Myasthenia Gravis

This study will have two Phases: Phase 1a and Phase 1b. The goals of this clinical study are to learn more about the study drug KITE-363, by evaluating its safety, tolerability and efficacy in participants with relapsed/refractory autoimmune neurologic diseases. The primary objectives of this study are: * To evaluate the safety and tolerability of KITE-363 in participants with autoimmune neurologic diseases * To determine the recommended dose for Phase 1b. * To evaluate the preliminary efficacy of KITE-363 in participants with autoimmune neurologic diseases.

The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG).

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.

The primary purpose of this study is to evaluate the efficacy of ublituximab in adult participants with MG responding to treatment with efgartigimod.

Neuroimmune diseases are more prevalent among women of reproductive age. Studies have shown that neuroimmune diseases may impact fertility. Therefore, effective management of neuroimmune diseases during pregnancy is particularly important. This study included a follow-up period of up to five years in patients with pregnancy-associated neuroimmune disorders. Data collected included relapse frequency, symptomatology, imaging findings, treatment regimens, peripheral blood profiles, EDSS scores, and MRI results. In addition, maternal drug concentrations, postpartum relapse rates, and neonatal development were monitored after delivery. Following the successful completion of the five-year follow-up, the research team plans to continue the prospective epidemiological study with ten-year follow-up phases. The aim of this study is to generate detailed clinical data on pregnancy-associated autoimmune diseases and to equip clinicians with evidence-based strategies for optimizing disease management during the reproductive age.

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.

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. The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170

The purpose of this study is to assess how well nipocalimab works when compared to efgartigimod in participants with generalized myasthenia gravis (a condition in which body's immune system mistakenly attacks and damages the connection between nerves and muscles causing muscle weakness).

The purpose of the study is to assess the clinical efficacy of rozanolixizumab in adult Chinese participants with generalized myasthenia gravis (gMG) in the first Treatment Cycle.

A Study of the Anti-CD 19 Chimeric Antigen Receptor T Cell Therapy for Patients with Myasthenia Gravis

Individuals with MG (IwMG) experience shortness of breath that may be activity-related, occur at rest and even happen during sleep. Dyspnea is a complex, multidimensional and multifactorial symptom involving sensory perception, cognition and emotion. Identifying the cause(s) of dyspnea in MG may assist in finding therapeutic strategies, reducing discomfort, improving QoL and potentially limiting respiratory deterioration and incidence of MG crisis.

Myasthenia gravis primarily results from neuromuscular junction dysfunction and damage, leading to chronic muscle weakness. While combined traditional and Western medicine treatments are now common, TCM tongue diagnosis remains a simple and practical clinical method. However, results can vary among different practitioners. Therefore, you are interested in using scientific methods to compare TCM tongue diagnosis images between patients with myasthenia gravis and healthy individuals to aid in diagnosis.

The overall purpose of this pilot study is to examine the feasibility, acceptability, and tolerability of light and moderate intensity exercise in adults with MGeffect of light vs. moderate intensity exercise on health outcomes. Participants will be enrolled into the NeuroWell exercise program, which is geared toward individuals with neurological disorders or injuries and led by Certified Exercise Physiologists (CEPs) at the HealthPartners Neuroscience Center. A total of 20 people with MG will be enrolled in this study and participate in a small group exercise program 3 times a week for 12 weeks. Participants will be randomized into two exercise groups: 1) Light intensity or 2) Moderate intensity. We hypothesize that light and moderate intensity exercise will be feasible, acceptable, and tolerable in adults with MG and that individuals in the light intensity exercise group will be able to achieve the same improvement in health outcomes as the moderate intensity group.

This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD). Due to motor unit impairments, NMD patients often cannot tolerate traditional exercise. WB-EMS bypasses voluntary activation limits by directly stimulating muscle contractions. Up to 50 adults with conditions like ALS, SMA, and MG will undergo 20-minute supervised WB-EMS sessions (1-2 times weekly for 4-8 weeks) using the Katalyst system. Outcomes include neural excitability (TMS), motor unit behavior (EMG, NCS), functional tests (walk, balance, strength), and patient-reported fatigue, pain, and quality of life. Strict safety monitoring and exclusion criteria are in place. This study will provide preliminary data on WB-EMS as a potential exercise modality for NMD.

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 (globally) and 8 to \<18 years of age (for Unites Stated (US) sites only), 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.

A Phase 1 dose-escalation 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.

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.

This is a non-interventional, prospective, post authorization safety study. Patients with gMG who are expected to start treatment with efgartigimod at enrolment or are within their first cycle of efgartigimod at enrolment will be eligible to enroll into 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 enrolment will be eligible to enroll into the non-efgartigimod cohort.

Myasthenia Gravis (MG) is a rare autoimmune disease that causes muscle weakness and fatigue. It occurs when the immune system produces antibodies that block communication between nerves and muscles. In some patients, the disease can suddenly worsen and cause severe breathing problems. This life-threatening situation is called a myasthenic crisis and requires immediate treatment in an intensive care unit (ICU). During such crises, patients may need to receive respiratory assistance through a ventilator. These episodes are often long and can lead to complications such as infections or heart problems. To manage a myasthenic crisis, doctors usually use treatments that remove or neutralize the harmful antibodies: plasma exchange (PLEX) or intravenous immunoglobulin (IVIg). Although both are effective, recovery can be slow, and many patients remain in the ICU for several weeks. Ravulizumab (Ultomiris®) is a new medicine that targets a specific part of the immune system called the complement system, which contributes to muscle damage in MG. It is already approved for adults with generalized MG who have anti-acetylcholine receptor (AChR) antibodies. Ravulizumab is given by intravenous infusion every eight weeks. Clinical studies have shown that it can improve symptoms within one week of starting treatment. Some doctors have started using ravulizumab early, after PLEX or IVIg, for patients hospitalized in the ICU for a myasthenic crisis. Early use of this treatment could help reduce the duration and severity of the crisis, leading to faster recovery and shorter hospital stays. However, there is currently no national study that systematically collects data on this approach. The EARLY-MG study aims to describe the condition and recovery of patients who receive ravulizumab early during a myasthenic crisis requiring ICU admission. The study will not test an experimental treatment or change medical care. It is an observational study. The main hypothesis of the study is that early administration of ravulizumab, after PLEX or IVIg, may help patients recover faster, improve muscle strength, and reduce complications and hospital stay. Around 30 adult patients with generalized MG and anti-AChR antibodies will be enrolled in 10 centers across France. Each patient will be followed for 26 weeks (about six months). Assessments will be performed at the start of the study and at weeks 2, 4, 10, 18, and 26. Investigators will collect information such as: * Duration of stay in the ICU and in the hospital after receiving ravulizumab * Duration of mechanical ventilation, if needed * Clinical improvement using standard evaluation scales (Myasthenia Gravis Activities of Daily Living, MG Foundation of America classification, and Garches' score) * Occurrence of any complications or additional treatments The study will last about 18 months in total, including one year for patient inclusion and six months of follow-up per patient. The results may help guide future recommendations and improve patient care in France and worldwide.

The goal of this clinical trial is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential efficacy of IM-101 in adult participants with AChR antibody-positive gMG. Subsequently, the safety and efficacy of the selected IM-101 dose-regimen will be tested in participants with AChR antibody-negative gMG and participants with AChR antibody-positive or AChR antibody-negative oMG.

The purpose of this study is to evaluate the efficacy and safety of nipocalimab compared to placebo in participants with generalized myasthenia gravis (gMG). The purpose of the subcutaneous substudy is to evaluate how well it works in the body (pharmacodynamic \[PD\]) when given as an injection under the skin (subcutaneous) compared to when given through a vein (intravenous) in participants with gMG.

This is an open label, single-site, dose-escalation study in up to 15 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells（QT-019C）.

Neurological Autoimmune Diseases (NADs) are disorders caused by abnormal immune system attacks on neural tissues, affecting multiple systems including the central nervous system, peripheral nervous system, and neuromuscular junctions. This study examines clinically significant NADs such as multiple sclerosis (MS), neuromyelitis optica spectrum disorders (NMOSD), myelin oligodendrocyte glycoprotein G antibody-related diseases (MOGAD), autoimmune encephalitis (AE), immune-mediated peripheral neuropathy (PN), myasthenia gravis (MG), and idiopathic inflammatory myopathy (IIM). While sharing the core pathogenesis of autoimmune response, these diseases exhibit significant heterogeneity in epidemiological patterns, clinical manifestations, therapeutic approaches, and disease progression. This heterogeneity stems from multiple factors: (1) Differences in immune targets: MS primarily involves T-cell-mediated myelin attack, NMOSD is mainly driven by astrocyte damage caused by anti-AQP4 antibodies, MOGAD results from myelin surface loss mediated by antibodies against myelin oligodendrocyte glycoprotein immunoglobulin G, while AE involves synaptic dysfunction due to antibodies against neuronal surface proteins (e.g., anti-NMDA-R antibodies); (2) Genetic-environmental interactions: MS is more prevalent in European and American populations, whereas NMOSD is more aggressive in Asian populations; (3) Variability in treatment response: Some diseases respond well to immunomodulatory therapy, but most still face challenges such as high relapse rates, progressive disability accumulation, and irreversible neurological damage. While randomized controlled trials (RCTs) provide high-quality core evidence for drug registration, their strict inclusion/exclusion criteria, relatively homogeneous patient populations, and short-term observation designs often fail to fully capture the complex disease progression and treatment response patterns in real-world clinical settings. Additionally, long-term RCTs are frequently constrained by economic factors and sustainability challenges. Therefore, conducting comprehensive real-world observational studies (RWS) on NADs-integrating multi-disease cohorts, long-term follow-up data, and diverse clinical practices-holds significant scientific and clinical value for optimizing treatment strategies and improving long-term patient outcomes.

This is an open label, single-site, dose-escalation study in up to 12 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells.

This study called "Multicenter Retrospective Study on the Short- and Medium-Term Efficacy and Tolerance of Zilucoplan Therapy in a Cohort of French Patients with Anti-AChR Myasthenia Gravis", is investigating the effects of a new treatment called Zilucoplan (generic name: ZILBRYSQ) on patients in France with a condition known as myasthenia gravis. Myasthenia gravis is an autoimmune disease that causes muscle weakness that worsens throughout the day with activity and impacts the ability to work and perform daily activities. The study aims to see if Zilucoplan can improve muscle strength and quality of life over three months, while also assessing its tolerance and safety by monitoring side effects. Researchers hope this treatment will offer significant benefits for people with this challenging condition.