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Study on Efgartigimod for Early Treatment in Patients 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 disease that affects the communication between nerves and muscles, leading to muscle weakness. The study will use a treatment called efgartigimod, which is also known by its code name ARGX-113. Efgartigimod is given as a solution through an intravenous infusion, which means it is administered directly into the bloodstream through a vein.

The purpose of the study is to evaluate how effective and tolerable efgartigimod is when used early in the treatment of patients with generalized myasthenia gravis. Participants in the study will receive efgartigimod along with their usual treatment regimen, which may include a medication called prednisone. The study will monitor changes in the participants' ability to perform daily activities and other health indicators over several weeks.

Throughout the study, participants will have regular assessments to track their progress. These assessments will occur at various intervals, such as weeks 4, 6, 8, 16, and 28, to see how the treatment affects their condition over time. The study aims to understand how quickly efgartigimod can improve symptoms and how it impacts the overall quality of life for those with generalized myasthenia gravis. Additionally, the study will observe any side effects or adverse events that may occur during the treatment period.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial visit
Upon joining the study, you will attend an initial visit. During this visit, your eligibility will be confirmed based on specific criteria, such as age, diagnosis of generalized myasthenia gravis, and other health conditions.
A series of tests will be conducted, including a pregnancy test for women of childbearing potential, and you will be required to sign an informed consent form.
Step 2
Baseline assessment
A baseline assessment will be conducted to evaluate your current health status. This includes measuring your score on the myasthenia gravis activities of daily living (MG-ADL) scale, which helps assess the impact of the disease on your daily life.
Blood samples will be taken to measure levels of certain antibodies and other markers relevant to your condition.
Step 3
Treatment initiation
You will begin treatment with efgartigimod, administered as an intravenous infusion. This means the medication will be given through a vein in your arm.
The dosage is Vyvgart 20 mg/mL concentrate for solution for infusion. The frequency and duration of administration will be determined by the study protocol.
Step 4
Follow-up visits
Regular follow-up visits will be scheduled at weeks 4, 6, 8, 16, and 28. During these visits, your progress will be monitored, and assessments similar to the baseline will be repeated.
Your scores on the MG-ADL scale and other relevant scales will be recorded to track changes in your condition.
Step 5
Monitoring and adjustments
Throughout the study, your response to the treatment will be closely monitored. Adjustments to your treatment plan, including the dosage of any additional medications like prednisone, may be made based on your progress.
Blood tests will be conducted periodically to measure antibody levels and other health indicators.
Step 6
Completion of study
At the end of the study period, a final assessment will be conducted to evaluate the overall effectiveness and tolerability of the treatment.
You will be asked to provide feedback on your experience and any side effects you may have encountered during the trial.

Who can join the trial?

8 criteria

Adult patients who are 18 years or older, both men and women.
Have a confirmed diagnosis of myasthenia gravis by a neurologist. This means having symptoms that suggest myasthenia gravis and positive anti-AChR antibodies or a test that shows a problem at the connection between nerves and muscles.
Belong to Class II, III, or IV according to the Myasthenia Gravis Foundation of America (MGFA) at the time of the screening visit. This is a way to classify the severity of the disease.
Have a score of 5 or more points on the MG-ADL scale, which measures how the disease affects daily activities, with more than half of the points related to non-eye symptoms.
Have not received previous treatment that suppresses the immune system for myasthenia gravis, except for corticosteroids, which can be started during the study. These patients are called naïve patients.
Women who can have children must have a negative pregnancy test at the start, use birth control during the study and for 90 days after the last dose, and agree not to donate eggs during this time.
Men must agree to use adequate birth control and not donate sperm until the end of the study and for 90 days after the last drug infusion.
Must have signed an Informed Consent Form, which means they understand the study and agree to participate.

Who cannot join the trial?

4 criteria

Patients who do not have **generalized myasthenia gravis** cannot participate. This is a condition that affects the muscles and causes weakness.
Patients who do not have **anti-AChR antibodies** cannot participate. These are specific proteins in the blood related to myasthenia gravis.
Patients who are not within the specified age range cannot participate. The study is open to certain age groups only.
Patients who are part of a vulnerable population cannot participate. This includes groups that may need special protection in research.

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Bellvitge University Hospital
L'hospitalet De Llobregat, Spain

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Investigated drugs

Efgartigimod is a medication being studied for its potential to help people with a condition called generalized myasthenia gravis. This condition can cause muscle weakness and fatigue. Efgartigimod works by targeting and reducing certain antibodies in the body that are believed to contribute to the symptoms of myasthenia gravis. By lowering these antibodies, the medication aims to improve the ability of patients to perform daily activities and reduce the severity of their symptoms. The study is focused on understanding how effective and well-tolerated efgartigimod is when used early in the treatment of this condition.

Efgartigimod Alfa

What is already known about the treatment

Efgartigimod – Efgartigimod is administered as an intravenous infusion, meaning it is given directly into the bloodstream through a vein. It is currently being studied as an early treatment for patients with generalized myasthenia gravis, a condition that causes muscle weakness. The medication works by reducing the levels of certain antibodies that interfere with communication between nerves and muscles, helping to improve muscle function. Efgartigimod is classified as an immunomodulatory drug, which means it helps to regulate the immune system.

Investigated diseases

Generalized Myasthenia Gravis – Generalized Myasthenia Gravis is an autoimmune disorder characterized by weakness and rapid fatigue of voluntary muscles. It occurs when the immune system produces antibodies that block or destroy muscle receptor sites for a neurotransmitter called acetylcholine. This disruption prevents muscles from contracting properly, leading to muscle weakness. The condition often affects muscles that control eye and eyelid movement, facial expression, and swallowing. Muscle weakness tends to worsen with activity and improve with rest. Over time, the severity of symptoms can fluctuate, and they may become more pronounced during periods of physical or emotional stress.