Efgartigimod for Consolidation Therapy in gMG

Официальное название

Multicenter Cohort Study on Efgartigimod for Consolidation Therapy After the Acute Exacerbation of Generalized Myasthenia Gravis in Adults

Обзор

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.

Первичные конечные точки

• 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. [Срок оценки: Consolidation baseline up to Month 6]

Вторичные конечные точки (8)

• Proportion of patients achieving both minimal symptom expression (MSE) and a daily corticosteroid dose ≤5 mg at Month 12. [Срок оценки: Consolidation baseline up to Month 12]
• Proportion of patients with MGFA Post-Intervention Status (MGFA-PIS) at Month 6 and Month 12 [Срок оценки: Consolidation baseline up to Month 6 AND Month 12]
• Change from baseline in MG-ADL scores at Month 6 and Month 12 [Срок оценки: Consolidation baseline up to Month 6 AND Month 12]
• Change from baseline in QMG scores at Month 6 and Month 12 [Срок оценки: Consolidation baseline up to Month 6 AND Month 12]
• Proportion of patients achieving minimal symptom expression (MSE) at Month 6 [Срок оценки: Consolidation baseline up to Month 6]
• Proportion of patients achieving minimal symptom expression (MSE) with daily corticosteroid dose ≤5 mg at Month 6 [Срок оценки: Consolidation baseline up to Month 6]
• Incidence of acute exacerbations, impending myasthenic crisis, or myasthenic crisis events during the study period [Срок оценки: Consolidation baseline up to Month 12]
• Types and frequencies of adverse events (AEs) and serious adverse events (SAEs) during the study period [Срок оценки: Consolidation baseline up to Month 12]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07079020 · LYS[2025]190-002

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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