Safety and Efficacy of Blinatumomab in Refractory Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 10
- 主要终点
- Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale
研究概览
简要总结
The goal of this clinical trial is to evaluating the efficacy and safety of Blinatumomab in the treatment of refractory myasthenia gravis and exploring the immunological mechanisms of Blinatumomab therapy in refractory myasthenia gravis. The main questions it aims to answer are:
Will Blinatumomab improve the symptoms of participants? What medical problems do participants have when using Blinatumomab?
Participants will:
Continuous intravenous infusion of Blinatumomab for 5 days, pause for one week, and continue continuous intravenous infusion for 5 days Visit the clinic once every 1 month for checkups and tests Keep a diary of their symptoms
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •18 years old ≤ Age ≤ 75 years old
- •patients with a Myasthenia Gravis Activities of Daily Living (MG-ADL) scale score of 6 or higher, and an ocular muscle score constituting less than 50% of the total score
- •after receiving an adequate course of at least two conventional immunotherapeutic drugs (including both steroidal and non-steroidal immunosuppressants), the Post-Intervention Status (PIS) remains unchanged or worsens; or after receiving an adequate course of at least two conventional immunotherapeutic drugs, the PIS improves, but the MG-ADL score is still ≥6 points and persists for at least half a year; or after receiving an adequate course of at least two conventional immunotherapeutic drugs, the PIS is in remission or improvement, yet during the regular tapering of immunotherapeutic drugs, there are still at least two annual exacerbations of disease symptoms (MG-ADL score ≥6 points); or after the occurrence of a crisis, despite multiple immunotherapies such as intravenous immunoglobulin, plasmapheresis, and high-dose intravenous methylprednisolone, and active infection control, the patient still cannot be weaned off the ventilator due to MG-induced respiratory muscle weakness for more than 14 days
- •positive for anti-AChR, and/or anti-MuSK, and/or anti-LRP4 antibodies.
- •patient or their legal representative signs an informed consent form in writing
排除标准
- •pregnant or lactating women
- •individuals with an allergy to Blinatumomab
- •individuals who have undergone thymectomy within the 12 months prior to baseline or who are planning to have a thymectomy during the 12-week study period
- •individuals who have received biologic agents targeting CD19 or CD20 within 6 months prior to baseline are eligible for enrollment (subjects with CD19 or CD20 positive B cell counts above the lower limit of normal are allowed to participate)
- •within the 3 months prior to baseline, use of Ecuzumab and Tocilizumab
- •received intravenous immunoglobulin or plasma exchange therapy within 4 weeks prior to baseline
- •currently participating in other clinical studies
研究组 & 干预措施
Treatment group
干预措施: Blinatumomab (Drug)
结局指标
主要结局
Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale
时间窗: At the 12th week
The minimum value is 0, and maximum value is 24. higher scores mean a worse outcome. The primary endpoint is the proportion of patients who achieved a reduction of at least 2 points in the MG-ADL score by week 12 and did not undergo rescue therapy.
次要结局
未报告次要终点
研究者
Zhongming Qiu
Professor
Xinqiao Hospital of Chongqing