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Study on Zilucoplan Sodium 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 is specifically looking at children aged 2 to less than 18 years who have this condition. The treatment being tested is a medication called zilucoplan sodium, which is given as an injection under the skin. The purpose of the study is to understand how zilucoplan moves through the body over time and to assess its safety in children with generalized myasthenia gravis.

Participants in the study will receive zilucoplan sodium once a day for a period of time. The study will monitor how the medication affects the body and any side effects that may occur. The study will also look at changes in the participants' ability to perform daily activities and their overall quality of life. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of zilucoplan sodium.

The study aims to gather information on how zilucoplan sodium can help manage symptoms of generalized myasthenia gravis in children and to ensure it is safe for use in this age group. The results will help determine if this treatment can be a beneficial option for managing this condition in young patients.

The research process

The trial runs in 6 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 be between 2 and less than 18 years old and have a confirmed diagnosis of generalized myasthenia gravis (gMG).

    The participant should have a positive serologic test result for acetylcholine receptor antibodies and meet specific clinical criteria.

  2. Step 2

    Vaccination requirement

    Participants must have documented vaccination against meningococcal infections within the last three years.

    If not fully vaccinated, appropriate antibiotic treatment is required until at least two weeks after the initial dose of the vaccine.

  3. Step 3

    Medication administration

    The participant will receive zilucoplan sodium as a solution for injection.

    The medication is administered subcutaneously, which means it is injected under the skin, once daily.

  4. Step 4

    Monitoring and assessments

    Plasma concentrations of zilucoplan will be measured at Week 4 (Day 29).

    Changes from baseline in specific blood components will also be assessed at this time.

  5. Step 5

    Safety evaluations

    The occurrence of any treatment-related adverse events will be monitored throughout the study.

    Serious adverse events and any events leading to withdrawal from the study will be recorded.

  6. Step 6

    Study duration

    The study is expected to continue until December 4, 2026.

    Recruitment for the study is estimated to start on December 6, 2023.

Who can join the trial?

6 criteria

  • The participant must be between 2 and less than 18 years old at the time of signing the consent form.
  • The participant must have a diagnosis of generalized myasthenia gravis (gMG), which is a condition confirmed by a previous positive blood test for the acetylcholine receptor (AChR).
  • The participant must meet the criteria set by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at the time of screening. This classification helps to determine the severity of the condition.
  • For participants aged 12 to less than 18 years, they must have a total score of 6 or more on the MG-activities of daily living (MG-ADL) scale, which measures how the condition affects daily activities.
  • For children aged 2 to less than 12 years, there must be documented weakness in at least one limb, neck, or bulbar muscle, which are muscles involved in speech and swallowing, at the time of screening.
  • The participant must have documented vaccination against meningococcal infections within the last 3 years before the study starts. If not fully vaccinated, they must receive appropriate preventive antibiotic treatment until at least 2 weeks after the initial dose of the vaccine(s).

Who cannot join the trial?

10 criteria

  • Patients who have any other serious health conditions that could interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent surgery or are planning to have surgery during the study period.
  • Patients who have a history of allergic reactions to similar medications.
  • Patients who are unable to follow the study procedures or attend the required visits.
  • Patients who are taking medications that could interfere with the study drug.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who are pregnant or breastfeeding.
  • Patients who have an infection that requires treatment with antibiotics.
  • Patients who have a history of certain heart conditions.
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Investigated drugs

Zilucoplan is a medication being studied for its effects on children and teenagers with a condition called generalized Myasthenia Gravis. This condition affects the muscles, making them weak and tired. Zilucoplan is given as an injection under the skin once a day. The study aims to understand how the body processes this medication and how it affects the disease. Researchers are also looking at how safe and tolerable the medication is for young patients.

What is already known about the treatment

Zilucoplan – Zilucoplan is administered through a subcutaneous injection, typically given once daily. It is currently being studied in clinical trials, particularly for its effects on pediatric patients with generalized Myasthenia Gravis, a condition characterized by muscle weakness. The medication works by inhibiting the complement system, a part of the immune system, which helps reduce the attack on the neuromuscular junctions. Zilucoplan is classified as a complement inhibitor and is being evaluated for its safety, tolerability, and effectiveness in managing symptoms of this autoimmune disorder.

Investigated diseases

Generalized Myasthenia Gravis – Generalized Myasthenia Gravis 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 receptors that receive nerve signals. This 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 symptoms can vary widely among individuals and may worsen with physical activity or stress. The condition is characterized by periods of exacerbation and remission, where symptoms may improve or worsen over time.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IVTrial ID2022-502072-23-00Protocol codeMG0014Estimated enrolment10 patientsSponsorUCB Biopharma

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