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

Fast replyRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a disease called Generalized Myasthenia Gravis (gMG), which is a condition that affects the muscles, making them weak and tired. The study is testing a treatment called ravulizumab, also known by its code name ALXN1210. Ravulizumab is given through an intravenous infusion, which means it is delivered directly into the bloodstream through a vein. The purpose of the study is to understand how ravulizumab works in children and teenagers aged 6 to under 18 years who have gMG.

Participants in the study will receive ravulizumab and will be monitored over a period of time to see how their bodies respond to the treatment. The study will look at various aspects such as how the drug moves through the body, its effects on the disease, and any side effects that may occur. The study will also assess how the treatment impacts the participants' quality of life and their ability to perform daily activities.

The trial is designed to gather information on the safety and effectiveness of ravulizumab in young people with gMG. Participants will be closely observed by healthcare professionals throughout the study to ensure their well-being and to collect data that will help in understanding the potential benefits and risks of using ravulizumab for treating gMG in this age group.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, you will have an initial visit. During this visit, you will receive a detailed explanation of the study procedures and what to expect throughout the trial.

    You will be asked to provide informed consent, which means you agree to participate in the study after understanding all the details.

  2. Step 2

    Screening

    During the screening phase, your eligibility for the study will be confirmed. This includes checking your age, weight, and medical history related to generalized myasthenia gravis (gMG).

    You will undergo tests to confirm your diagnosis and ensure you meet all the study criteria.

  3. Step 3

    Vaccination

    To reduce the risk of infection, you must be vaccinated against certain bacteria, including meningococcal infection, Haemophilus influenzae type b, and Streptococcus pneumoniae.

    If you have not been vaccinated within the last three years, you will receive these vaccinations before starting the study treatment.

  4. Step 4

    Treatment administration

    You will receive the study medication, ravulizumab, through an intravenous infusion. This means the medication will be given directly into your vein.

    The dosage and frequency of the medication will be determined by the study team based on your specific needs and condition.

  5. Step 5

    Regular follow-up visits

    You will have regular follow-up visits to monitor your response to the treatment and check for any side effects.

    These visits will include physical examinations, blood tests, and assessments of your generalized myasthenia gravis (gMG) symptoms.

  6. Step 6

    End of treatment evaluation

    At the end of the treatment period, you will have a final evaluation to assess the overall effects of the medication.

    This evaluation will include a review of your symptoms, any changes in your condition, and any side effects experienced during the study.

  7. Step 7

    Long-term follow-up

    After completing the treatment, you may be asked to participate in a long-term follow-up to monitor your health and any lasting effects of the medication.

    This phase is important to gather information on the long-term safety and effectiveness of the treatment.

Who can join the trial?

14 criteria

  • Must be between 6 and 17 years old and weigh at least 10 kg (about 22 pounds).
  • Must have a confirmed diagnosis of Generalized Myasthenia Gravis (gMG) through a positive blood test for specific antibodies.
  • Must have a certain score on a test called QMG (Quantitative Myasthenia Gravis) that measures muscle strength, or have documented limb weakness if younger than 12 years old.
  • Must be classified as Class II to Class IV according to the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification, which describes the severity of the condition.
  • If currently receiving treatments like eculizumab or ravulizumab, must have been on the treatment for at least 6 months and on a stable dose for at least 2 months. If previously received these treatments, there must be a gap of at least 67 days for eculizumab and 308 days for ravulizumab before starting the study drug.
  • Must be on a stable dose of certain medications for a specific period before the study, such as:
    • AZA (azathioprine) for at least 6 months and stable for 2 months.
    • ISTs (immunosuppressive therapies like MMF, MTX, CYC, TAC, or CY) for at least 3 months and stable for 4 weeks.
    • Chronic IVIg (intravenous immunoglobulin) for at least 6 months and stable for 3 months.
    • Oral corticosteroids for at least 4 weeks.
    • AChIs (acetylcholinesterase inhibitors) for at least 2 weeks.
    • Must be vaccinated against meningococcal infection (a type of bacterial infection) within the last 3 years or at least 14 days before starting the study. If not, must receive the vaccine and take preventive antibiotics if starting the study less than 2 weeks after vaccination.
    • Must have received vaccinations against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to current guidelines.
    • Female participants who can have children and male participants must agree to use specific birth control methods during the study.
    • The participant's legal guardian must be able to give written consent, and the participant must be able to give written agreement to participate, if required.

Who cannot join the trial?

9 criteria

  • Patients who do not have Generalized Myasthenia Gravis (gMG) cannot participate. This is a condition that affects the muscles, causing weakness.
  • Patients who are not within the specified age range for the study cannot participate. The study is for children and teenagers.
  • Patients who are not able to follow the study procedures or take the study medication as required cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are taking certain medications that might interfere with the study cannot participate.
  • Patients who have had a recent infection or illness that might interfere with the study cannot participate.
  • Patients who have a history of allergic reactions to similar medications cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently cannot participate.
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Investigated drugs

Ravulizumab is a medication used in this clinical trial to help treat a condition called generalized myasthenia gravis (gMG) in children and teenagers. This condition affects the muscles, making them weak and tired. Ravulizumab works by blocking a part of the immune system that is overactive in people with gMG. By doing this, it helps to reduce muscle weakness and improve the ability to perform daily activities. In this study, the medication is given through an intravenous infusion, which means it is delivered directly into the bloodstream through a vein. The trial aims to understand how the body processes the medication, how it affects the disease, and to ensure it is safe and effective for young patients.

What is already known about the treatment

Ravulizumab – Ravulizumab is administered through an intravenous infusion, meaning it is given directly into a vein. It is currently being studied in a Phase 3 clinical trial to assess its effects in children aged 6 to under 18 years with Generalized Myasthenia Gravis (gMG), a condition that causes muscle weakness. Ravulizumab works by blocking a part of the immune system that can attack the body's own cells, helping to reduce symptoms of the disease. It belongs to a class of drugs known as complement inhibitors, which help control the immune response.

Investigated diseases

Generalized Myasthenia Gravis (gMG) – Generalized Myasthenia Gravis is an autoimmune disorder that affects the communication between nerves and muscles, leading to muscle weakness. It occurs when the immune system mistakenly attacks the body's own tissues, specifically targeting the acetylcholine receptors at the neuromuscular junction. This disruption impairs the transmission of nerve signals to muscles, causing fluctuating muscle weakness and fatigue. The condition often begins with ocular symptoms, such as drooping eyelids and double vision, and can progress to involve other muscle groups, including those responsible for breathing and limb movement. Muscle weakness in gMG typically worsens with activity and improves with rest. The severity and distribution of muscle weakness can vary among individuals, and symptoms may fluctuate over time.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-501882-29-00Protocol codeALXN1210-MG-319Estimated enrolment12 patientsSponsorAlexion Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

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