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 Prospective Cohort Study of Myasthenia Gravis in China
The goal of this prospective cohort study is to investigate long-term therapeutic strategies for myasthenia gravis (MG) and identify potential biomarkers. The main questions it aims to answer are: 1. Whether low-dose oral steroids may lead to a reduction in the recurrence rate among patients with MG. 2. To identify potential biomarkers that can predict disease progression and prognosis. This study recruits well-controlled patients with MG. Based on patient preferences and considerations such as coexisting conditions (e.g., uncontrolled hypertension, diabetes, severe osteoporosis, obesity), the participants will be non-randomly divided into two groups: a maintenance steroid therapy group and a withdrawal group (withdraw all immunosuppresants). Subsequently, these groups of patients will undergo long-term follow-up assessments.
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA/RNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic/digitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.