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 email 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
Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad) – Active and Recruiting
The purpose of this clinical study is to determine the efficacy and safety of a new oral cladribine formulation in participants with Generalized Myasthenia Gravis (gMG) in comparison to placebo.
Sponsor: Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
ClinicalTrials.gov page https://clinicaltrials.gov/study/NCT06463587
Study Webpage
Evaluate the Safety, Tolerability, Pharmacodynamics and Efficacy of CNP-106 in Subjects With Myasthenia Gravis – Active and Recruiting
This is a Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106. The clinical study lasts 222-days (up to 42 days for Screening, 180 Study Days). Subjects ages 18-75 with generalized myasthenia gravis (MG) will be screened up to 42 days prior to enrollment into the clinical study.
Sponsor: COUR Pharmaceutical Development Company
ClinicalTrials.gov page https://clinicaltrials.gov/study/NCT06106672?cond=Generalized%20Myasthenia%20Gravis&intr=CNP-106&rank=1
Study Webpage
MuSK Myasthenia 1000 Study - Active and Recruiting
The purpose of this study to collect saliva samples from 1000 subjects with laboratory confirmed diagnosis of MuSK antibody-positive myasthenia. These saliva samples will be then sent to a neurogenetics laboratory where a genome-wide association study (GWAS) will be conducted which will provide important information of genetic factors leading to MuSK MG.
Sponsor: Myasthenia Gravis Rare Disease Network (MGNet) https://mgnet.rarediseasesnetwork.org/
Clinical Trials page https://clinicaltrials.gov/study/NCT06259071
Study webpage
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG) - Active and Recruiting
The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
Sponsor: Alexion
ClinicalTrials.gov page https://clinicaltrials.gov/study/NCT05644561
Study Webpage
A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis - Active and Recruiting
The primary purpose of this study is to measure the efficacy and safety of efgartigimod intravenously (IV) compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG).
Sponsor: argenx
ClinicalTrials.gov page https://clinicaltrials.gov/study/NCT06298552?cond=Myasthenia%20Gravis&aggFilters=status:not&rank=9
Study Webpage
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis (ziMyG) - Active and Recruiting
The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
Sponsor: UCB
ClinicalTrials.gov page
Descriptive Analysis of Real-world Data Collected With ME&MGopen – Active and Recruiting
The objectives of the study are to collect data on patients' symptoms with a mobile phone application in a real life setting, to assess adherence to the use of the tool, user experience and satisfaction with the application, and safety of use.
Sponsor: Ad scientiam
ClinicalTrials.gov page
Study Webpage
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
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
Acupuncture Treatment for Individuals with Myasthenia Gravis – Active; Not Recruiting
The purpose of the study is to determine the effect of acupuncture treatment on individuals with a diagnosis of myasthenia gravis (MG). We hypothesize that patients with MG who receive acupuncture treatment will have improved quality of life and activities of daily living compared to no treatment.
Sponsor: HealthPartners Institute & Park Nicollet Foundation
ClinicalTrials.gov page
Study Webpage
Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis – Active; Not 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