Myasthenia gravis is an autoimmune disorder where the body attacks the connections between nerves and muscles, causing weakness that worsens with activity. It affects about 20 in 100,000 people. Current treatments include steroids, immunosuppressants, and medications that temporarily improve nerve-muscle communication. Some people have a thymus gland removed.
What's actually going on in research
Trials are testing complement inhibitors and neonatal Fc receptor blockers that may reduce antibody attacks with fewer side effects than steroids. Several B-cell targeting drugs are being studied, along with faster-acting rescue treatments for myasthenic crisis. Researchers are also developing better ways to measure treatment response beyond patient-reported symptoms.
FcRn blockers
These drugs block a receptor that recycles antibodies, causing disease-causing antibodies to break down faster. Efgartigimod was FDA-approved in 2021, and several similar drugs are in trials.
Complement inhibitors
Drugs that block the complement system prevent antibodies from damaging the nerve-muscle junction. Eculizumab and ravulizumab are approved for generalized myasthenia gravis and work differently than traditional immunosuppression.
B-cell depletion
Drugs like rituximab target the B cells that produce harmful antibodies. Trials are testing whether depleting these cells can provide long-lasting control with less frequent dosing.
What to know before you search
Eligibility typically depends on disease severity, antibody type, prior treatments, and whether symptoms are generalized or limited to eye muscles.
What types of trials are currently open
- FcRn blocker trials — Testing newer drugs that lower antibody levels by blocking their recycling pathway. These often involve regular infusions or injections.
- Complement inhibitor trials — Studies of drugs that prevent antibodies from damaging nerve-muscle connections, often comparing them to current treatments or placebo.
- B-cell targeting trials — Testing drugs that deplete or block the immune cells that make disease-causing antibodies.
- Rescue treatment trials — Testing faster-acting treatments for sudden severe worsening or myasthenic crisis.
- Biomarker studies — Observational work tracking antibody levels and muscle function over time to predict treatment response.
Recently added Myasthenia Gravis trials
PANORAMA: Neuromuscular Organoids for Refractory AChR+ Myasthenia Gravis
Myasthenia gravis (MG) is an autoimmune disease in which autoantibodies attack the neuromuscular junction, the site at which nerve cells communicate with muscle fibres, impairing signal transmission and causing fluctuating muscle weakness that worsens with sustained activity. In most patients this dysfunction is reversible and improves with treatments that suppress the immune response. About 10 to 15 percent of patients do not respond adequately to standard therapy, and the mechanisms of this refractory course remain unclear. The study is based on the hypothesis that in refractory patients the autoantibody attack causes irreversible damage to the neuromuscular junction, and that this damage sustains symptoms despite appropriate treatment. A further aim is to identify circulating biomarkers reflecting such damage that may help predict response to therapy. The study includes adults with generalised MG positive for antibodies against the acetylcholine receptor, stratified by disease duration and treatment response into treatment-naive, treatment-sensitive and treatment-refractory MG. Subjects without neuromuscular disease and negative for these antibodies serve as controls. Blood samples (serum, plasma and mononuclear cells) are obtained from material left over from blood draws performed as part of routine care, together with clinical data including disease duration, symptom severity measured with validated scales (MG-ADL and QMG), antibody titre and treatment history. No study-specific visit or blood draw is required. Antibodies purified from participants are applied to human neuromuscular organoids, three-dimensional models grown from stem cells of healthy donors that reproduce key features of the neuromuscular junction. Exposing these organoids to antibodies from patients at different disease stages reproduces the antibody-mediated attack under controlled laboratory conditions and allows the resulting structural and electrical changes to be measured. Molecules released by damaged organoids, including microRNAs and proteins, are identified and then measured in participants' blood. The immune profile of participants, including complement factors, lymphocyte subsets and cytokines, is characterised in parallel. The study will determine whether irreversible neuromuscular junction damage distinguishes treatment-refractory MG from treatment-responsive disease, and whether specific circulating biomarkers can identify a refractory course.
Take an antibody therapy to maintain improvement in myasthenia gravis
The primary purpose of this study is to evaluate the efficacy of ublituximab in adult participants with MG responding to treatment with efgartigimod.
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