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Study on the Long-Term Safety of Zilucoplan in Children with Generalized Myasthenia Gravis

Verified siteRegistered 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 the long-term safety and effects of a medication called zilucoplan sodium in children with a condition known as generalized myasthenia gravis. Generalized myasthenia gravis is a chronic autoimmune disease that causes muscle weakness. The medication, zilucoplan sodium, is administered as a solution for injection under the skin using a pre-filled syringe. The study aims to understand how safe and tolerable zilucoplan sodium is when used over an extended period.

Participants in this study will receive zilucoplan sodium through daily injections for up to 52 weeks. The study will monitor the occurrence of any side effects or adverse events that may arise during this period. Additionally, the study will assess the levels of zilucoplan sodium in the blood and its impact on certain biological markers related to the disease. The study will also evaluate how the medication affects the participants' ability to perform daily activities and their overall muscle strength.

This study is an extension of a previous research project involving zilucoplan sodium, and it is designed to provide further insights into the medication's long-term use in treating generalized myasthenia gravis in children. Participants who have completed the earlier study and for whom continued treatment is deemed beneficial by their healthcare provider may be eligible to participate in this extension study.

The research process

The trial runs in 5 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, you will begin a treatment period that lasts for an additional 52 weeks. This is to assess the long-term safety and tolerability of the medication zilucoplan.

    You will receive zilucoplan through a subcutaneous injection, which means it is injected under the skin. This will be done once daily.

  2. Step 2

    Medication administration

    The medication, zilucoplan sodium, is provided in a solution for injection using a pre-filled syringe. You will administer this injection yourself or with the help of a caregiver.

    It is important to follow the daily schedule for the injections to ensure the effectiveness of the treatment.

  3. Step 3

    Monitoring and assessments

    Throughout the study, you will be monitored for any side effects or adverse reactions to the treatment. This includes any new symptoms or changes in your health.

    Regular assessments will be conducted to measure the concentration of zilucoplan in your blood, as well as other health indicators such as the Myasthenia Gravis Activities of Daily Living (MG ADL) score and the Quantitative Myasthenia Gravis (QMG) score.

  4. Step 4

    Vaccinations

    If necessary, you may be required to receive booster vaccinations against meningococcal infections during the study. This will be determined based on local healthcare guidelines and your specific health needs.

  5. Step 5

    Completion of the study

    At the end of the 52-week period, a final assessment will be conducted to evaluate the overall effects of the treatment.

    The study aims to gather information on the long-term safety and effectiveness of zilucoplan in treating generalized myasthenia gravis in pediatric patients.

Who can join the trial?

4 criteria

  • The participant must have completed the previous study called MG0014 according to its rules, and the study doctor must believe that continuing treatment with zilucoplan is beneficial for the participant.
  • The participant must agree to receive additional vaccinations to protect against meningococcal infections, if needed, based on local healthcare guidelines.
  • The study is open to both male and female participants.
  • The study includes participants who are considered part of a vulnerable population, which means they may need extra protection or care.

Who cannot join the trial?

10 criteria

  • Patients who do not have **Generalized Myasthenia Gravis** cannot participate.
  • Patients who are not within the age range specified for the study cannot participate.
  • Patients who are not able to receive a daily injection under the skin (subcutaneous injection) cannot participate.
  • Patients who are not able to commit to the study duration of 52 weeks cannot participate.
  • Patients who have any other medical conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are participating in another clinical trial cannot participate.
  • Patients who have had a recent major surgery or are planning to have surgery during the study cannot participate.
  • Patients who have a history of drug or alcohol abuse cannot participate.
  • Patients who have any allergies to the study medication or its ingredients cannot participate.
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Investigated drugs

Zilucoplan is a medication being studied for its potential to help people with a condition called generalized myasthenia gravis. This condition affects the muscles, making them weak and tired. Zilucoplan is designed to be injected under the skin once a day. The goal of this medication is to improve muscle strength and reduce symptoms by targeting specific parts of the immune system that are involved in this condition. In this trial, researchers are looking at how safe and tolerable zilucoplan is when used over a long period, especially in children who have this condition.

What is already known about the treatment

Zilucoplan – Zilucoplan is administered as a subcutaneous injection, meaning it is injected under the skin, and is given once daily. It is currently being studied in clinical trials to assess its long-term safety and effectiveness, particularly in children with a condition called generalized myasthenia gravis, which affects muscle strength. The medication works by targeting and inhibiting specific proteins in the immune system that are involved in the disease process, helping to reduce symptoms. Zilucoplan is classified as an immunomodulatory drug, which means it helps to regulate or normalize the immune system's activity.

Investigated diseases

Generalized Myasthenia Gravis – 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 weakness in the eye muscles, leading to drooping eyelids and double vision. Over time, it can progress to involve other muscle groups, including those responsible for facial expressions, swallowing, and limb movements. The severity of muscle weakness can vary throughout the day and may worsen with activity.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2022-502073-42-00Protocol codeMG0015Estimated enrolment10 patientsSponsorUCB Biopharma

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