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Myasthenia gravis – Trials in Disease

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Ongoing Clinical Trials for Myasthenia Gravis

There are currently 33 clinical trials investigating new treatments for myasthenia gravis, a chronic autoimmune disorder that causes muscle weakness. These trials are testing various medications including monoclonal antibodies, cell therapies, and immunomodulatory agents across multiple countries in Europe. (Also known as: Generalized Myasthenia Gravis, Ocular Myasthenia Gravis, Refractory Generalized Myasthenia Gravis, Seronegative Generalized Myasthenia Gravis)

Clinical trial locations

Study of Descartes-08 compared to placebo in adults with generalized myasthenia gravis

This trial is evaluating Descartes-08, an innovative cell therapy approach for generalized myasthenia gravis. The treatment uses modified CD8+ T cells engineered to target specific proteins involved in the disease process.

Main inclusion criteria: Participants must be at least 18 years old with a confirmed diagnosis of generalized myasthenia gravis classified as MGFA grades II-IV. A minimum MG-ADL score of 6 or higher is required, along with positive acetylcholine receptor antibody test results documented within the past 10 years. If taking immunosuppressive medications, participants must be on stable doses for at least 8 weeks before starting the trial.

Main exclusion criteria: The trial excludes individuals below age 18 or above 65, pregnant or breastfeeding women, and patients with severe myasthenia gravis requiring mechanical ventilation. Those with recent investigational treatment exposure, allergic reactions to similar treatments, active infections, serious medical conditions, compromised immune systems, or significant heart, liver, or kidney disease are also excluded.

Trial focus: The study aims to determine if Descartes-08 can improve the ability to perform daily activities in people with generalized myasthenia gravis. The treatment is administered through intravenous infusion, with participants monitored for 4 months to assess changes in symptoms and daily functioning abilities. The primary outcome measure is achieving at least a 3-point improvement in the MG-ADL score by month 4.

Investigational drug: Descartes-08 is a type of cell therapy using the patient’s own immune cells that have been modified in a laboratory to target mechanisms causing muscle weakness. It is currently in Phase 3 clinical trials.

Study of Efgartigimod PH20 SC for Children Aged 2 to 17 with Generalized Myasthenia Gravis

This trial investigates efgartigimod PH20 SC, a solution for injection administered under the skin, in children aged 2 to less than 18 years with generalized myasthenia gravis.

Main inclusion criteria: Participants must be between 2 and 18 years old with a confirmed diagnosis of generalized myasthenia gravis and positive seropositivity for anti-acetylcholine receptor antibodies. They should have had an unsatisfactory response to previous treatments such as immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors, and must be on stable doses of these medications for at least one month before screening.

Main exclusion criteria: The trial excludes patients with severe allergic reactions to the study medication, those currently participating in other clinical trials, individuals with recent drug or alcohol abuse history, pregnant or breastfeeding women, those with uncontrolled high blood pressure or severe heart disease, active infections requiring treatment, recent cancer history, and those who received recent live vaccines or immune system-affecting treatments.

Trial focus: The study aims to confirm the appropriate dose of efgartigimod PH20 SC for young patients and evaluate how the body processes the treatment. It monitors pharmacokinetic and pharmacodynamic parameters, total IgG levels, anti-acetylcholine receptor antibody levels, and assesses changes in symptoms using various quality of life measures. The treatment is given through subcutaneous injection with the specific dosage and frequency determined during the study.

Investigational drug: Efgartigimod PH20 SC is a medication designed to help improve muscle strength by targeting specific parts of the immune system. It reduces levels of certain antibodies that interfere with communication between nerves and muscles.

Study of KYV-101 Therapy for Patients with Refractory Generalized Myasthenia Gravis

This study tests KYV-101, a type of Chimeric Antigen Receptor T-Cell therapy, in patients with refractory generalized myasthenia gravis who have not responded well to standard treatments.

Main inclusion criteria: Participants must have a confirmed diagnosis of refractory generalized myasthenia gravis with autoantibodies to AChR and MuSK, classified as MGFA Class IIB-IV. They must be between 18 and 64 years old. Both male and female patients are eligible.

Main exclusion criteria: The trial excludes patients without the specific diagnosis, those outside the specified age range, individuals not meeting the clinical trial group criteria, and those not identified as male or female.

Trial focus: The study evaluates the safety, tolerability, and effectiveness of KYV-101 in treating refractory generalized myasthenia gravis. The treatment involves intravenous infusion of modified immune cells designed to target specific proteins involved in the disease. The study monitors adverse events, laboratory abnormalities, and assesses changes in MG-ADL scores, QMG scores, and MGC scores at various timepoints. It also measures changes in specific antibodies, CAR-positive T-cell counts, B-cell counts, and systemic cytokine concentrations.

Investigational drug: KYV-101 uses the patient’s own immune cells, genetically modified to express a chimeric antigen receptor targeting the CD19 protein. It is administered as an infusion of autologous T-cells and belongs to the category of CAR T-cell therapies.

Study of Telitacicept for Treating Generalized Myasthenia Gravis in Patients

This double-blind, placebo-controlled trial evaluates telitacicept, given as an injection, for treating generalized myasthenia gravis over 24 weeks, followed by an open-label extension phase.

Main inclusion criteria: Participants must be at least 18 years old with a confirmed diagnosis of generalized myasthenia gravis showing typical muscle weakness patterns. They must have positive antibodies against AChR or MuSK at screening, an MG-ADL score of 6 points or more (with less than 50% related to eye symptoms), and a QMG score of 11 points or more. Patients can take up to 2 other medications if they meet stability criteria and must agree to use highly effective contraception during the study.

Main exclusion criteria: The trial excludes patients without generalized myasthenia gravis, those outside the specified age range, individuals not meeting specific health criteria, and members of vulnerable populations requiring special protection in research.

Trial focus: The study compares telitacicept to placebo over 24 weeks, measuring changes in MG-ADL scores and other health indicators. The main goal is to determine the effectiveness of telitacicept in improving symptoms. Participants receive the medication through a solution for injection in a pre-filled syringe, with dosage and frequency determined by the study protocol.

Investigational drug: Telitacicept is administered as an injection under the skin and works by targeting specific proteins involved in the immune system to reduce abnormal immune response. It is classified as an immunomodulatory agent currently being evaluated in clinical trials.

Study on Corticosteroids and Rituximab to Prevent Worsening of Ocular Myasthenia Gravis in Newly Diagnosed Patients

This trial examines whether immediate corticosteroid treatment combined with rituximab (if symptoms return during corticosteroid tapering) can prevent ocular myasthenia gravis from spreading to other muscles over two years.

Main inclusion criteria: Patients must be over 18 years old with ocular myasthenia diagnosed within the last 6 months, confirmed by specialist examination or specific tests. Symptoms should be limited to the eyes or surrounding muscles for at least one month. Participants should not have received immune-suppressing treatments for ocular myasthenia gravis and must provide informed consent.

Main exclusion criteria: The trial excludes patients with conditions other than myasthenia gravis, those outside the specified age range, and members of vulnerable populations requiring special protection.

Trial focus: The study monitors participants over time to assess whether the treatment plan prevents generalization of symptoms. Corticosteroids are given immediately upon joining, with rituximab added if ocular symptoms recur during corticosteroid reduction. The trial measures progression rates, symptom severity, hospital visits, and quality of life between treatment groups.

Investigational drugs: Corticosteroids (administered orally or intravenously) reduce inflammation and suppress the immune system. Rituximab (administered through intravenous infusion) targets specific immune cells to reduce immune response. Both are well-established treatments for autoimmune conditions.

Study on Efgartigimod for Children Aged 2-17 with Generalized Myasthenia Gravis

This trial aims to find the best dose of efgartigimod alfa for children aged 2 to less than 18 years with generalized myasthenia gravis, administered through intravenous infusion.

Main inclusion criteria: Participants must be boys or girls between 2 and 18 years old with a confirmed diagnosis of generalized myasthenia gravis meeting MGFA clinical criteria classes II, III, and IVa. They must have a positive serologic test for anti-AChR antibodies and have had unsatisfactory responses to immunosuppressants, steroids, or AChE inhibitors, with stable gMG treatment for sufficient time before the trial.

Main exclusion criteria: The trial excludes patients with severe allergic reactions to the study medication, those in other clinical trials, individuals with recent drug or alcohol abuse, pregnant or breastfeeding women, those with uncontrolled high blood pressure, severe heart disease, active infections, recent cancer history, certain autoimmune diseases, and those who received recent immune system-affecting treatments.

Trial focus: The study monitors how the body processes efgartigimod and its effects on the disease by measuring total IgG levels and anti-acetylcholine receptor antibodies. Regular evaluations assess muscle strength, daily functioning abilities, and quality of life changes throughout the treatment period. The medication is given through intravenous infusion with dosage adjusted based on age and size.

Investigational drug: Efgartigimod is administered intravenously and works by targeting and reducing levels of certain antibodies contributing to myasthenia gravis symptoms. It is classified as an immunomodulatory agent currently in investigational stages.

Study on Efgartigimod for Early Treatment in Patients with Generalized Myasthenia Gravis

This study evaluates the effectiveness and tolerability of efgartigimod when used early in treating patients with generalized myasthenia gravis, administered through intravenous infusion.

Main inclusion criteria: Participants must be 18 years or older with a confirmed diagnosis of myasthenia gravis by a neurologist, classified as MGFA Class II, III, or IV. They need an MG-ADL score of 5 or more points with more than half related to non-eye symptoms. Participants should not have received previous immune-suppressing treatment except corticosteroids. Women of childbearing potential must have negative pregnancy tests and use birth control, while men must use adequate birth control and agree not to donate sperm.

Main exclusion criteria: The trial excludes patients without generalized myasthenia gravis, those without anti-AChR antibodies, individuals outside the specified age range, and members of vulnerable populations.

Trial focus: The study monitors how quickly efgartigimod can improve symptoms and its impact on quality of life. Regular assessments occur at weeks 4, 6, 8, 16, and 28 to measure changes using the MG-ADL scale and other relevant measures. The treatment is given through intravenous infusion with dosage specified by the study protocol.

Investigational drug: Efgartigimod works by reducing levels of certain antibodies that interfere with communication between nerves and muscles. It is administered intravenously and classified as an immunomodulatory drug currently being studied for early treatment of generalized myasthenia gravis.

Study on Nipocalimab for Adults with Generalized Myasthenia Gravis

This double-blind, placebo-controlled trial evaluates nipocalimab, given as an infusion, for adults with generalized myasthenia gravis to assess its effectiveness and safety.

Main inclusion criteria: Participants must be at least 18 years old with a diagnosis of generalized myasthenia gravis confirmed by physical exam and positive anti-acetylcholine receptor or anti-MuSK antibody tests. They need an MG-ADL score of 6 or higher and must have a less than ideal response to current stable treatment. Good vein access is required, and participants should be up-to-date on vaccinations.

Main exclusion criteria: The trial excludes patients without a generalized myasthenia gravis diagnosis, those outside the specified age range, individuals unable to follow study instructions, and members of vulnerable populations.

Trial focus: The study evaluates how effective nipocalimab is in improving daily living activities by monitoring changes in the MG-ADL scale. Blood samples are collected periodically to monitor safety and measure medication levels. Regular monitoring includes assessing participant responses, checking for side effects, and conducting health examinations.

Investigational drug: Nipocalimab is administered intravenously and works by targeting and blocking a specific receptor involved in the immune response. It is classified as an immunomodulatory agent currently in Phase 3 clinical trials.

Study on Nipocalimab for Children Aged 2-17 with Generalized Myasthenia Gravis

This open-label study evaluates nipocalimab’s effectiveness, safety, and behavior in children aged 2 to less than 18 years with generalized myasthenia gravis who have not responded well to current treatment.

Main inclusion criteria: Participants must be between 2 and 17 years old with a diagnosis of generalized myasthenia gravis confirmed by positive blood tests for acetylcholine receptor or MuSK antibodies. They must meet MGFA Class IIB-IV criteria and not be responding well to current treatment according to their doctor. Vaccination requirements against specific bacterial infections must be met.

Main exclusion criteria: The trial excludes patients outside the age range, those with good response to current treatment, individuals on unstable treatment plans, those unable to follow study procedures, patients with other interfering health conditions, pregnant or breastfeeding women, recent clinical trial participants, and those with allergies or recent drug/alcohol abuse history.

Trial focus: The study monitors how nipocalimab is processed in the body and its effects on the disease through intravenous infusion. Regular monitoring includes physical examinations, blood tests, and assessments of muscle strength and overall well-being. The trial evaluates safety through monitoring adverse events and tracking health markers.

Investigational drug: Nipocalimab is administered through intravenous infusion and works by targeting and blocking a specific protein involved in immune response. It is classified as an immunomodulatory agent currently being studied to help reduce symptoms by regulating immune system activity.

Study on Ravulizumab for Children Aged 6 to 17 with Generalized Myasthenia Gravis

This study examines ravulizumab’s effects on children and teenagers aged 6 to under 18 years with generalized myasthenia gravis, administered through intravenous infusion.

Main inclusion criteria: Participants must be between 6 and 17 years old, weigh at least 10 kg, and have a confirmed diagnosis of generalized myasthenia gravis with positive AChR antibody tests. They must have a QMG score of 11 or more (or documented limb weakness for younger participants) and be classified as MGFA Class II to IV. Vaccination requirements against specific bacterial infections must be met within the specified timeframes.

Main exclusion criteria: The trial excludes patients with other serious health conditions, those in other clinical trials, recent surgery patients or those planning surgery, individuals with allergic reactions to similar medications, those unable to follow procedures, patients taking interfering medications, those with drug or alcohol abuse history, pregnant or breastfeeding women, patients with active infections, and those with certain heart conditions.

Trial focus: The study monitors how ravulizumab moves through the body and its effects on the disease through regular assessments. The treatment is administered through intravenous infusion with dosage and frequency determined by the study team. Regular visits monitor health, symptoms, and any side effects through physical examinations, blood tests, and symptom assessments.

Investigational drug: Ravulizumab is administered through intravenous infusion and works by blocking a part of the immune system that can attack the body’s own cells. It belongs to the class of complement inhibitors designed to help control immune response.

Study on Rituximab for Patients with Generalized Myasthenia Gravis

This study evaluates rituximab’s effectiveness and safety in patients with generalized AChR-antibody positive myasthenia gravis, aiming to reduce symptoms and improve quality of life.

Main inclusion criteria: Participants must be 18 years or older with a diagnosis of myasthenia gravis confirmed by specific tests or medical history. They must be classified as MGFA Class II, III, or IV with an MG-ADL score of 5 or greater (with more than 50% related to non-eye symptoms) and a QMG score of 11 or greater. Participants must be taking only corticosteroids with stable doses and willing to follow the study protocol.

Main exclusion criteria: The trial excludes patients with other serious health conditions, recent infections requiring antibiotics, recent myasthenia gravis treatments, severe allergic reactions to medications, pregnant or breastfeeding women, recent clinical trial participants, drug or alcohol abuse history, recent cancer diagnosis, and uncontrolled high blood pressure or heart disease.

Trial focus: The study monitors changes in QMG and MG-ADL scores throughout treatment. Rituximab is given through intravenous infusion with specific dosage and frequency determined by protocol. Regular follow-up visits ensure safety and monitor effectiveness, with assessments conducted at week 12 to evaluate condition changes and potential corticosteroid dosage adjustments.

Investigational drug: Rituximab is administered through intravenous infusion and works by targeting and reducing specific B lymphocytes involved in immune response. It is classified as a monoclonal antibody designed to alter immune system activity.

Summary

The 33 ongoing clinical trials for myasthenia gravis demonstrate significant international research efforts, with particularly strong representation in Poland, Italy, Spain, France, and Germany. Many trials are concentrated in multiple European countries, reflecting collaborative research networks.

Several investigational drugs appear across multiple trials, notably efgartigimod (in various formulations including IV and subcutaneous), nipocalimab, and zilucoplan, suggesting these medications are at advanced stages of development. The trials encompass diverse therapeutic approaches including monoclonal antibodies, complement inhibitors, immunomodulatory agents, and innovative cell therapies.

A notable feature is the inclusion of pediatric populations, with multiple trials specifically designed for children aged 2-17 years, addressing an important gap in treatment options for younger patients. Several trials focus on specific patient subgroups, such as those with refractory disease, seronegative forms, or ocular presentations, demonstrating efforts to address diverse disease manifestations.

Most trials are Phase 2 or Phase 3 studies, indicating these treatments have progressed beyond early safety testing. The research spans various administration routes including intravenous infusion, subcutaneous injection, and oral capsules, offering potential convenience advantages for patients. The trials generally employ rigorous methodologies including double-blind, placebo-controlled designs to ensure reliable results.

Ongoing Clinical Trials on Myasthenia gravis

  • A long-term safety study of KYV-101 in patients previously treated for lupus nephritis, systemic sclerosis, myasthenia gravis, rheumatoid arthritis, or stiff person syndrome

    Recruiting

    1 1 1
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    Investigated drugs:
    Germany

  • Study of oral cladribine compared to placebo for patients with generalized myasthenia gravis

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Bulgaria Czechia France Germany Greece +6

  • Study of IMVT-1402 in Adults with Mild to Severe Generalized Myasthenia Gravis

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Denmark Germany Greece Hungary Italy +3

  • Study of Descartes-08 compared to placebo in adults with generalized myasthenia gravis

    Recruiting

    1 1
    Investigated diseases:
    Italy Poland Spain

  • Study of Gefurulimab for Children and Teens with Generalized Myasthenia Gravis

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Poland

  • Study on the Safety and Effects of YTB323 and Drug Combination for Patients with Treatment-Resistant Generalized Myasthenia Gravis

    Recruiting

    1 1 1
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    Investigated drugs:
    France

  • Study on Efgartigimod for Early Treatment in Patients with Generalized Myasthenia Gravis

    Recruiting

    1 1 1 1
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    Investigated drugs:
    Spain

  • Study of Efgartigimod PH20 SC for Children Aged 2 to 17 with Generalized Myasthenia Gravis

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia France Germany Italy The Netherlands +2

  • Study on the Long-term Safety of Efgartigimod for Children with Generalized Myasthenia Gravis

    Recruiting

    1 1 1 1
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    Investigated drugs:
    Austria Belgium Czechia France Germany Italy +3

  • Study on the Safety and Effectiveness of Rozanolixizumab in Children with Generalized Myasthenia Gravis

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Poland

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