Efficacy and Safety of Tocilizumab in the Treatment of Generalized Myasthenia Gravis

Официальное название

A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of the Efficacy and Safety of Tocilizumab in the Treatment of Generalized Myasthenia Gravis

Обзор

Randomized, double-blind, placebo-controlled, parallel-group study with optional open-label extension.

Подробное описание

This study is a randomized, double-blind, placebo-controlled study, to be conducted at 6 study sites. Approximately 64 subjects will be enrolled. Patients with MG who are positive for anti-AChR antibodies will be enrolled. Patients who do not have anti-AChR or anti-MuSK antibodies will not be enrolled. Patients with MGFA classification II, III, or IV disease, MG-ADL score ≥ 5, QMG score ≥ 11, and use of a corticosteroid and/or non-steroidal immunosuppressant will be included in the study.

All subjects who complete the randomized controlled period will have the option to enroll in a 1-year open-label period.

Вмешательства

• Препарат Tocilizumab Injectable Product
  Participants will receive IV tocilizumab

Первичные конечные точки

• Change in Quantitative Myasthenia Gravis (QMG) scores. [Срок оценки: 16 weeks]

Вторичные конечные точки (7)

• Proportion of subjects with both (1) ≥ 3-point improvement in QMG and (2) lasts ≥4 weeks [Срок оценки: 16 weeks]
• Proportion of subjects with both (1) ≥ 2-point improvement in MG-ADL and (2) lasts ≥4 weeks [Срок оценки: 16 weeks]
• Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Profile score. [Срок оценки: 16 weeks]
• Change in Myasthenia Gravis Composite (MGC) score [Срок оценки: 16 weeks]
• Change in Myasthenia Gravis Quality of Life-15, revised (MGQOL-15r) score. [Срок оценки: 16 weeks]
• Change in Myasthenia Gravis Impairment Index (MGII) score. [Срок оценки: 16 weeks]
• Number of participants with treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and treatment-emergent serious adverse events (TESAEs) during the randomized controlled period and open-label period. [Срок оценки: 16 weeks]

Критерии участия

Критерии включения

• Diagnosis of MG with anti-AChRantibody.
• MGFA Clinical Classification Class II, III, or IV.
• MG-ADL score of 5 or greater at screening and at randomization with > 50% of this score attributed to non-ocular items.
• QMG score of 11 or greater.
• Subjects must be on:

• Cholinesterase inhibitor, with no dose increase within 4 weeks prior to randomization;
• Corticosteroids, with no dose increase within 4 weeks prior to randomization; or/and c. non-steroidal IST (including azathioprine, mycophenolate mofetil, tacrolimus, cyclosporine A), with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization.

Критерии исключения

• Participants had clinically relevant active infections (such as sepsis, pneumonia, or abscess) or severe infections (resulting in hospitalization or requiring antibiotic treatment) in the 4 weeks before randomization;
• Those with a history of high-risk tuberculosis infection, acquired tuberculosis infection, and chronic hepatitis;
• Human immunodeficiency virus (HIV) infection;
• Thymomas that have received thymectomy or planned thymectomy during RCP within 6 months before randomization, or require chemotherapy and/or radiotherapy at any time;
• Received rituximab treatment in the past 6 months before randomization;
• Received tocilizumab or eculizumab treatment within 3 months before randomization;
• Received IVIG or plasma exchange within 4 weeks before randomization;
• Unresected thymoma.
• History of other tumor diseases.

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

Четверное слепое

Основная цель

Лечение

Центры проведения

Китай · 6 центров

• Beijing Tiantan Hospital, Capital Medical University — Пекин
• Xiangya Hospital Central South University — Чанша
• Tangdu Hospital, The Fourth Military Medical University — Сиань
• Huashan Hospital — Шанхай
• West China Hospital of Sichuan University — Чэнду
• Tianjin medical university general hospital — Тяньцзинь

Идентификаторы

NCT: NCT05067348 · V4.0, 20220410

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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