BMJ neurology open · 2024
Batoclimab as induction and maintenance therapy in patients with myasthenia gravis: rationale and study design of a phase 3 clinical trial.
СтатьяНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Benatar Michael, Wiendl Heinz, Nowak Richard, Zheng Yan, Macias William
Аннотация
INTRODUCTION: Batoclimab, a fully human monoclonal antibody that inhibits the neonatal fragment crystallisable receptor, has shown promising phase 2 clinical trial results in patients with generalised myasthenia gravis (gMG).
METHODS AND ANALYSIS: In this phase 3, randomised, quadruple-blind, placebo-controlled study, adults with gMG will be randomised 1:1:1 to induction therapy with batoclimab 680 mg, batoclimab 340 mg, or placebo, administered once weekly (QW) for 12 weeks as a subcutaneous injection. The primary endpoint is the change from baseline to week 12 on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Batoclimab-treated patients achieving a ≥2-point improvement from baseline on MG-ADL at week 10 or week 12 will be re-randomised to maintenance treatment with batoclimab 340 mg QW, batoclimab 340 mg every other week (Q2W), or placebo for 12 weeks; batoclimab-treated patients with a <2-point improvement at week 10 and week 12 will be switched to placebo for the maintenance period and discontinued thereafter. Placebo-treated patients from the induction period will be re-randomised to batoclimab 340 mg QW or Q2W in the maintenance period. All patients who complete the maintenance period and achieve a ≥2-point improvement from baseline in MG-ADL during ≥1 of the final 2 visits of the induction and/or maintenance periods will continue their current batoclimab dose (or switch to batoclimab 340 mg QW for those on placebo) for a 52-week long-term extension (LTE-1). Patients who complete LTE-1 may enter a second, optional 52-week LTE (LTE-2).
ETHICS AND DISSEMINATION: This trial is being conducted in accordance with the International Council for Harmonisation Guideline for Good Clinical Practice, the Declaration of Helsinki, and each site's Institutional Review Board/Independent Ethics Committee. All patients must provide written informed consent. Results from this study will be published in peer-reviewed journals and presented at national and global conferences.
TRIAL REGISTRATION NUMBER: NCT05403541.
METHODS AND ANALYSIS: In this phase 3, randomised, quadruple-blind, placebo-controlled study, adults with gMG will be randomised 1:1:1 to induction therapy with batoclimab 680 mg, batoclimab 340 mg, or placebo, administered once weekly (QW) for 12 weeks as a subcutaneous injection. The primary endpoint is the change from baseline to week 12 on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Batoclimab-treated patients achieving a ≥2-point improvement from baseline on MG-ADL at week 10 or week 12 will be re-randomised to maintenance treatment with batoclimab 340 mg QW, batoclimab 340 mg every other week (Q2W), or placebo for 12 weeks; batoclimab-treated patients with a <2-point improvement at week 10 and week 12 will be switched to placebo for the maintenance period and discontinued thereafter. Placebo-treated patients from the induction period will be re-randomised to batoclimab 340 mg QW or Q2W in the maintenance period. All patients who complete the maintenance period and achieve a ≥2-point improvement from baseline in MG-ADL during ≥1 of the final 2 visits of the induction and/or maintenance periods will continue their current batoclimab dose (or switch to batoclimab 340 mg QW for those on placebo) for a 52-week long-term extension (LTE-1). Patients who complete LTE-1 may enter a second, optional 52-week LTE (LTE-2).
ETHICS AND DISSEMINATION: This trial is being conducted in accordance with the International Council for Harmonisation Guideline for Good Clinical Practice, the Declaration of Helsinki, and each site's Institutional Review Board/Independent Ethics Committee. All patients must provide written informed consent. Results from this study will be published in peer-reviewed journals and presented at national and global conferences.
TRIAL REGISTRATION NUMBER: NCT05403541.