Myasthenia Gravis Clinical Trials | MGFA
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Clinical Trial Opportunities

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

 

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 GravisActive 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

 

A World Without MG