EFG vs IVIG in TAMG

Официальное название

Perioperative Efficacy and Safety of Efgartigimod Versus Intravenous Immunoglobulin for Thymoma Associated Myasthenia Gravis: a Prospective, Multicenter, Randomized Controlled Study

Обзор

This study is a prospective, multi-center, randomized controlled study to evaluate the efficacy and safety of efgartigimod (EFG) and intravenous immunoglobulin (IVIG) in the perioperative application for acetylcholine receptor (AChR) antibody-positive thymoma patients with myasthenia gravis in order to provide reliable evidence for clinical decision-making.

Вмешательства

• Комбинированный продукт Efgartigimod + Thymectomy
  Patients receive weight-based EFG infusions (10 mg/kg; fixed 1200 mg for ≥120 kg) administered over 1 hour once weekly for 4 total doses. If a scheduled infusion is missed, EFG may be administered within 48 hours of the planned time, followed by resumption of the original schedule to complete all 4 infusions. Thymectomy is performed on the day following the second EFG infusion with a permitted ±3-day surgical window.
• Комбинированный продукт Intravenous immunglobulin + Thymectomy
  Patients receive weight-based IVIG infusions (400 mg/kg) administered daily for 5 consecutive days. Thymectomy is performed approximately 7 days after completing the final IVIG infusion, with a permitted ±3-day surgical window.

Первичные конечные точки

• Quantitative Myasthenia Gravis (QMG) reduction [Срок оценки: From baseline to 2 weeks postoperatively]

Вторичные конечные точки (12)

• Myasthenia Gravis Activities of Daily Living (MG-ADL) reduction [Срок оценки: From baseline to 2 weeks postoperatively]
• Myasthenia Gravis Composite (MGC) reduction [Срок оценки: From baseline to 2 weeks postoperatively]
• QMG change (1 day preoperatively) [Срок оценки: From baseline to 1 day preoperatively]
• QMG change (1 week postoperatively) [Срок оценки: From baseline to 1 week postoperatively]
• QMG change (1 month postoperatively) [Срок оценки: From baseline to 1 month postoperatively]
• QMG change (2 months postoperatively) [Срок оценки: From baseline to 2 months postoperatively]
• MG-ADL change (1 day preoperatively) [Срок оценки: From baseline to 1 day preoperatively]
• MG-ADL change (1 week postoperatively) [Срок оценки: From baseline to 1 week postoperatively]
• MG-ADL change (1 month postoperatively) [Срок оценки: From baseline to 1 month postoperatively]
• MG-ADL change (2 months postoperatively) [Срок оценки: From baseline to 2 months postoperatively]
• MGC change (1 day preoperatively) [Срок оценки: From baseline to 1 day preoperatively]
• MGC change (1 week postoperatively) [Срок оценки: From baseline to 1 week postoperatively]

Критерии участия

Критерии включения

• Patients aged >18 years and ≤75 years, with an expected survival time >12 months;
• Patients with generalized myasthenia gravis;
• MG-ADL ≥6;
• Positive for AChR antibody;
• Diagnosed with thymoma by enhanced chest CT or MRI;
• Patients with thymoma combined with myasthenia gravis who are diagnosed by MDT and need to undergo thymectomy;
• Patients with American Society of Anesthesiologists (ASA) grade 1-2;
• Able to understand the study situation and sign the Informed Consent.

Критерии исключения

• MGFA type V;
• Patients who have undergone median sternotomy;
• Confirmed history of congestive heart failure; poorly controlled angina pectoris with drug treatment; electrocardiogram (ECG) confirmed transmural myocardial infarction; poorly controlled hypertension; clinically significant valvular heart disease; or high-risk uncontrolled arrhythmia;
• Patients with weight loss of more than 5kg in the past month; severe uncontrolled systemic disease, such as active infection or poorly controlled diabetes; patients with hemorrhagic diseases and bleeding tendencies; coagulation dysfunction, bleeding tendency or receiving thrombolytic or anticoagulant therapy; patients with grade II-IV bone marrow suppression;
• Females with positive serum pregnancy test or in lactation period, and males and females of childbearing age who are unwilling to use adequate contraceptive measures during treatment;
• History of organ transplantation (including autologous bone marrow transplantation and peripheral stem cell transplantation);
• Patients with peripheral nervous system disorders or significant mental disorders and a history of central nervous system disorders;
• Patients participating in other clinical studies simultaneously;
• Patients who cannot tolerate single-lung ventilation during surgery; severe cardiac complications, cardiovascular decompensation, patients with implanted cardiac pacemakers;
• Patients in the acute inflammatory period due to bacterial, viral or other microbial infections; known active infections of human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV), or known HIV seropositivity;
• Patients who have undergone thoracic surgery due to tuberculous pleurisy, mesothelioma, lung disease, or diaphragmatic disease, with ipsilateral lung atelectasis involving one lobe or more than one lobe;
• Serum IgG level <4.5 g/L;
• Received biological agents such as rituximab or ibritumomab tiuxetan within 6 months; underwent plasma exchange within 1 month;
• Thrombosis, renal impairment or renal failure;
• Allergic to human immunoglobulin or have other severe allergic history;
• Selective IgA deficiency with anti-IgA antibodies;
• Subjects with impaired function of major organs, abnormal blood routine, lung, liver, kidney and cardiac function, and the following laboratory test results:

Blood: white blood cells <4.0×10\^9/L, absolute neutrophil count (ANC) <2.0×10\^9/L, platelet count <100×10\^9/L, hemoglobin <90g/L; Liver function: serum bilirubin >1.5 times the upper normal limit; ALT and AST >1.5 times the upper normal limit; Renal function: serum creatinine (SCr) >120 μmol/L, creatinine clearance rate (CCr) <60 ml/min;

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Shanghai Zhongshan Hospital — Шанхай

Идентификаторы

NCT: NCT07231523 · PROMISE-TAMG

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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