Clinical Research Directory

Evaluation of the Condition of Patients Receiving EARLY Ravulizumab and Admitted in ICU for gMG Crisis

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.

Comparative Efficacy of Nipocalimab and Efgartigimod in Participants With Generalized Myasthenia Gravis

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).

PPDTM CorEvitasTM Myasthenia Gravis (MG) Drug Safety and Effectiveness Registry

The design is a prospective, observational (non-interventional) registry for subjects with myasthenia gravis under the care of a neurology provider. Longitudinal data are collected from both subjects and their treating neurology provider during routine clinical encounters using a structured and standardized data collection method. Approximately 1,500 myasthenia gravis subjects with no defined upper limit and 50 clinical neurology sites will be recruited.

Investigation of the Effects of Proprioceptive Neuromuscular Facilitation Exercises in Patients With Myasthenia Gravis

The goal of this clinical trial is to investigate of the effects of proprioceptive neuromuscular facilitation exercises on fatigue, muscle strength and functional parameters in patient with Myasthenia Gravis. The main questions it aims to answer are: * Do proprioceptive neuromuscular facilitation exercises reduce fatigue in patients with Myasthenia Gravis? * Do proprioceptive neuromuscular facilitation exercises increase muscle strength in patients with Myasthenia Gravis? * Do proprioceptive neuromuscular facilitation exercises improve functional parameters in patients with Myasthenia Gravis? * How well can patients with Myasthenia Gravis tolerate proprioceptive neuromuscular facilitation exercises? Participants will be divided into 2 groups as control group and exercise group with block randomization method. In this single-blind randomized controlled study, the participants in the control group will be placed on a waiting list without any intervention during the 6-week study period and at the end of the study, the interventions applied to the exercise group will be applied exactly the same. Proprioceptive neuromuscular facilitation exercises will be applied to the exercise group 3 days a week, 1 hour a day for 6 weeks. Individuals will be evaluated at the beginning and at the end of the study. Researchers will compare exercise and control group to see if there are effects of proprioceptive neuromuscular exercises patients with Myasthenia Gravis.

Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis

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.

ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis

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.

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

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 More information can be found here: https://clinicaltrials.argenx.com/adaptforward1

A Study to Evaluate the Efficacy and Safety of Maintenance Ublituximab Following Induction With Efgartigimod Administration in Participants With Myasthenia Gravis (MG)

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

A Study Evaluating the Safety and Efficacy of KITE-363 in Relapsed/Refractory Autoimmune Neurologic Diseases

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.

A Study to Evaluate the Safety and Preliminary Efficacy of SYS6020 CAR T-cells in Patients With Refractory Generalized Myasthenia Gravis

This study is a single-arm, open, 2-stage (dose-escalation phase and dose-expansion phase), multi-center, phase I clinical trial to evaluate the safety and tolerance of SYS6020 injection in the participants with refractory systemic myasthenia gravis, and determine the recommended dose (RD) for subsequent studies of the product, and to preliminarily evaluate the clinical efficacy of the product, as well as to explore the pharmacokinetics and immunogenicity of the product in vivo. The dose-escalation phase and dose-expansion phase include 7 periods, and they are respectively in sequence as follows: the screening period, apheresis period, pre-dosing assessment, SYS6020 injection infusion, DLT observation period, the primary follow-up period (6 months), and the long-term follow-up period (5 years). The DLT observation period is 28 days after receiving SYS6020 injection. The participants will not undergo lymphodepleting chemotherapy. The efficacy and safety profile of the participants will be continuously assessed during the trial. Efficacy measurement includes the MG-ADL, QMG, MGC, MG-QoL 15R scale, MGFA clinical classification, and MGFA post-intervention state (MGFA PIS) grading scales, as well as self-antibodies, etc. Safety measurement includes vital signs, physical examination, laboratory tests, cytokines, and ECG, etc. The adverse events and concomitant therapy will be continuously collected during the trial. In addition, during the study period, blood samples will be collected from participants who have received SYS6020 treatment for PK/PD test, and immunogenicity test. For the dose-escalation phase, 3 to 5 dose levels are proposed to be explored. The Safety Monitoring Committee (SMC) will discuss the safety data and make a decision if the next SYS6020 injection could be initiated or dose-escalation could be initiated. After the completion of the dose-escalation phase, the recommended doses would be determined for dose-expansion phase. For the dose-expansion phase, further safety and efficacy data will be collected among the participants who will receive the recommended dose of SYS6020 injection.

Pregnancy Registry, Infants, Serum/Milk Analysis (PRISMA)

PRISMA, is a pregnancy registry study, focused on comprehensively collecting information about pregnancy in women with chronic neurological conditions from across the United States and internationally. Depending on their specific condition (MS, CIS, NMOSD, or other) and their specific treatment, participants will be asked to contribute to different aspects of the study. (1) The biosamples will be blood, breast milk, infant stool, maternal stool and vaginal swab samples, collected at specific time points. (2) The online surveys will be collected at specific time points. All study activities will be discussed with participants upon enrollment. By collecting this information, the investigators hope to gain deeper insights into the relationship between pregnancy, the neurological condition, and maternal and infant health. For example, one of the sub-studies focuses on breast milk collection for women planning postpartum treatment with Ocrevus, Rituxan, Briumvi or Kesimpta. This study is fully remote and all sample collection is optional, so participants can choose which types of samples they wish to provide. For blood draws, participants can schedule a home visit through ExamOne, making participation even more convenient. The investigators aim to enroll women with chronic neurological conditions who are planning pregnancy, currently pregnant, or within one year postpartum.

A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

The primary purpose of this study is to measure the efficacy and safety of efgartigimod IV compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). The study consists of a Part A where participants will be randomized to receive either efgartigimod IV or placebo IV and a Part B where participants completing part A will receive open-label efgartigimod IV. Participants will be in the study for up to (approximately) 3.5 years.

MuSK Myasthenia 1000 Study

The MuSK myasthenia gravis 1000 study seeks to collect saliva samples from 1000 subjects with laboratory confirmed diagnosis of MuSK myasthenia to identify genetic variations associated with MuSK MG. The data collected may be used by researchers to gain a better understanding of the cause of MuSK MG and to identify biomarkers and targeted therapy for MuSK MG.

A Study to Evaluate the Safety, Tolerability, Efficacy, and Drug Levels of CC-97540 in Participants With Relapsing Forms of Multiple Sclerosis, Progressive Forms of Multiple Sclerosis or Refractory Myasthenia Gravis (MG) (Breakfree-2)

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).

A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

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.

Biomarkers in Autoimmune Disease of Nervous System

Neurological autoimmune diseases are a group of disorders characterized by the abnormal immune response attacking the nervous system, including the brain, spinal cord and peripheral nerves. These diseases exhibit high heterogeneity, diverse clinical presentations, and are challenging to diagnose and manage due to a lack of effective treatments. In this study, the investigators will recruit eight kinds of autoimmune diseases of nervous system including Neuromyelitis Optica Spectrum Disorder (NMOSD), Myasthenia Gravis (MG), Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), idiopathic inflammatory myopathy (IIM), and multiple sclerosis (MS), autoimmune encephalitis (AE), Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD). Through this study, the investigators aim to discover biomarkers with high sensitivity, specificity, and stability, which can support early diagnosis, disease monitoring, and personalized treatment for neurological autoimmune diseases, thereby improving the quality of life and prognosis for patients.

KYSA-6: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Patients With Generalized Myasthenia Gravis

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

A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis

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.

Interventions Against Fatigue in Patients With Myasthenia Gravis

A prospective assessor-blinded randomized clinical trial investigating the effect of aerobic exercise therapy or cognitive behavioural therapy on fatigue in patients with myasthenia gravis.

Capturing Key MG-symptoms Using Smartphone Recordings.

This study will make use of a cross-sectional design of MG patients and non-MG participants to quantitatively assess key MG symptoms, and to explore the applicability of machine learning algorithms to their measurement.

A Study of LCAR-AIO CAR-T Cells for Treating Relapsed/Refractory Neurological Autoimmune Diseases

This is a prospective, single-arm, open-label, dose-exploration and expansion clinical study of LCAR-AIO in adult subjects with relapsed/refractory neurological autoimmune diseases.

Depressive Tendency and Counseling Outcomes in Thymoma-Associated MG

This study aimed to examine whether baseline depressive tendency independently predicts post-counseling clinical outcomes in patients with thymoma-associated MG. This single-group pre-post exploratory pilot study enrolled patients with thymoma-associated MG who received eight sessions of Gestalt-oriented psychological counseling. Depressive tendency was defined as a baseline Patient Health Question-naire-9 (PHQ-9) score ≥10. Clinical outcomes, including the Myasthenia Gravis Activities of Daily Living scale (MG-ADL), Myasthenia Gravis Quality of Life 15-item scale (MG-QoL15), and mental BMI (mBMI), were assessed before and after the intervention. Multivariable regression analyses were performed to evaluate whether baseline clinical severity independently predicted post-intervention outcomes after sequential adjustment for baseline scores, age, and sex.

Cohort Study on Neuroimmune Diseases in the Reproductive Age

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.

Exploring Outcomes and Characteristics of Myasthenia Gravis 2

The goal of this prospective observational study is to create a network repository of clinical data and biological samples to help researchers learn more about myasthenia gravis.