Efficacy and Safety of Low-dose Rituximab in the Treatment of Refractory Myasthenia Gravis
试验速览
- 阶段
- 3 期
- 发起方
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Time to reach MM-1, the proportion of drug remission.
研究概览
简要总结
This study is designed to evaluate the effectiveness and safety of low-dose rituximab in patients with refractory myasthenia gravis.
The traditional treatment of MG is immunosuppressive therapy, usually beginning with corticosteroids. However, up to 70% of treated patients show an incomplete response, including 10 - 30% who are unresponsive. Corticosteroids and other immunosuppressive therapies presented also many side effects. The investigators propose to evaluate in a pilot, open, prospective, single central study, the interest of rituximab (RTX) in the treatment of patients with refractory MG. Fifty patients with refractory MG will be included in the study and divided into two stages: 14 patients in the first stage were followed up after using RTX according to the study protocol. The study will move into the second stage on if the number of effective cases is greater than 3, otherwise, the study will be discontinued (based on Simon's Optimal Two-stage Design). The remaining 36 patients were enrolled in the second stage. In the first and second stages, the treatment plan and follow-up plan were consistent. The therapeutical schema is rituximab 100-200 mg/week for a total of 2-4 times, followed by adequate organ function, laboratory parameters and assessment of MG after each injection and end of follow up for 24 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 80 and under;
- •Myasthenia gravis:
- •Patients who are diagnosed as generalized or ocular myasthenia gravis
- •Patients must have disease refractory to treatment: the condition did not improve (QMG score decreased by less than 3 points after treatment) or even deteriorated after treatment with sufficient prednisone or other immunosuppressive agents.
- •Patients sign informed consent forms
排除标准
- •Over the age of 80
- •Patients with serious complications such as infection
- •Patients with active TB (during the screening period, a chest X-ray or chest CT would be performed unless the patient can provide chest X-ray or chest CT reports in the last month); or patients with active HBV, HBV DNA> 200
- •Patients suffering from cardiomyopathy, acute coronary events, or severe arrhythmia.
- •Patients who were allergic to rituximab
- •Pregnant or suckling period woman
- •Patients accompanied with mental disorders and have difficult to communication
- •Patients with a significant abnormality in white blood cells, hemoglobin, and platelet count.
研究组 & 干预措施
Rituximab Treated
rituximab
干预措施: Rituximab (Drug)
结局指标
主要结局
Time to reach MM-1, the proportion of drug remission.
时间窗: 27 weeks
Mild clinical Manifestations（MM-1）was the primary efficacy endpoint observed. Record the time the patient appears from the time of medication to the time of MM-1，and calculate the percentage of Pharmacologic Remission (PR). Criteria for PR: The patient has had no symptoms or signs of MG and continues to take some form of therapy for MG. There is no weakness of any muscle on careful examination by someone skilled in the evaluation of neuromuscular disease. Isolated weakness of eyelid closure is accepted. Patients taking cholinesterase inhibitors are excluded from this category because their use suggests the presence of weakness.
次要结局
- The changes of the disease severity(27 weeks)
研究者
Liu Weibin
Director of Neurology Department
First Affiliated Hospital, Sun Yat-Sen University