NCT07499323尚未招募不适用
A Preliminary Case Study to Evaluate the Efficacy and Safety of Talquetamab in Patients With Refractory Generalized Myasthenia Gravis
First Affiliated Hospital of Chongqing Medical University0 个研究点目标入组 2 人开始时间: 2026年3月20日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 2
- 主要终点
- MG-ADL score
研究概览
简要总结
Myasthenia Gravis (MG) is a chronic autoimmune disease mediated by pathogenic antibodies. Approximately 10%-15% of patients present with refractory status, defined as having an inadequate response to existing therapies or an inability to tolerate the side effects of the medication, highlighting an urgent need for the development of more targeted innovative therapies. Talquetamab is a bispecific antibody that targets G Protein-Coupled Receptor Class C Group 5 Member D (GPRC5D) and the Cluster of Differentiation 3 (CD3) molecule on the surface of T cells, thereby inducing T cells to precisely eliminate GPRC5D-positive cells. This study will conduct an exploratory case series to investigate the efficacy and safety of Talquetamab in refractory MG.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient must sign the informed consent form for participation in the study and agree to comply with the study procedures.
- •Age 18-70 years.
- •Must meet the clinical diagnostic criteria for MG, defined as presenting with a typical pattern of weakness (predominantly affecting proximal muscles, fatiguability, fluctuating severity, worse with prolonged effort and better after rest) and meeting any one of the following three conditions: documented history of abnormal neuromuscular transmission confirmed by single-fiber electromyography or repetitive nerve stimulation; documented history of a positive neostigmine test; or improvement in MG signs following oral administration of an acetylcholinesterase inhibitor. Additionally, the Myasthenia Gravis Foundation of America (MGFA) clinical classification must be II-IV, and the patient must be positive for anti-AChR antibodies.
- •Must meet the definition of "refractory myasthenia gravis" according to the Chinese Guidelines for the Diagnosis and Treatment of Myasthenia Gravis (2025 Edition): defined as having a suboptimal response to conventional immunotherapeutic drugs, intolerance to or having contraindications for adverse drug reactions, or experiencing frequent disease relapses requiring regular rescue therapy, making it difficult to achieve treatment goals.
- •Myasthenia Gravis Activities of Daily Living (MG-ADL, Appendix 1) score ≥6, with >50% of the score derived from non-ocular items.
- •Quantitative Myasthenia Gravis (QMG) score (Appendix 2) ≥8, with >50% of the score derived from non-ocular items.
- •The dosage of pyridostigmine bromide must be stable for at least 1 week prior to the treatment period.
排除标准
- •Patients who have had their glucocorticoid dose adjusted, or who have received immunoglobulins, plasma exchange, neonatal Fc receptor inhibitors, or complement inhibitors within ≤4 weeks.
- •Patients who have undergone thymectomy, received B-cell depletion therapy, or had their dose of non-steroidal immunosuppressants (such as azathioprine, tacrolimus, mycophenolate mofetil, cyclophosphamide, etc.) adjusted within ≤3 months.
- •Patients who have participated in any interventional clinical trial or received investigational treatment within ≤3 months, or within 5 half-lives of the received investigational drug (whichever is longer).
- •Patients with concurrent severe liver, kidney, cardiac, pulmonary, or coagulation dysfunction.
- •Patients with concurrent chronic infections such as Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus, or Mycobacterium tuberculosis.
结局指标
主要结局
MG-ADL score
时间窗: baseline, and 1-6 months
次要结局
- QMG score(baseline and 1-6 months)
研究者
Fei Xiao
Professor
First Affiliated Hospital of Chongqing Medical University
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