A Phase 2 Study Evaluating the Safety and Effectiveness of Intravenous Tocilizumab Compared to Placebo for Adults with Generalized Myasthenia Gravis

What this Trial Is About

This research investigates the effects of tocilizumab in adults with generalized myasthenia gravis MG who test positive for anti-AChR antibodies. The study enrolls patients with moderate to severe MG classified as MGFA class II, III, or IV, who have significant symptoms measured by MG-ADL and QMG scores. The trial is designed as a randomized, double-blind, placebo-controlled study to assess the efficacy and safety of tocilizumab in this population. Participants are randomly assigned to receive either tocilizumab or a placebo, both given by intravenous infusion at weeks 1, 5, 9, and 13 during the controlled study period. After completing this phase, subjects may join a one-year open-label extension where all participants can receive tocilizumab. The study is conducted across multiple centers and includes approximately 64 participants. Throughout the trial, participants will undergo regular evaluations including muscle strength and daily living activity assessments such as the Quantitative Myasthenia Gravis QMG score and MG-ADL profile. Researchers will monitor changes in these scores and quality of life, as well as track any adverse events during both the controlled and open-label periods. The total study duration includes a 16-week controlled period followed by optional extended treatment and observation.

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