Study on the Effects of Efgartigimod Alfa for Patients with Generalized Myasthenia Gravis

1 1 1 1

What is this study about?

This clinical trial is focused on studying a condition called Generalized Myasthenia Gravis (gMG), which is a chronic autoimmune disease that causes muscle weakness. The study is testing a treatment called efgartigimod alfa, which is given through an intravenous infusion, meaning it is administered directly into the bloodstream through a vein. The purpose of the study is to evaluate how different dosing schedules of efgartigimod alfa can maximize and maintain clinical benefits for patients with gMG.

Participants in the study will receive efgartigimod alfa in one of two dosing regimens. The study will compare a continuous regimen, where the medication is given every two weeks, to a cyclic regimen, where the medication is given in cycles. The study will monitor changes in the participants’ symptoms over time to determine which dosing schedule is more effective. The study will also track any side effects or adverse events that participants may experience during the trial.

The trial aims to provide valuable information on how to best use efgartigimod alfa to help manage symptoms of Generalized Myasthenia Gravis. By comparing different dosing regimens, researchers hope to find the most effective way to use this treatment to improve the quality of life for individuals living with this condition.

1 joining the study

Upon joining the study, the patient is required to provide signed informed consent, confirming understanding and agreement to follow the study protocol.

The patient must be at least 18 years old and have a confirmed diagnosis of generalized myasthenia gravis (gMG).

The patient should meet specific clinical criteria and have a Myasthenia Gravis – Activities of Daily Living (MG-ADL) total score of 5 or more, with more than 50% of the score due to non-ocular symptoms.

2 treatment administration

The patient will receive efgartigimod alfa, administered intravenously as a solution for infusion.

The dosage is 10 mg per kg of body weight, given in a continuous regimen every two weeks (q2w) or in a cyclic regimen, depending on the study group assignment.

3 monitoring and assessments

Throughout the study, the patient’s MG-ADL total score will be monitored to assess changes from baseline.

The primary endpoint is the mean change in the MG-ADL total score from baseline during weeks 1 through 21.

Secondary endpoints include the incidence and severity of adverse events, changes in laboratory test results, vital signs, and electrocardiogram results.

4 study duration

The study is expected to continue until May 31, 2026.

The patient will participate in regular visits and assessments throughout the study period to evaluate the clinical efficacy of the treatment.

Who Can Join the Study?

  • Must be able to provide signed informed consent, which means agreeing to participate after understanding the study details.
  • Must be at least 18 years old at the time of signing the informed consent.
  • Must have been diagnosed with Generalized Myasthenia Gravis (gMG), a condition confirmed by a physical exam and a test showing the presence of specific antibodies called anti-acetylcholine receptor antibodies (AChR-Abs).
  • Must meet the clinical criteria set by the Myasthenia Gravis Foundation of America (MGFA) for class II, III, or IV, which are categories used to describe the severity of the condition.
  • Must have a Myasthenia Gravis – Activities of Daily Living (MG-ADL) total score of 5 or higher at the time of the study, with more than half of the score due to symptoms that are not related to the eyes.
  • Can continue current treatment for gMG, which may include nonsteroidal immunosuppressive drugs (NSIDs), steroids, and/or acetylcholinesterase (AChE) inhibitors. If taking corticosteroids or NSIDs, the dose must be stable for at least 1 month before the study.
  • Must agree to use contraceptive measures as per local regulations. Women who can have children must have a negative blood pregnancy test at the start and a negative urine pregnancy test before receiving the study drug.

Who Cannot Join the Study?

  • Patients who have a different condition than Generalized Myasthenia Gravis (gMG) cannot participate. This is a condition that affects the muscles and causes weakness.
  • Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.
  • Patients who do not meet the specific health requirements set by the study cannot participate. These requirements ensure the safety and effectiveness of the treatment being tested.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Medical University Of Vienna Vienna Austria
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Centre Hospitalier Universitaire De Lille Lille France
Katholieke Universiteit te Leuven Leuven Belgium
Galen Clinic Lublin Poland

Other Sites

Site Name City Country Status
Charite Universitaetsmedizin Berlin KöR Berlin Germany
IRCCS Ospedale Policlinico San Martino Genoa Italy
Universita’ Di Pisa Pisa Italy
Universidade De Santiago De Compostela Santiago De Compostela Spain
Centre Hospitalier Universitaire De Nice Nice France
Hopital Beaujon Clichy France
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Uygzrifolufrjrmythpdb Ecqzy Azp Essen Germany
Mdnqvmxeb i Pqqzqwuep Lhnvvsn Ssehto Pcqcoavtcbx Cracow Poland
Aipkesgpxu Pvncogqv Hbikmtxn Dm Meppcjgoj Marseille France
Hcshzyfo Dd Lk Syljq Cksv I Sxqg Phi Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
25.10.2021
Belgium Belgium
Not recruiting
25.10.2021
France France
Not recruiting
25.10.2021
Germany Germany
Not recruiting
25.10.2021
Italy Italy
Not recruiting
25.10.2021
Poland Poland
Not recruiting
25.10.2021
Spain Spain
Not recruiting
25.10.2021

Trial locations

Investigated drugs:

Efgartigimod is a medication being studied for its effectiveness in treating patients with generalized myasthenia gravis. It is administered intravenously and works by modulating the immune system to help reduce the symptoms of this autoimmune condition. The trial is exploring different dosing schedules to determine the best way to maximize and maintain the clinical benefits for patients.

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 controlling eye movements, facial expressions, and swallowing. The severity of symptoms can vary widely among individuals and may worsen with physical activity or stress. Over time, the condition can lead to more persistent muscle weakness, impacting daily activities and quality of life.

Trial ID:
2024-510932-36-00
Protocol code:
ARGX-113-2003
NCT ID:
NCT04980495
Trial Phase:
Therapeutic confirmatory (Phase III)

Other Trials to Consider

  • Phase 2 Randomized Study of Povetacicept to Assess Safety and Effectiveness in Adults with Generalized Myasthenia Gravis

    Recruiting

    Poland
  • A study testing how inebilizumab works and how safe it is in children and teenagers aged 2 to 17 years with generalized myasthenia gravis

    Recruiting

    1 1 1
    Investigated drugs:
    France Italy Poland Spain