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Study on Long-Term Safety and Effectiveness of Zilucoplan in Adults 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 effectiveness of a treatment called zilucoplan sodium for individuals with generalized myasthenia gravis. Generalized myasthenia gravis is a chronic autoimmune disease that causes muscle weakness. The treatment being tested, zilucoplan sodium, is administered as a solution for injection using a pre-filled syringe. It is designed to be self-administered by patients through a subcutaneous injection, which means it is injected under the skin.

The purpose of this study is to evaluate how safe and tolerable zilucoplan sodium is for patients who have already participated in a previous study involving this medication. Participants will receive the treatment over an extended period to monitor its effects and any potential side effects. The study will not involve any new medications or diseases, focusing solely on zilucoplan sodium and generalized myasthenia gravis.

Throughout the study, participants will be regularly assessed to ensure their well-being and to gather information on how the treatment impacts their daily activities and quality of life. The study aims to provide valuable insights into the long-term use of zilucoplan sodium for managing symptoms of generalized myasthenia gravis.

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

    Participation begins after completing a previous study involving zilucoplan.

    Informed consent is required, which involves signing and dating a consent form.

    Vaccination against meningococcal infections is necessary, including a quadrivalent vaccine and, if available, a serotype B vaccine. A booster may be needed based on local guidelines.

  2. Step 2

    Initial health checks

    Female participants of childbearing potential must undergo a urine pregnancy test within 24 hours before the first dose of the study drug.

    All participants must agree to use effective contraception during the study and for 40 days after the last dose.

  3. Step 3

    Medication administration

    Zilucoplan sodium is administered as a solution for injection using a pre-filled syringe.

    The route of administration is subcutaneous, meaning the injection is given under the skin.

  4. Step 4

    Monitoring and assessments

    The primary focus is on the incidence of treatment-emergent adverse events (TEAEs).

    Secondary assessments include changes from baseline to Week 12 in various scores related to myasthenia gravis, such as the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score, Quantitative Myasthenia Gravis (QMG) Score, Myasthenia Gravis Composite (MGC) Score, and Myasthenia Gravis – Quality of Life revised (MG-QOL15r) Score.

  5. Step 5

    Study duration

    The study is an open-label extension, meaning it continues from a previous study and participants know they are receiving zilucoplan.

    The estimated end date for the study is May 20, 2026.

Who can join the trial?

5 criteria

  • You must have completed a previous study involving the drug zilucoplan.
  • You need to be able to understand and agree to the study details, which includes signing a form called the informed consent form.
  • You must have received a vaccine that protects against a type of bacteria called meningococcal, which can cause serious infections. This includes a specific vaccine for a type called serotype B, if available. If you have been vaccinated before, you might need a booster shot, which is an additional dose to keep you protected.
  • If you are a woman who can have children, you must have a negative pregnancy test within 24 hours before taking the first dose of the study drug.
  • If you are a woman who can have children or a man who can father children, you must agree to use effective birth control during the study and for 40 days after the last dose of the study drug. This is to prevent pregnancy during the study.

Who cannot join the trial?

2 criteria

  • Patients who have not completed a previous clinical study with the medication called zilucoplan.
  • Patients who are part of a vulnerable population, which means groups of people who might need special protection or care.
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Investigated drugs

Zilucoplan is a medication being studied for its long-term safety and tolerability in patients with generalized myasthenia gravis (gMG). It is designed to help manage the symptoms of this autoimmune disorder, which affects the communication between nerves and muscles, leading to muscle weakness. This study is an extension of previous research to further understand how well patients tolerate the medication over an extended period.

What is already known about the treatment

Zilucoplan – Zilucoplan is administered as a subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials, specifically in a Phase 3 study, to assess its long-term safety and tolerability for treating generalized myasthenia gravis (gMG). This medication is primarily indicated for individuals with gMG, a chronic autoimmune neuromuscular disorder. Zilucoplan works by inhibiting the complement system, a part of the immune system, which helps reduce the attack on the neuromuscular junction, thereby alleviating symptoms. It is classified as a complement inhibitor in pharmacology.

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, and swallowing. The severity of symptoms can vary throughout the day, often worsening with activity and improving with rest. Over time, the condition may lead to more persistent muscle weakness, impacting daily activities and quality of life.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-512399-37-00Protocol codeRA101495-02.302Estimated enrolment190 patientsSponsorUCB Biopharma

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