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. MGFA is not affiliated with the trials listed below. 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.
Trials listed below are segmented by adult and pediatric patients.
ACTIVE ADULT CLINICAL TRIALS
ADAPT Forward – Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis – Active and Recruiting
ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients’ side effects and improve their quality of life.
Sponsor: argenx
Webpage: https://www.clinicaltrials.gov/study/NCT07294170
Study website: https://clinicaltrials.argenx.com/adaptforward
Study Contact: clinicaltrials@argenx.com
A Phase 4, Single Arm Study to Investigate OCS Taping in Adult Participants with Generalized Myasthenia Gravis Treated with Ravulizumab (OCTAGON) – Active and Recruiting
This is a prospective, multicenter single arm study designed to evaluate the effectiveness and safety of a predefined oral corticosteroids (OCS) tapering schedule to reduce OCS use in adult participants with acetylcholine receptor positive (AChR+) generalized myasthenia gravis (gMG) being treated with intravenous ravulizumab.
Sponsor: Alexion Pharmaceuticals
Webpage: https://clinicaltrials.gov/study/NCT07221838
Study Contact: clinicaltrials@alexion.com
ADAPT Forward 1 – ISA1 – a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod – Active and Recruiting
This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients’ side effects and improve their quality of life. The aim of this ISA1 is to evaluate the safety and therapeutic relevance of empasiprubart as add-on therapy to efgartigimod in participants with AChR-Ab seropositive generalized myasthenia gravis.
Sponsor: argenx
Webpage: https://clinicaltrials.gov/study/NCT07284420
Study website: https://clinicaltrials.argenx.com/adaptforward1
Study Contact: clinicaltrials@argenx.com
A Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adults With New-onset Generalized Myasthenia Gravis (gMG) (ADAPT-EARLY) – Recruiting
The main purpose of this study is to measure how well adults with new-onset gMG (which means they’ve had generalized disease signs and/or symptoms for less than 1 year) respond to treatment with efgartigimod PH20 SC. The study consists of a treatment period of 51 weeks. The study duration for each participant will be approximately 58 weeks.
Sponsor: argenx
Webpage: https://clinicaltrials.gov/study/NCT06909214
Study contact: clinicaltrials@argenx.com
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants’ symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7, MUSK, AGRN, or LRP4.
Sponsor: argenx
Study Webpage: https://clinicaltrials.argenx.com/cms
Webpage: https://clinicaltrials.gov/study/NCT06078553
Study contact: clinicaltrials@argenx.com
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 phase 3, randomized, double-blind, placebo-controlled clinical study is to investigate the sustained efficacy, the need for retreatment, and the long-term safety of a new formulation of oral cladribine as compared with placebo in participants with generalized myasthenia gravis (MyClad).
Sponsor: EMD Serono
Webpage: https://clinicaltrials.gov/study/NCT06463587
Study Webpage: www.mycladstudy.com
Study Contact: eMediUSA@emdserono.com
A Non-interventional, Post-authorisation Safety Study of Patients Treated With Efgartigimod Alfa – Recruiting
This is a non-interventional, prospective, post authorization safety study. Patients with gMG who are expected to start treatment with efgartigimod at enrollment or are within their first cycle of efgartigimod at enrollment will be eligible to participate in the efgartigimod cohort. Patients with gMG who have not been exposed to efgartigimod and for whom it is not planned to start treatment with efgartigimod at enrollment will be eligible to enroll in the non-efgartigimod cohort.
Sponsor: argenx
Webpage: https://clinicaltrials.gov/study/NCT006298565
Study contact: clinicaltrials@argenx.com
A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases – Active and Recruiting
This is a Phase 1 study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG). Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.
Sponsor: Arcellx
Webpage: clinicaltrials.gov/study/NCT06626919
RESET-MGTM: A Phase 1/2 Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis – Active and Recruiting
The RESET-MG™ Study is a clinical research trial evaluating the effects of an investigational chimeric antigen receptor (CAR) T cell therapy, CABA-201, in patients with generalized myasthenia gravis.
Sponsor: Cabaletta Bio
Webpage: clinicaltrials.gov/study/NCT06359041
Study Webpage: www.cabalettabio.com/patients/phase-12-trial-in-myasthenia-gravis or download a flyer
A Worldwide Pregnancy Safety Study to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to Efgartigimod During Pregnancy and/or Breastfeeding. – Recruiting
This is a multi-country, prospective safety study of pregnant women exposed to efgartigimod or efgartigimod PH20 SC any time within 25 days prior to conception or any time during pregnancy. Women exposed to efgartigimod or efgartigimod PH20 SC only during breastfeeding will also be eligible to enroll. Background rates of major congenital malformations (MCMs) will be obtained from populations within the same countries/regions as the countries/regions in which the efgartigimod or efgartigimod PH20 SC exposed pregnancies were reported.
Sponsor: argenx
Webpage: https://clinicaltrials.gov/study/NCT006299748
Study contact: clinicaltrials@argenx.com
Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (AURORA) – Active and Recruiting
This is a randomized, double blind, placebo-controlled Phase III study to evaluate the efficacy, safety and tolerability of Descartes-08, mRNA CAR T-cell therapy, in patients with generalized myasthenia gravis.
Sponsor: Cartesian Therapeutics
Webpage: https://clinicaltrials.gov/study/NCT06799247
Sponsor Study Webpage: https://myastheniagravis.study.science/
Patient Webpage: Patients – Cartesian Therapeutics
Study Contact: trials@cartesiantx.com
KYSA-6: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Patients With Generalized Myasthenia Gravis – Active and Recruiting
B cells play a role in MG, and the disease is characterized by the presence of autoantibodies such as anti-AChR and anti-MuSK antibodies. CD-19 target chimeric antigen receptor (CAR) T cells harness the ability of cytotoxic T cells to directly and specifically lyse target cells to effectively deplete both normal and autoreactive B cells in the circulation as well as impacted lymphoid and potentially non-lymphoid tissues.
Sponsor: Kyverna Therapeutics
Webpage: www.clinicaltrials.gov/study/NCT06193889
Study Webpage: www.myastheniagravistrials.com
Study Contact: Clinicaltrials@kyvernatx.com
Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Generalized Myasthenia Gravis – Active and Recruiting
This is a phase I/II study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia gravis. YTB323 is a Biological CAR-T cell therapy.
Sponsor: Novartis Pharmaceuticals
Webpage: clinicaltrials.gov/study/NCT06704269
Study Contact: novartis.email@novartis.com
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy – Active and Recruiting
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of selected fetal/neonatal/infant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.
Sponsor: Alexion Pharmaceuticals
Webpage: clinicaltrials.gov/study/NCT06312644
Study Website: www.UltomirisPregnancyStudy.com
Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults with Ocular Myasthenia Gravis (ADAPT oculus)
The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC.
Sponsor: argenx
Webpage: clinicaltrials.gov/study/NCT06558279
Study Contact: clinicaltrials@argenx.com
A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (RemeMG) – Active and Recruiting
The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis. This study is a randomized, double-blind, placebo-controlled Phase 3 study with an open-label extension to evaluate the efficacy and safety of telitacicept in a global patient population with gMG.
Sponsor: Vor Biopharma Inc.
Webpage: clinicaltrials.gov/study/NCT06456580?term=RC18G006&rank=1#contacts-and-locations
Study Contact: study@vorbio.com
Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis (SYNAPSE-MG) – Active and Recruiting
This Phase 2 proof-of-concept, dose range finding study aims to evaluate the safety and efficacy of 3 dose levels of NMD670 vs placebo in adult patients with MG with antibodies against AChR or MuSK, administered twice a day (BID) for 21 days. To qualify for this study, participants must be 18 to 75 years of age, diagnosed with MG, and able to swallow tablets.
Sponsor: NMD Pharma A/S
Webpage: clinicaltrials.gov/study/NCT06414954
Study Contact: contact@nmdpharma.com
Published Data: www.science.org/doi/10.1126/scitranslmed.adk9109?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG. – Active and Recruiting
The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment.
Sponsor: Novartis Pharmaceuticals
Webpage: clinicaltrials.gov/study/NCT04833894
Study Contact: novartis.email@novartis.com
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG. – Active and Recruiting
The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment.
Sponsor: Novartis Pharmaceuticals
Webpage: clinicaltrials.gov/study/NCT04833894
Study Contact: novartis.email@novartis.com
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
Webpage: https://clinicaltrials.gov/study/NCT06106672
Study Webpage: courpharma.com/our-clinical-trials/cnp-106-clinical-trial-myasthenia-gravis/
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)
Webpage: https://clinicaltrials.gov/study/NCT06259071
Study Webpage: musk1000.smhs.gwu.edu/
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
Webpage: https://clinicaltrials.gov/study/NCT06055959
Study Contact: ucbcares@ucb.com
ACTIVE PEDIATRIC PATIENT TRIALS
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis – Active and Recruiting
The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants ages 6 to less than 18 with AChR+ gMG for the duration of the study.
Sponsor: Alexion Pharmaceuticals
Webpage: https://clinicaltrials.gov/study/NCT06607627
Study Webpage: alexion.com
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
Webpage: https://clinicaltrials.gov/study/NCT05644561
Study Webpage: alexion.com/
A Phase 2/3 Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis – Active and Recruiting
The Vibrance-MG study evaluates 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. The study will evaluate how long the investigational medication, nipocalimab, remains in the body.
Sponsor: Janssen Research & Development, LLC
Webpage: clinicaltrials.gov/study/NCT05265273
Study Webpage: clinicaltrials.jnj.com/en/study-detail/NCT05265273??page=1&conditions=Myasthenia+Gravis
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis (ADAPT Jr) – 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. Details include maximum trial duration for each invidual participant will be approximately 28 weeks and treatment duration will be 8 weeks for the dose-confirmatory (Part A) and 18 weeks for the treatment response – confirmatory part (Part B).
Sponsor: argenx
Webpage: https://clinicaltrials.gov/study/NCT04833894
Study Contact: clinicaltrials@argenx.com
A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis (ADAPT Jr SC) – Active and Recruiting
The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to <18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.
Sponsor: argenx
Webpage: https://clinicaltrials.gov/study/NCT06392386
Study Contact: clinicaltrials@argenx.com
ACTIVE, NOT RECRUITING
Myasthenia Gravis Inebilizumab Trial – Active, Not Recruiting
This MINT study is a phase 3, randomized, double-blind, placebo-controlled study, to be conducted at approximately 120 study sites.
Sponsor: Amgen (acquired from Horizon Therapeutics in 2023)
Webpage: clinicaltrials.gov/ct2/show/NCT04524273
Study Webpage: https://www.amgentrials.com/
Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants with Generalized Myasthenia Gravis – Active, Not Recruiting
The purpose of this 3-period study is to confirm the efficacy and safety of batoclimab in participants with gMG.
Sponsor: Immunovant
Webpage: clinicaltrials.gov/ct2/show/NCT05403541
Study Webpage: flex.researchstudytrial.com/
A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis (ADAPT SERON) – Active, NOT 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). Other objectives are to assess long-term efficacy, safety, and tolerability of efgartigimod.
Sponsor: argenx
Webpage: clinicaltrials.gov/study/NCT06298552
Study Contact: clinicaltrials@argenx.com
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) – Active, NOT Recruiting
The purpose of this phase 1b study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function.
Sponsor: argenx
Webpage: clinicaltrials.gov/study/NCT06436742
Study Contact: clinicaltrials@argenx.com
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
Webpage: https://clinicaltrials.gov/study/NCT06298552
Study Webpage: www.us.argenx.com/
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 Research & Development, LLC
Webpage: clinicaltrials.gov/show/NCT04951622
Study Webpage: clinicaltrials.jnj.com/en/study-detail/NCT04951622??page=1&conditions=Myasthenia+Gravis
A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis (NIMBLE) – Active and Recruiting
This study is researching an experimental combination treatment with pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on patients with generalized myasthenia gravis (gMG).
Sponsor: Regeneron
Webpage: https://clinicaltrials.gov/study/NCT05070858
Study Contact: clinicaltrials@regeneron.com