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Study of Efgartigimod PH20 SC for Children Aged 2 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 condition called Generalized Myasthenia Gravis, which is a chronic autoimmune disease that causes muscle weakness. The study involves a treatment called efgartigimod PH20 SC, which is a solution for injection. This treatment is being tested in children aged 2 to less than 18 years who have this condition. The purpose of the study is to confirm the appropriate dose of this treatment for young patients with Generalized Myasthenia Gravis.

Participants in the study will receive the treatment through a subcutaneous injection, which means it is injected under the skin. The study will monitor how the body processes the treatment and its effects on the disease. This includes checking the levels of certain proteins in the blood and observing any changes in the symptoms of Generalized Myasthenia Gravis. The study will also look at the safety of the treatment by recording any side effects and changes in health indicators like heart rate and blood tests.

The study will take place over a period of time, during which participants will have regular check-ups to assess their response to the treatment. The goal is to gather information that will help determine the best way to use efgartigimod PH20 SC in treating children with Generalized Myasthenia Gravis. This research is important for understanding how to manage this condition in younger patients and improve their quality of life.

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

    Joining the study

    Upon joining the study, the participant must provide written informed consent or assent, which indicates understanding of the study requirements.

    The participant must be between 2 and less than 18 years old and have a diagnosis of generalized myasthenia gravis confirmed by a physical examination and seropositivity for anti-acetylcholine receptor antibodies.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to ensure the participant has had an unsatisfactory response to previous treatments such as immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors.

    The participant must be on a stable dose of these medications for at least one month before the screening.

  3. Step 3

    Medication administration

    The participant will receive efgartigimod PH20 SC through a subcutaneous injection. The specific dosage and frequency will be determined during the study to confirm an appropriate dose for pediatric participants.

    The medication is provided as a solution for injection under the product name Vyvgart 1,000 mg.

  4. Step 4

    Monitoring and evaluation

    Throughout the study, the participant's efgartigimod serum concentrations will be monitored to analyze age and size dependency.

    Total IgG levels and anti-acetylcholine receptor antibody levels will be measured for pharmacokinetic and pharmacodynamic modeling.

  5. Step 5

    Safety and side effects

    The study will track the incidence and severity of any adverse events, including serious adverse events and those of special interest.

    Changes in vital signs, electrocardiograms, and clinical laboratory tests will be observed to ensure participant safety.

  6. Step 6

    Quality of life assessment

    The participant's quality of life will be evaluated using various tools, such as the Myasthenia Gravis Activities of Daily Living score and the Quality of Life in Neurological Disorders Questionnaire.

    Changes in fatigue levels and overall improvement will be assessed using the Clinical Global Impression of Improvement scale.

  7. Step 7

    Study duration

    The study is expected to continue until September 30, 2026, with recruitment starting on September 1, 2024.

    Participants will be involved in the study for its entire duration, with regular assessments and monitoring.

Who can join the trial?

5 criteria

  • The participant, or their legally authorized representative, must understand what the study involves and be able to give written permission to take part.
  • The participant must be between 2 and less than 18 years old at the time they agree to join the study.
  • The participant must have been diagnosed with **Generalized Myasthenia Gravis**, a condition confirmed by a physical exam and a positive test for specific antibodies called anti-acetylcholine receptor antibodies.
  • The participant must have tried treatments like immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but did not have a satisfactory response. They must be on a stable treatment plan for **Myasthenia Gravis** for at least one month before the study starts.
  • The participant must agree to use birth control as required by local rules. If they can have children, they must have a negative blood pregnancy test at the start and a negative urine pregnancy test before receiving the study medication.

Who cannot join the trial?

10 criteria

  • Patients who have a history of severe allergic reactions to any of the study medications.
  • Patients who are currently participating in another clinical trial.
  • Patients who have a serious infection that requires treatment with antibiotics.
  • Patients who have received a live vaccine within the last 4 weeks. A live vaccine is a type of vaccine that uses a weakened form of the germ that causes a disease.
  • Patients who have a history of drug or alcohol abuse within the past year.
  • Patients who have any other medical condition that the study doctors believe would make it unsafe for them to participate.
  • Patients who are pregnant or breastfeeding.
  • Patients who have had a major surgery within the last 3 months.
  • Patients who have a history of cancer, except for certain types of skin cancer that have been treated successfully.
  • Patients who have a history of heart problems, such as a heart attack or heart failure.
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Investigated drugs

Efgartigimod PH20 SC is a medication being studied for its effects on children and teenagers with generalized myasthenia gravis, a condition that causes muscle weakness. This medication is designed to help improve muscle strength by targeting specific parts of the immune system that are involved in the disease. The study aims to find the right dose for young patients to ensure it is safe and effective.

What is already known about the treatment

Efgartigimod PH20 SC – This medication is administered subcutaneously, meaning it is injected under the skin. It is currently being studied in clinical trials, particularly for its use in treating generalized myasthenia gravis in children aged 2 to less than 18 years. The main therapeutic indication is to manage symptoms of generalized myasthenia gravis, a condition that causes muscle weakness. Efgartigimod works by reducing the levels of certain antibodies that attack the body's own tissues, helping to improve muscle strength. It is classified as an immunomodulatory agent, which means it modifies the immune system's activity.

Investigated diseases

Generalized Myasthenia Gravis – This is a chronic autoimmune disorder that affects the communication between nerves and muscles, leading to muscle weakness. It occurs when the immune system mistakenly attacks the connections between nerves and muscles, specifically targeting the acetylcholine receptors. This disruption results in fluctuating muscle weakness and fatigue, which can affect various muscle groups, including those responsible for eye movement, facial expression, chewing, talking, and swallowing. The severity of muscle weakness can vary throughout the day and may worsen with activity. Symptoms often improve with rest, and the condition can affect individuals of any age, though it is more common in women under 40 and men over 60.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IVTrial ID2023-506159-12-00Protocol codeARGX-113-2207Estimated enrolment17 patientsSponsorArgenx

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