MGFA is dedicated to driving research to better understand, treat and cure myasthenia gravis for good. To achieve this goal, we are committed to creating awareness about clinical trials for those with myasthenia gravis and related neuromuscular joint disorders.
Researchers: If you would like your clinical trial posted to our website, please complete the Research Announcement Form and email to mgfa@myasthenia.org with “Clinical Trial Announcement” in the subject line.
Patients: Trials listed here have been shared with the MGFA by researchers and are enrolling new patients to the best of our knowledge. Please check directly with the research sponsor to confirm. Additional trials may be available at clinicaltrials.gov. We encourage you to learn more about options and talk to your medical team to find out if a trial is right for you.
ACTIVE CLINICAL TRIALS
A Phase 1, Open-label, Safety and Dose-finding Study of Autologous Muscle-specific Tyrosine Kinase Chimeric Autoantibody Receptor T Cells (MuSK-CAART) in Subjects With Anti-MuSK-antibody-positive Myasthenia Gravis – Active and Recruiting
This phase 1 study is being conducted to evaluate the safety of various dosing regimens of an investigational cell therapy, MuSK-CAART, that can be given to patients with anti-MuSK antibody positive Myasthenia Gravis (MuSK MG), who have active disease.
Sponsor: Cabaletta Bio
ClinicalTrials.gov page
Cabaletta Study Webpage
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis – Active and Recruiting
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.
Sponsor: argenx
ClinicalTrials.gov page
Study Webpage
Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis – Active and Recruiting
This study will evaluate the efficacy and safety of ALXN2050 (120 milligrams [mg], 180 mg) in participants with generalized myasthenia gravis (gMG).
Sponsor: Alexion
ClinicalTrials.gov page
Study Webpage
Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis - Active and Recruiting
The purpose of this 3-period study is to confirm the efficacy and safety of batoclimab in participants with gMG.
Sponsor: Immunovant
ClinicalTrials.gov page
Study Webpage
A Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis – Active and Recruiting
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in participants with generalized myasthenia gravis (gMG)
Sponsor: Roche
ClinicalTrials.gov page
Study Webpage
An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthenia Gravis – Active and Recruiting
Evaluate the safety and efficacy of an investigational medication, nipocalimab, for the treatment of generalized myasthenia gravis in children aged 2 to less than 18 years old. Evaluate how long the investigational medication, nipocalimab, remains in the body.
Sponsor: Janssen
ClinicalTrials.gov page
Study Webpage
Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis – Active and Recruiting
Establish if nipocalimab is useful for treating Myasthenia Gravis in adult patients. Determine if nipocalimab is safe and record any unexpected or unwanted side effects. Identify how long nipocalimab remains in the body.
Sponsor: Janssen
ClinicalTrials.gov page
Study Webpage
A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (NIMBLE) – Active and Recruiting
To evaluate the effect of pozelimab + cemdisiran on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG)
Sponsor: Regeneron
ClinicalTrials.gov page
Study Contact Email
A Randomized, Double-blind, Multicenter, Placebo-controlled Phase 3 Study With Open-label Period to Evaluate the Efficacy and Safety of Inebilizumab in Adults With Myasthenia Gravis – Active and Recruiting
This MINT study is a phase 3, randomized, double-blind, placebo-controlled study, to be conducted at approximately 100 study sites.
Sponsor: Horizon Therapeutics
ClinicalTrials.gov page
Study Contact Email
Autologous T-Cells Expressing A Chimeric Antigen Receptor Directed To B-Cell Maturation Antigen (BCMA) In Patients With Generalized Myasthenia Gravis (MG) – Active and Recruiting
This is a Phase Ib/IIa study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with generalized myasthenia gravis.
Sponsor: Cartesian Therapeutics
ClinicalTrials.gov page
Study Webpage
Quantitative Ultrasonography and Electrical Impedance Myography: Speech and Swallowing Technologies for Use in Neurological Disorders – Active and Recruiting
This study is evaluating the use of two painless, non-invasive technologies in the assessment of muscle health over time in both healthy volunteers and patients who have diseases that affect the nervous system.
Sponsor: Beth Israel Deaconess Medical Center
ClinicalTrials.gov page
A Phase 3, Open-Label Study of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis – Active, Not Recruiting
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of eculizumab in the treatment of pediatric refractory gMG based on change from Baseline in the Quantitative Myasthenia Gravis (QMG) score for disease severity.
Sponsor: Alexion Pharmaceuticals
ClinicalTrials.gov page