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
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: https://clinicaltrials.gov/study/NCT06312644?term=NCT06312644&rank=1
Study Website: www.UltomirisPregnancyStudy.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: https://clinicaltrials.gov/study/NCT06704269?cond=Myasthenia%20Gravis,%20Generalized&term=NCT06704269&rank=1
Study Contact: novartis.email@novartis.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: https://clinicaltrials.https://clinicaltrials.gov/study/NCT06558279?cond=Ocular%20Myasthenia%20Gravis&spo
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: RemeGen Co., Ltd.
Webpage: https://clinicaltrials.gov/study/NCT06456580?term=RC18G006&rank=1#contacts-and-locations
Study Contact: RC18_MGstudy@remegenbio.com
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: https://clinicaltrials.gov/study/NCT06359041
Study Webpage: https://www.cabalettabio.com/patients/phase-12-trial-in-myasthenia-gravis
Descartes-08 CAR-T Cells in 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
Webpage: https://clinicaltrials.gov/study/NCT04146051
Study Webpage: www.cartesiantherapeutics.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: https://clinicaltrials.gov/study/NCT06414954
Study Contact: contact@nmdpharma.com
Published Data: https://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: https://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: https://clinicaltrials.gov/study/NCT04833894
Study Contact: novartis.email@novartis.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 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). Other objectives are to assess long-term efficacy, safety, and tolerability of efgartigimod.
Sponsor: argenx
Webpage: https://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 and 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: https://clinicaltrials.gov/study/NCT06436742
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 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
Webpage: https://clinicaltrials.gov/study/NCT06463587
Study Webpage: https://clinicaltrials.emdgroup.com/en/trial-details/?id=MS700568_0183
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 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 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
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
Webpage: www.clinicaltrials.gov/study/NCT05566964
Study Webpage: https://www.meandmgopen.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
Webpage: clinicaltrials.gov/show/NCT04951622
Study Webpage: www.globaltrialfinder.janssen.com/trial/CR109046
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
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. Evaluate how long the investigational medication, nipocalimab, remains in the body.
Sponsor: Janssen/Johnson & Johnson
Webpage: https://clinicaltrials.gov/study/NCT05265273
Study Webpage: globaltrialfinder.janssen.com/trial/CR109137
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
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 – 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.
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/