NCT07079020尚未招募不适用
Multicenter Cohort Study on Efgartigimod for Consolidation Therapy After the Acute Exacerbation of Generalized Myasthenia Gravis in Adults
Da, Yuwei, M.D.0 个研究点目标入组 130 人开始时间: 2025年7月30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
- 130
- 主要终点
- Proportion of patients achieving and maintaining minimal symptom expression (MSE) for at least 4 months after first attaining MSE status at any time during the consolidation therapy period within 2 months.
研究概览
简要总结
The goal of this observational study is to evaluate the efficacy and safety of consolidation therapy with corticosteroids and/or immunosuppressants combined with efgartigimod in patients with generalized myasthenia gravis after the acute exacerbation.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years, male or female.
- •Diagnosis of myasthenia gravis (MG) based on the following:
- •Typical clinical features of MG.
- •At least one of the following:
- •Seropositive for anti-AChR antibodies (confirmed by reliable assay). Abnormal neuromuscular transmission on electrophysiological testing (e.g., RNS or SFEMG).
- •Positive neostigmine test or documented response to cholinesterase inhibitors.
- •MGFA classification II-IV at consolidation baseline.
- •Seropositive for anti-AChR antibodies (required for enrollment).
- •Hospitalized for myasthenic crisis (MC) in the acute exacerbation and completed one treatment cycle of efgartigimod: acute exacerbation definition: Presence of MC warning signs (new or worsening within 2 weeks): Bulbar symptoms: Dysphagia, choking, weak cough, dysarthria. Respiratory symptoms: Dyspnea, respiratory muscle weakness. Generalized weakness: Head drop, jaw weakness, facial weakness. One treatment cycle of efgartigimod: 10 mg/kg IV, once weekly for 4 doses.
- •≥2-point improvement in MG-ADL score from acute exacerbation baseline by Day 4 of efgartigimod treatment.
- •Willing and able to provide written informed consent before study participation.
排除标准
- •Treatment with IVIg, plasma exchange (PE), IV methylprednisolone (IVMP), or immunoadsorption (IA) within 1 month before consolidation baseline.
- •Thymectomy within 6 months before exacerbation baseline.
- •Use of immunomodulatory monoclonal antibodies or investigational drugs (not listed above) within 3 months before baseline or 5 half-lives (whichever is longer).
- •Pregnancy or lactation (women of childbearing potential must have negative pregnancy test and use contraception).
- •Known hypersensitivity to efgartigimod or any FcRn-targeting therapy.
- •Clinically significant comorbidities, including severe cardiovascular, hepatic, renal, pulmonary, or hematologic disorders, active malignancy and uncontrolled systemic infections.
- •Other autoimmune diseases that may interfere with efficacy assessment (e.g., uncontrolled thyroid disease, severe rheumatoid arthritis).
- •Any other condition deemed unsuitable by the investigator.
结局指标
主要结局
Proportion of patients achieving and maintaining minimal symptom expression (MSE) for at least 4 months after first attaining MSE status at any time during the consolidation therapy period within 2 months.
时间窗: Consolidation baseline up to Month 6
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered achieving MSE if there was a 1 or 0 on the MG-ADL total score ≥4 consecutive months after first attaining MSE status at any time during the consolidation therapy period within 2 months.
次要结局
- Proportion of patients achieving both minimal symptom expression (MSE) and a daily corticosteroid dose ≤5 mg at Month 12.(Consolidation baseline up to Month 12)
- Proportion of patients with MGFA Post-Intervention Status (MGFA-PIS) at Month 6 and Month 12(Consolidation baseline up to Month 6 AND Month 12)
- Change from baseline in MG-ADL scores at Month 6 and Month 12(Consolidation baseline up to Month 6 AND Month 12)
- Change from baseline in QMG scores at Month 6 and Month 12(Consolidation baseline up to Month 6 AND Month 12)
- Proportion of patients achieving minimal symptom expression (MSE) at Month 6(Consolidation baseline up to Month 6)
- Proportion of patients achieving minimal symptom expression (MSE) with daily corticosteroid dose ≤5 mg at Month 6(Consolidation baseline up to Month 6)
- Incidence of acute exacerbations, impending myasthenic crisis, or myasthenic crisis events during the study period(Consolidation baseline up to Month 12)
- Types and frequencies of adverse events (AEs) and serious adverse events (SAEs) during the study period(Consolidation baseline up to Month 12)
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