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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.

 

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.

 

ACTIVE CLINICAL TRIALS


Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK 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: https://clinicaltrials.gov/ct2/show/NCT05451212

Study Webpage: https://www.cabalettabio.com/patients/muscaartes-phase-1-trial

 

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: https://clinicaltrials.gov/ct2/show/NCT04833894?term=efgartigimod&cond=Myasthenia+Gravis&draw=1&rank=2

Study Webpage: https://www.argenx.com/patients/clinical-trials

 

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: https://clinicaltrials.gov/ct2/show/NCT05218096?term=2050&cond=Myasthenia+Gravis&draw=2&rank=1

Study Webpage: https://expandstudy.alexionclinicaltrials.com/?utm_source=Newsletter%2FSocial&utm_medium=Digital&utm_campaign=MGFA+Campaign%2F2050MG

 

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: https://clinicaltrials.gov/ct2/show/NCT05403541 

Study Webpage: https://flex.researchstudytrial.com/ 


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: https://clinicaltrials.gov/ct2/show/NCT04963270 

Study Webpage: https://forpatients.roche.com/en/trials/autoimmune-disorder/myasthenia-gravis-iab/a-study-to-evaluate-efficacy--safety--pharmacokinetics--13414.html


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: https://clinicaltrials.gov/ct2/show/NCT05265273

Study Webpage: https://globaltrialfinder.janssen.com/trial/CR109137


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: https://clinicaltrials.gov/show/NCT04951622

Study Webpage: https://www.globaltrialfinder.janssen.com/trial/CR109046


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: https://www.clinicaltrials.gov/ct2/show/NCT05070858?term=pozelimab&draw=2 

Study Webpage: clinicaltrials@regeneron.com 


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: https://clinicaltrials.gov/ct2/show/NCT04524273 

Study Webpage: clinicaltrials@horizontherapeutics.com


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: https://clinicaltrials.gov/ct2/show/NCT04146051?term=cartesian&draw=2 

Study Webpage: https://www.cartesiantherapeutics.com/ 


An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis – Active and Recruiting 

The primary objective of this study is to evaluate the efficacy of eculizumab in the treatment of pediatric refractory generalized myasthenia gravis (gMG) based on change from Baseline in the Quantitative Myasthenia Gravis score for disease severity (QMG). 

Sponsor: Alexion Pharmaceuticals 

ClinicalTrials.gov page: Alexion.com


A Randomized, Placebo-control, Parallel Group Study to Evaluate the Effect of Amifampridine Phosphate in Patients With MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis Patients – Active and Recruiting 

Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis. 

Sponsor: Catalyst Pharmaceuticals 

ClinicalTrials.gov page: https://www.clinicaltrials.gov/ct2/show/NCT03304054 


Multi-modal Exercise Program for Chronic Stable Myasthenia Gravis – Active and Recruiting 

Generalized fatigue in myasthenia gravis results in physical deconditioning that reduces fitness and increases risk of obesity, hypertension, elevated cholesterol and type 2 diabetes. This study will examine how active and fit are 30 individuals with chronic, generalized myasthenic subjects. This study will also determine whether a 3 month home exercise program with aerobic, resistive, and pulmonary training can improve physical activity, strength, fitness, lung function and reduce cardiovascular disease risk. 

Sponsor: University of Maryland and Baltimore VA Medical Center 

ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT01047761?term=exercise+for+stable+mg 


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: https://clinicaltrials.gov/ct2/show/NCT02118805?term=Innovative+measures+of+speech+and+swallowing+dysfunction+in+Neurological+Disorders+%28QUESST%29&rank=1 

 

A World Without MG