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

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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, a chronic autoimmune disease that causes muscle weakness. The trial is testing a treatment called efgartigimod alfa, which is given as an intravenous infusion. The purpose of the study is to find the best dose of efgartigimod alfa for children aged 2 to less than 18 years who have this condition.

Participants in the study will receive efgartigimod alfa through a vein, and the study will monitor how the body processes the medication and how it affects the disease. The study will also look at the safety of the treatment and any side effects that may occur. The trial aims to provide evidence of how well the treatment works in children with Generalized Myasthenia Gravis.

Throughout the study, researchers will collect information on the levels of certain proteins in the blood, such as total IgG and anti-acetylcholine receptor antibodies, which are important in understanding the disease and the treatment's effects. The study will also assess changes in the participants' quality of life and any improvements in their symptoms. The trial is expected to continue until March 2025.

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 trial

    Upon joining the trial, the participant or their legally authorized representative must provide written informed consent or assent. This indicates an understanding of the trial requirements and a willingness to comply with the procedures.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to confirm the diagnosis of generalized myasthenia gravis (gMG) and to ensure the participant meets the clinical criteria defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.

    Participants must have a positive serologic test for anti-acetylcholine receptor antibodies (AChR-Ab).

  3. Step 3

    Treatment administration

    The participant receives efgartigimod alfa through an intravenous infusion. The product used is Vyvgart 20 mg/mL concentrate for solution for infusion.

    The dosage and frequency are adjusted based on age and size to determine the optimum dose.

  4. Step 4

    Monitoring and evaluation

    Throughout the trial, the participant's response to the treatment is monitored. This includes measuring efgartigimod concentrations and evaluating pharmacokinetic and pharmacodynamic parameters.

    The levels of total immunoglobulin G (IgG) and AChR-Ab are assessed to understand the treatment's impact.

  5. Step 5

    Safety assessments

    The incidence and severity of adverse events (AEs) are recorded, including serious adverse events (SAEs) and adverse events of special interest (AESIs).

    Regular laboratory tests, physical examinations, vital sign measurements, and electrocardiograms (ECGs) are conducted to ensure participant safety.

  6. Step 6

    Quality of life evaluation

    The participant's quality of life is assessed using various scores, such as the MG-ADL total score, total QMG score, and EQ-5D-Y score.

    Changes in the Quality of Life in Neurological Disorders (Neuro-QoL) Pediatric Fatigue Score are also evaluated.

  7. Step 7

    Trial completion

    The trial is estimated to end by March 31, 2025. Upon completion, the data collected will be analyzed to confirm the age-adjusted optimum dose of efgartigimod and provide evidence for a treatment response.

Who can join the trial?

6 criteria

  • The participant or their legally authorized representative must be able to understand the trial requirements and provide written consent or agreement to participate. They must also be willing and able to follow the trial procedures.
  • The participant must be a boy or girl between 2 to less than 18 years old at the time of giving consent or agreement to participate.
  • The participant must have a diagnosis of **Generalized Myasthenia Gravis (gMG)**, which is a condition affecting the muscles, with confirmed documentation.
  • The participant must meet the clinical criteria set by the **Myasthenia Gravis Foundation of America (MGFA)**, specifically classes II, III, and IVa. These classes describe the severity of the condition.
  • The participant should have had an unsatisfactory response, meaning the treatment did not work well or caused problems, to medications like immunosuppressants, steroids, or **AChE inhibitors** (medicines that help with muscle strength). They should also be on a stable treatment plan for gMG for a sufficient time before the trial starts.
  • The participant must have a positive result on a blood test for **anti-AChR antibodies** at the time of screening. For younger participants weighing less than 15 kg, past test results can be used.

Who cannot join the trial?

10 criteria

  • Patients who have a history of severe allergic reactions to the study medication.
  • Patients who are currently participating in another clinical trial.
  • Patients with a history of drug or alcohol abuse within the past year.
  • Patients who are pregnant or breastfeeding.
  • Patients with uncontrolled high blood pressure.
  • Patients with severe heart disease.
  • Patients with active infections that require treatment.
  • Patients with a history of cancer within the past five years, except for certain skin cancers.
  • Patients with a history of certain autoimmune diseases, which are conditions where the body's immune system attacks its own tissues.
  • Patients who have received certain treatments that affect the immune system within the past three months.
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Investigated drugs

Efgartigimod is a medication being studied for its effects in children with generalized myasthenia gravis. This trial aims to understand how the body processes the medication, how it affects the body, and its safety and effectiveness. The goal is to find the best dose for children aged 2 to less than 18 years.

What is already known about the treatment

Efgartigimod – This medication is administered intravenously and is currently being studied in clinical trials for its effectiveness in treating generalized myasthenia gravis, particularly in children aged 2 to less than 18 years. It is in the investigational stage, with ongoing research to determine the optimal dosage for different age groups. Efgartigimod works by targeting and reducing the levels of certain antibodies that contribute to the symptoms of myasthenia gravis. It is classified as an immunomodulatory agent, which means it helps to regulate 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 body's own tissues, specifically targeting the acetylcholine receptors at the neuromuscular junction. This interference prevents muscles from contracting properly, resulting in symptoms such as drooping eyelids, difficulty swallowing, and general fatigue. The severity of muscle weakness can vary throughout the day, often worsening with activity and improving with rest. Over time, the condition can affect various muscle groups, including those responsible for breathing and limb movement.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IVTrial ID2024-513854-31-00Protocol codeARGX-113-2006Estimated enrolment19 patientsSponsorArgenx

sourced from the EU Clinical Trials Register and site verification

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