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
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
A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis – Active and Recruiting
The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to less than (<) 18 years of age, the safety and tolerability of treatment with nipocalimab in children and adolescents and to evaluate the pharmacokinetics (PK) of nipocalimab in children and adolescents with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing, stable standard-of-care therapy.
Sponsor: Janssen
ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT05265273
Study Webpage: https://globaltrialfinder.janssen.com/trial/CR109137
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis – Active and Recruiting
The purpose of this study is to evaluate the efficacy and safety of nipocalimab compared to placebo in participants with generalized myasthenia gravis (gMG).
Sponsor: Janssen
ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT04951622
Study Webpage: Participate-In-This-Study@its.jnj.com
Efficacy and Safety of Tolebrutinib (SAR442168) Tablets in Adult Participants With Generalized Myasthenia Gravis (URSA) – Active and Recruiting
This is a multicenter, randomized, double blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of tolebrutinib compared with placebo in adult participants aged 18 to 85 years old with moderate-to-severe gMG, who are receiving Standard of Care (SoC). The efficacy of tolebrutinib versus placebo during DB period will be assessed by clinical evaluations, that include scales based on physician examination or direct participant feedback i.e., patient reported outcomes (PROs).
ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT05132569
Study Webpage: https://www.sanofistudies.com/us/en/listing/289543/efficacy-and-safety-of-tolebrutinib-sar442168-tablets-in-adult-participants-with-generalized-myasthenia-gravis/
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
Study of Pyridostigmine with Ondansetron in Subjects with Anti-AchR Positive Myasthenia Gravis – Active and Recruiting
The Phase II segment of the trial is designed to evaluate the safety and tolerability of pyridostigmine
when given with ondansetron to subjects with anti-AchR positive myasthenia gravis and pyridostigminerelated gastrointestinal side effects.
Sponsor: DAS Therapeutics
ClinicalTrials.gov page: https://gwdocs.com/research/details/?trial=1382
Study Webpage: http://dastherapeutics.com/
Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis (ADAPTsc) – Active and Recruiting
The purpose of this study is to investigate the Pharmacodynamics (PD), Pharmacokinetics (PK), safety, tolerability, immunogenicity, and clinical efficacy of efgartigimod coformulated with recombinant human hyaluronidase PH20 (rHuPH20) as compared to efgartigimod IV infused in patients with generalized myasthenia gravis (gMG). The study duration is approximately 12 weeks. After screening, patients will be randomized to receive either efgartigimod infusions or efgartigimod PH20 subcutaneously (SC)
Sponsor: argenx
ClinicalTrials.gov page:https://clinicaltrials.gov/ct2/show/NCT04735432?term=efgartigimod&cond=Myasthenia+Gravis&draw=2
Study Webpage: clinicaltrials@argenx.com
Efgartigimod Expanded Access for Generalized Myasthenia Gravis – Active and Recruiting
This expanded access protocol applies to patients with gMG who are not enrolled in an ongoing clinical trial. The aim of the trial is to provide patients with generalized myasthenia gravis (gMG), who are ineligible to participate in a clinical trial, access to efgartigimod treatment before regulatory approval. There are country-specific protocols and also individual use EAP. Recruitment for the treatment protocol in US is now closed (ARGX-113-EAP-2101)
Sponsor: argenx
ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/study/NCT04777734?term=efgartigimod&cond=Myasthenia+Gravis&draw=2
Study Webpage: argenx.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
A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis – Active and Recruiting
The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis.
Sponsor: UCB
ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT04115293
Study Webpage: www.ucb.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 Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis – Active and Recruiting
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
Sponsor: Alexion Pharmaceuticals
ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT03920293?term=Alexion&cond=Myasthenia+-Gravis%2C+Generalized
A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 3 Trial to Evaluate the Efficacy, Safety and Tolerability of ARGX-113 in Patients With Myasthenia Gravis Having Generalized Muscle Weakness – Active, Not Recruiting
The primary objective of the this study is to evaluate the efficacy of ARGX-113 as assessed by the percentage of “Myasthenia Gravis Activities of Daily Living [MG-ADL] responders” after the first treatment cycle in the acetylcholine receptor [AChR]-antibody [Ab] seropositive population
Sponsor: argnex
ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT03669588?term=argenx
Study Webpage: clinicaltrials@argenx.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