What the trial was testing
The RAISE enrolled 174 patients with myasthenia gravis. The study was sponsored by UCB and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Roughly twice the improvement in daily activity scores compared with placebo at 12 weeks.
The Lancet Neurology · 2023 · NCT04115293
These findings — that point change in daily activity scores on zilucoplan vs. placebo at 12 weeks (a bigger drop is better) — were published in the The Lancet Neurology and represent the headline result of the study.
Researchers tracked outcomes across 174 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with myasthenia gravis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Zilucoplan (Zilbrysq) is FDA-approved for generalized myasthenia gravis in AChR-antibody-positive adults and available now. It is a daily injection patients give themselves at home. Vaccination against meningococcal disease is required before starting because of a small risk of serious infections. Ask a neuromuscular specialist whether you qualify.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open myasthenia gravis trials
A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab
This is a prospective, multicenter single arm study designed to evaluate the effectiveness and safety of a predefined oral corticosteroids (OCS) tapering schedule to reduce OCS use in adult participants with acetylcholine receptor positive (AChR+) generalized myasthenia gravis (gMG) being treated with intravenous ravulizumab.
Rituximab EfFicacy IN MyasthEnia Gravis (REFINE)
The primary objective of this phase III trial is to investigate if Rituximab can reduce patients' functional impairment caused by MG. The secondary objectives of this trial are to assess whether treatment with rituximab in patients with MG will: * Allow faster and greater corticosteroid tapering * Reduce the frequency of exacerbations * Improve quality of life * Offer an acceptable safety and tolerability profile.