscles.(16 weeks)
- Change From Baseline in the MG-Activity of Daily Living Profile (MG-ADL)(16 weeks)
- Change From Baseline to the End of Treatment (16 Weeks) in the Two Most Affected QMG Items for Disease Severity (Individual Test Item: Double Vision)(16 weeks)
研究者
研究点 (25)
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