What the trial was testing
The ADAPT enrolled 167 patients with myasthenia gravis. The study was sponsored by argenx 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
68% of antibody-positive patients responded — vs. 30% on placebo.
The Lancet Neurology · 2021 · NCT03669588
These findings — that of AChR-antibody-positive patients responded to efgartigimod vs. placebo — were published in the The Lancet Neurology and represent the headline result of the study.
Researchers tracked outcomes across 167 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
Efgartigimod (Vyvgart) is FDA-approved for generalized myasthenia gravis in adults who test positive for AChR antibodies and available now. It is given as weekly IV infusions in cycles, with weeks off between treatment runs. Most common side effects are headache and respiratory 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 of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis
The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.
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.