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Study on the Safety and Effectiveness of Gefurulimab for Adults 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 the disease known as Generalized Myasthenia Gravis, a condition that causes muscle weakness due to the immune system attacking the communication between nerves and muscles. The study will evaluate a treatment called Gefurulimab, which is administered as a solution for injection. This treatment is being compared to a placebo to assess its effectiveness and safety in managing the symptoms of this disease.

The purpose of the study is to determine how well Gefurulimab works in improving the daily living activities of adults with Generalized Myasthenia Gravis. Participants in the study will receive either the treatment or a placebo and will be monitored over a period of time to observe any changes in their condition. The study aims to see if there is a significant improvement in the ability to perform daily activities and a reduction in muscle weakness.

Throughout the study, participants will undergo regular assessments to track their progress and any changes in their symptoms. The study is designed to provide valuable information on the potential benefits of Gefurulimab for individuals living with Generalized Myasthenia Gravis, contributing to the understanding of how this treatment can help manage the disease.

The research process

The trial runs in 4 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 is required to meet specific criteria, including being 18 years or older and having a diagnosis of generalized myasthenia gravis with muscle weakness, as defined by the Myasthenia Gravis Foundation of America (MGFA) Class II, III, or IV.

    A positive serological test for autoantibodies against the acetylcholine receptor (AChR) is also necessary.

  2. Step 2

    Treatment phase

    During the treatment phase, participants receive either the investigational drug gefurulimab or a placebo. The medication is administered as a subcutaneous injection, which means it is injected under the skin.

    The specific dosage and frequency of administration are determined by the study protocol, which is designed to evaluate the safety and effectiveness of the treatment.

  3. Step 3

    Evaluation of treatment effectiveness

    The primary goal is to assess changes in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at week 26. This score helps measure the impact of the treatment on daily activities.

    Secondary evaluations include changes in the Quantitative Myasthenia Gravis (QMG) total score and the Myasthenia Gravis Composite (MGC) total score, both at week 26, as well as the percentage of responders based on score reductions.

  4. Step 4

    Completion of the study

    The study is expected to conclude by July 31, 2027. Participants will have completed all required assessments and treatments by this time.

    Final evaluations will be conducted to determine the overall effectiveness and safety of the treatment.

Who can join the trial?

3 criteria

  • Must be at least 18 years old at the time of signing the informed consent.
  • Must have a diagnosis of **Myasthenia Gravis (MG)**, which is a condition that causes muscle weakness. The muscle weakness should be generalized, meaning it affects multiple areas of the body, and should meet the clinical criteria defined by the **Myasthenia Gravis Foundation of America (MGFA)** as Class II, III, or IV.
  • Must have a positive serological test, which is a blood test, showing the presence of **autoantibodies against acetylcholine receptor (AChR)**. These are specific proteins in the blood that are involved in MG.

Who cannot join the trial?

5 criteria

  • Patients who do not have **Generalized Myasthenia Gravis** with autoantibodies against the **acetylcholine receptor (AChR)** cannot participate. This means the study is only for those with a specific type of muscle weakness condition.
  • Patients who are not within the specified age range cannot participate. The study is for certain age groups only.
  • Patients who are not part of the specified clinical trial groups cannot participate. This means the study is for specific groups of people.
  • Both **male** and **female** patients can participate, but those who do not meet other criteria cannot join.
  • Patients who are considered part of a **vulnerable population** may not be eligible. This refers to groups who might need special protection or care.
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Investigated drugs

ALXN1720 is a medication being studied for its potential to treat adults with generalized myasthenia gravis, a condition that causes muscle weakness. The trial aims to evaluate how effective and safe this medication is in improving symptoms of the disease.

What is already known about the treatment

ALXN1720 – This medication is administered through an injection, typically under the skin. It is currently being studied in clinical trials for its effectiveness and safety in treating generalized myasthenia gravis, a condition characterized by muscle weakness due to antibodies against acetylcholine receptors. ALXN1720 works by targeting specific proteins involved in the immune response, helping to reduce the attack on these receptors. It is classified as a monoclonal antibody, a type of medication designed to interact with specific molecules in the body to alter disease processes.

Investigated diseases

Generalized Myasthenia Gravis with autoantibodies against acetylcholine receptor (AChR) – This is a chronic autoimmune disorder where the body's immune system mistakenly attacks the communication between nerves and muscles. It specifically targets the acetylcholine receptors, which are crucial for muscle contraction. As a result, individuals experience muscle weakness that can vary in severity and may worsen with activity. Commonly affected muscles include those controlling the eyes, face, throat, and limbs. The condition can lead to difficulties in speaking, swallowing, and breathing. Symptoms often fluctuate, with periods of improvement and worsening.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-508284-77-00Protocol codeALXN1720-MG-301Estimated enrolment243 patientsSponsorAlexion Pharmaceuticals Inc.

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