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Efficacy of efgartigimod alfa as first‑line add‑on to IV methylprednisolone for moderate‑to‑severe attacks in patients with demyelinating diseases

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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study focuses on demyelinating diseases of the central nervous system, a group of conditions where the protective covering of nerve fibers is damaged, leading to problems such as weakness, vision loss, or coordination difficulty. It includes people who have had a first episode called CIS, the more common form known as RRMS, as well as disorders identified by specific antibodies such as AQP4+ NMOSD and MOGAD. All participants receive the standard anti‑inflammatory steroid intravenous methylprednisolone, and the trial tests whether adding an experimental antibody‑removing drug called efgartigimod, which works by targeting the protein FcRn, leads to better recovery compared with a placebo. The purpose of the study is to determine if the new drug improves the chance of complete remission after a moderate‑to‑severe attack.

After a qualifying attack, participants are randomly assigned to receive either the experimental infusion or the placebo, both given through a vein. They continue the usual steroid treatment and are followed for about three months, with visits to check vision, strength, walking speed, hand dexterity, and overall disability using simple tests and questionnaires. The study records whether additional rescue treatments are needed, how quickly symptoms improve, and any side effects, allowing researchers to see if the added drug provides a meaningful benefit.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization and start of treatment

    After you join the study you are assigned to receive either efgartigimod or placebo without knowing which one you receive.

    On the same day you also receive the standard therapy called intravenous methylprednisolone (ivmp) as the first‑line attack treatment.

  2. Step 2

    Baseline assessments

    Before the first infusion a series of examinations are performed, including neurological function scores, visual acuity tests, and blood samples for biomarkers.

    The results form the reference point for all later comparisons.

  3. Step 3

    First infusion of study medication

    You receive a single intravenous infusion of the study medication.

    If you are in the active group the infusion contains efgartigimod at a dose of 2400 (dose unit not specified).

    If you are in the control group the infusion contains placebo with no active drug.

    The infusion is given on day 0, the same day as the ivmp infusion.

  4. Step 4

    Day 4 follow‑up

    A safety visit is scheduled four days after the first infusion.

    Blood samples are taken to measure immunological biomarkers such as total igg, complement proteins, and neuro‑degenerative markers.

  5. Step 5

    Day 10 assessment

    Clinical evaluation is performed to determine whether you have achieved complete remission of neurological deficits without needing rescue therapy.

    Visual acuity and other functional tests are repeated.

    If the attack is not controlled, rescue therapies such as plasmapheresis or immunoadsorption may be considered.

  6. Step 6

    Day 21 follow‑up

    Additional safety and biomarker assessments are carried out.

    Neurological scores are recorded again to track progress.

  7. Step 7

    Day 28 assessment

    A comprehensive visit is conducted to evaluate remission status, quality‑of‑life questionnaires, and visual function.

    The need for rescue therapy is reviewed; patients who have not required rescue therapy are noted for the secondary endpoint.

  8. Step 8

    Day 84 final evaluation

    The primary endpoint is assessed by determining the proportion of patients who have complete remission of neurological deficits at day 84 without rescue therapy.

    Final safety assessments, biomarker measurements, and quality‑of‑life surveys are completed.

    The cumulative dose of ivmp received during the trial is recorded.

Who can join the trial?

8 criteria

  • You must be 18 years old or older, and any gender is accepted.
  • You need to have had a recent neurological attack that matches one of these conditions: CIS (first episode of demyelination), RRMS (relapsing‑remitting multiple sclerosis), AQP4+ NMOSD (neuromyelitis optica spectrum disorder with AQP4 antibodies), or MOGAD (MOG antibody disease). These are different types of demyelinating diseases.
  • You must sign a written consent form (or have an impartial witness sign) and be able to follow the study’s procedures.
  • The first dose of the study medication (called the investigational medicinal product, or IMP) must be started within 10 days after your attack began and no later than the fifth dose of the standard steroid treatment (IVMP).
  • You need to have a record (or be able to assess) of your disability level before the attack using the Expanded Disability Status Scale (EDSS), including any specific neurological deficits (functional system scores, FSS), and, if you had optic neuritis, a measurement of your visual sharpness called high‑contrast visual acuity (HCVA).
  • Your pre‑attack EDSS score must be 6.0 or lower.
  • During the current attack, your EDSS score must be between 3.0 and 7.5.
  • The attack must be at least moderate in severity. This means you must have at least one of the following problems at screening: a noticeable increase in weakness or coordination (pyramidal, brainstem, or cerebellar function) with a change of more than 2.0 points and a minimum score of 3.0; a significant change in sensory function with a change of more than 2.0 points and a minimum score of 4.0; or, if you have optic neuritis, a visual acuity of 20/200 or worse (equivalent to ≥1.0 logMAR) or a drop of at least two lines on the ETDRS eye chart.

Who cannot join the trial?

13 criteria

  • If you are having a current attack that mainly affects the brain (called a cerebral functional system score problem) at the time of screening, you cannot join the study.
  • If you have used any of the following treatments recently, you are excluded: anti‑FcRn therapy within the past 3 months; high‑dose steroids (IVMP), plasma exchange, immunoadsorption, or any intravenous, intramuscular or subcutaneous immunoglobulin (IgG) within the past 4 weeks (except up to 5 doses of IVMP for the current attack); or any CAR T‑cell therapy or hematopoietic stem cell transplant before screening.
  • If you do not have a recent (within 12 months) best‑corrected eye vision test and you have any serious eye disease such as retinal disease, cataract, or glaucoma in the eye affected by the attack, you cannot participate. (Being born with color‑blindness does not disqualify you.)
  • If you have an active or uncontrolled infection—bacterial, fungal, or viral—including a positive HIV test with AIDS‑defining illness or a CD4 count ≤200 cells/mm³, an active hepatitis B infection, or a positive test for hepatitis C, you are excluded.
  • If you have a known condition called common variable immunodeficiency (a disorder where the immune system does not work properly), you cannot join.
  • If you have had cancer in the past, you may be excluded unless the cancer was treated and you have been cancer‑free for at least 3 years. Exceptions that may be allowed at any time include basal cell or squamous cell skin cancer, carcinoma in situ of the cervix or breast, and very early‑stage prostate cancer (stage T1a or T1b).
  • If you received a live or live‑attenuated vaccine (such as measles‑mumps‑rubella or yellow fever) less than 4 weeks before screening, you cannot participate. Inactivated vaccines are allowed, but they must not be given within 48 hours before the study drug infusion.
  • If you are pregnant, breastfeeding, or plan to become pregnant during the study period, you are not eligible.
  • If you have severe kidney disease defined by an estimated glomerular filtration rate (eGFR) less than 30 mL/min/1.73 m² at screening, you cannot take part.
  • If you are known to be allergic (have hypersensitivity) to the study drug efgartigimod, the steroid methylprednisolone, prednisone, or any of the ingredients in these medicines, you are excluded.
  • If you have another serious medical condition, have had major surgery within the past 3 months, or are planning to have major surgery during the study, you cannot participate.
  • If your doctor believes any other medical problem would make it difficult to accurately assess your symptoms or would place you at undue risk, you will be excluded.
  • If you are currently enrolled in another clinical trial that uses investigational drugs or devices, or if you have been in such a trial within the past 30 days (or within five half‑lives of the other study drug, whichever is longer), you cannot join this study.
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Investigated drugs

  • Efgartigimod

    is a medication that targets a protein called FcRn, which helps protect certain antibodies in the body. By blocking this protein, efgartigimod helps lower the level of harmful antibodies that can cause attacks in demyelinating diseases of the central nervous system. In the trial, it is given through an intravenous (IV) infusion and is added on to the usual steroid treatment to see if it improves recovery during moderate‑to‑severe attacks.

  • Intravenous methylprednisolone (IVMP)

    is a strong anti‑inflammatory steroid that is routinely used to treat attacks of conditions like multiple sclerosis, NMOSD, or MOG‑associated disease. It is delivered by IV infusion and works quickly to reduce swelling and immune activity in the nervous system. In this study, it serves as the standard therapy that all patients receive, and the researchers are testing whether adding efgartigimod provides additional benefit.

What is already known about the treatment

EFGARTIGIMOD ALFA - This medication is administered by a slow intravenous infusion, where the drug is mixed in a bag of fluid and given through a vein. It is already approved in several countries for treating a muscle‑weakening disease called generalized myasthenia gravis and is being studied in clinical trials for attacks of demyelinating diseases of the central nervous system. The drug works by attaching to a protein called the neonatal Fc receptor (FcRn), which stops the body from recycling harmful antibodies, thereby lowering their levels. It belongs to the class of monoclonal antibodies that act as FcRn antagonists.

Investigated diseases

  • Clinically Isolated Syndrome

    A brief episode of neurological symptoms that lasts at least 24 hours and is caused by inflammation in the central nervous system. It often involves a single area such as the optic nerve or spinal cord. The condition may evolve over time, showing new symptoms or lesions.

  • Relapsing‑Remitting Multiple Sclerosis

    A form of multiple sclerosis characterized by clear attacks of neurological problems followed by periods of partial or complete recovery. During relapses, new symptoms appear or old ones worsen, then stabilize during remission. Over time, the frequency and severity of attacks can change.

  • Aquaporin‑4 Antibody Positive Neuromyelitis Optica Spectrum Disorder

    An autoimmune condition where antibodies target a water channel protein, leading to inflammation mainly of the optic nerves and spinal cord. Symptoms include vision loss, weakness, and sensory changes that develop over days to weeks. Episodes may recur, each affecting the same or different parts of the nervous system.

  • Myelin Oligodendrocyte Glycoprotein Antibody‑Associated Disease

    An immune‑mediated disease in which antibodies attack a protein on the surface of myelin, causing inflammation of the brain, spinal cord, or optic nerves. Patients experience sudden vision problems, weakness, or balance issues that can improve and then return. New attacks can involve different regions of the nervous system.

  • Demyelinating diseases

    A group of disorders in which the protective covering of nerve fibers in the brain and spinal cord is damaged. This loss of myelin interferes with the rapid transmission of nerve signals, leading to a variety of sensory, motor, and visual symptoms. The diseases often follow a pattern of episodes separated by periods of relative stability.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2025-523654-13-00Estimated enrolment116 patientsSponsorMedizinische Hochschule Hannover

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