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
Comparative Efficacy of Nipocalimab and Efgartigimod in Participants With Generalized Myasthenia Gravis
The purpose of this study is to assess how well nipocalimab works when compared to efgartigimod in participants with generalized myasthenia gravis (a condition in which body's immune system mistakenly attacks and damages the connection between nerves and muscles causing muscle weakness).
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org/CoRDS to enroll.