A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of the Efficacy and Safety of Tocilizumab in the Treatment of Generalized Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 64
- 试验地点
- 6
- 主要终点
- Change in Quantitative Myasthenia Gravis (QMG) scores.
研究概览
简要总结
Randomized, double-blind, placebo-controlled, parallel-group study with optional open-label extension.
详细描述
This study is a randomized, double-blind, placebo-controlled study, to be conducted at 6 study sites. Approximately 64 subjects will be enrolled. Patients with MG who are positive for anti-AChR antibodies will be enrolled. Patients who do not have anti-AChR or anti-MuSK antibodies will not be enrolled. Patients with MGFA classification II, III, or IV disease, MG-ADL score ≥ 5, QMG score ≥ 11, and use of a corticosteroid and/or non-steroidal immunosuppressant will be included in the study.
All subjects who complete the randomized controlled period will have the option to enroll in a 1-year open-label period.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of MG with anti-AChRantibody.
- •MGFA Clinical Classification Class II, III, or IV.
- •MG-ADL score of 5 or greater at screening and at randomization with > 50% of this score attributed to non-ocular items.
- •QMG score of 11 or greater.
- •Subjects must be on:
- •Cholinesterase inhibitor, with no dose increase within 4 weeks prior to randomization;
- •Corticosteroids, with no dose increase within 4 weeks prior to randomization; or/and c. non-steroidal IST (including azathioprine, mycophenolate mofetil, tacrolimus, cyclosporine A), with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization.
排除标准
- •Participants had clinically relevant active infections (such as sepsis, pneumonia, or abscess) or severe infections (resulting in hospitalization or requiring antibiotic treatment) in the 4 weeks before randomization;
- •Those with a history of high-risk tuberculosis infection, acquired tuberculosis infection, and chronic hepatitis;
- •Human immunodeficiency virus (HIV) infection;
- •Thymomas that have received thymectomy or planned thymectomy during RCP within 6 months before randomization, or require chemotherapy and/or radiotherapy at any time;
- •Received rituximab treatment in the past 6 months before randomization;
- •Received tocilizumab or eculizumab treatment within 3 months before randomization;
- •Received IVIG or plasma exchange within 4 weeks before randomization;
- •Unresected thymoma.
- •History of other tumor diseases.
研究组 & 干预措施
Tocilizumab
Participants will receive tocilizumab administered intravenously (IV) on weeks 1,5,9 and 13 of the randomized controlled period.
干预措施: Tocilizumab Injectable Product (Drug)
Placebo
Participants will receive placebo administered intravenously (IV) on weeks 1,5,9 and 13 of the randomized controlled period.
干预措施: Tocilizumab Injectable Product (Drug)
结局指标
主要结局
Change in Quantitative Myasthenia Gravis (QMG) scores.
时间窗: 16 weeks
次要结局
- Change in Myasthenia Gravis Impairment Index (MGII) score.(16 weeks)
- Number of participants with treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and treatment-emergent serious adverse events (TESAEs) during the randomized controlled period and open-label period.(16 weeks)
- Proportion of subjects with both (1) ≥ 3-point improvement in QMG and (2) lasts ≥4 weeks(16 weeks)
- Proportion of subjects with both (1) ≥ 2-point improvement in MG-ADL and (2) lasts ≥4 weeks(16 weeks)
- Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Profile score.(16 weeks)
- Change in Myasthenia Gravis Composite (MGC) score(16 weeks)
- Change in Myasthenia Gravis Quality of Life-15, revised (MGQOL-15r) score.(16 weeks)