Inebilizumab for Myasthenia Gravis

This trial tests a medication called inebilizumab, an antibody treatment, to evaluate its effectiveness and safety for children with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. Participants will receive the treatment through an IV to monitor how the body processes the drug and to check for side effects. Children aged 2 to 17 with a confirmed gMG diagnosis, who frequently experience muscle weakness and are on stable medication, may be eligible. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

You may need to keep your current medications stable. If you're on corticosteroids or certain immunosuppressive therapies, your dose should not have increased recently. Acetylcholinesterase inhibitors should also be stable for at least 2 weeks before joining the study.

Research has shown that inebilizumab is generally well-tolerated by people with generalized myasthenia gravis (gMG). In a study with 238 participants, those who took inebilizumab demonstrated good safety results. Over 26 weeks, participants experienced improvements and manageable side effects.

Unlike the standard treatments for myasthenia gravis, which often include medications like anticholinesterase inhibitors and immunosuppressants, inebilizumab offers a fresh approach by targeting CD19, a protein on the surface of B cells. This mechanism helps reduce the number of B cells, which are believed to play a role in the autoimmune response that characterizes myasthenia gravis. Researchers are excited about inebilizumab because it is administered intravenously, which could potentially offer more controlled dosing and faster action compared to oral medications. This innovative approach might provide a more effective and efficient way to manage symptoms for patients.

Research has shown that inebilizumab, which participants in this trial will receive, effectively treats generalized myasthenia gravis (gMG). Studies found that patients taking inebilizumab experienced better muscle function and could perform daily activities more easily. For instance, one study revealed that 72.3% of patients taking the drug improved significantly in daily activities, compared to 45.2% of those not taking it. After 52 weeks, patients continued to benefit, especially those with specific antibodies (AChR+). This evidence suggests that inebilizumab could be a promising treatment option for people with gMG.¹⁴⁵⁶⁷