AuthorisedPhase II and Phase III (Integrated)Interventional

A multicentre, randomized, double-blind, placebo-controlled phase II/III trial investigating the efficacy of anti-FcRn targeting with efgartigimod as a first-line add-on therapy to IVMP in moderate-to-severe attacks of demyelinating diseases of the central nervous system

Demyelinating diseases, including multiple sclerosis (MS) and related conditions, occur when the body's immune system mistakenly attacks the protective coating around nerves in the brain, spinal cord, or eyes. During a sudden attack or flare-up, people can lose vision, balance, or movement. The standard treatment is a course of strong steroid drips to reduce swelling. However, steroids do not always work well enough on their own. This trial tests whether adding a new drug, efgartigimod, to standard steroid drips improves recovery. Efgartigimod works by quickly clearing away harmful immune proteins that damage nerve coatings. Researchers want to find out if this combination helps people return to their normal health within 84 days and reduces the need for emergency hospital procedures like blood filtering.

At a glance

Status

Authorised

Phase

Phase II and Phase III (Integrated)

Sponsor

Medizinische Hochschule Hannover

Enrolment target

116

Start

17 Jun 2026

Demyelinating diseases

What is this study about?

Demyelinating conditions happen when the immune system accidentally attacks myelin, the protective shield around your nerves. This can cause sudden flare-ups or attacks that lead to muscle weakness, numbness, balance troubles, or blurred vision. These flare-ups can happen in conditions such as multiple sclerosis (MS), neuromyelitis optica spectrum disorder (NMOSD), or MOG antibody disease (MOGAD).

The standard treatment for a moderate-to-severe attack is high-dose steroids given through a drip in hospital. While steroids calm down inflammation, some people do not fully recover their normal abilities. In severe cases, doctors may need to use intensive rescue treatments, such as filtering the blood, to remove harmful antibodies.

This study looks at whether adding a medicine called efgartigimod to standard steroid drips works better than steroids alone. Efgartigimod is designed to target and lower the levels of specific antibodies causing the nerve damage. By clearing these away quickly, the trial hopes to help nerves heal better, restore daily abilities, and reduce the chance of long-term disability.

Key takeaways

Tests whether adding efgartigimod to standard steroid drips helps people recover better from nerve flare-ups.
Open to adults aged 18 and over with a moderate-to-severe attack of a demyelinating condition.
Participation lasts about 84 days and involves regular hospital visits, movement checks, and blood tests.
Everyone receives standard steroid treatment; half also receive the trial drug and half receive a dummy drip.
Participation is completely voluntary and will not affect your standard NHS care.

Who may be eligible?

This study is open to men and women aged 18 and older who are currently having a moderate-to-severe flare-up of a demyelinating nerve condition.

To take part, you must be experiencing new or worsening nerve symptoms affecting your vision, movement, or coordination that require treatment with high-dose steroids given into a vein.

You may not be able to join if you have certain active infections, other serious medical conditions, or reasons why you cannot safely receive the study medication. A doctor on the study team will review your health history and run standard checks to make sure the trial is safe for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you aged 18 or older?
Do you have a diagnosed demyelinating condition (like MS, NMOSD, or MOGAD)?
Are you currently having a moderate or severe flare-up of nerve or vision symptoms?
Has your doctor recommended high-dose steroid drips for this attack?
Are you able to attend clinic visits over the next three months?

Answer every question to see your result.

What does participation involve?

If you decide to join, your participation will last for about 84 days (roughly three months). You will be randomly assigned to receive either the study drug (efgartigimod) or a dummy treatment (placebo), both given as a drip alongside your standard steroid medication. Neither you nor your doctor can choose which group you are in.

You will visit the hospital clinic several times during the study (such as on days 4, 10, 21, 28, and 84). During these visits, doctors will check your vision, test your walking speed, and see how well you can move your hands and fingers. You will also provide blood samples to measure immune proteins and fill out short surveys about your everyday quality of life and general well-being.

Potential risks and benefits

A potential benefit is that the study medicine may help you recover more fully from your attack, although this cannot be promised. You will also receive frequent, specialist health checks from the study team. The risks include possible side effects from efgartigimod, such as mild drip-site reactions or a temporarily higher risk of common infections like colds. There is also a 50% chance you will receive the placebo drip alongside your regular steroids. Taking part is entirely your choice. If you decide to join, you can change your mind and leave the study at any point without it affecting your normal medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany
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Unverified
Austria

Common questions

What is an add-on therapy?

An add-on therapy is a new medicine given alongside the standard, proven treatment (in this case, steroid drips) to see if the combination works better than the standard treatment alone.

Will I still receive treatment if I get the placebo?

Yes. Every person in this trial receives the standard, high-dose steroid treatment for their attack. The placebo is simply an inactive drip given in addition to standard care.

What happens if my symptoms do not improve?

Your safety comes first. If your symptoms do not improve or get worse, your doctors can give you emergency rescue treatments, such as blood filtering (plasma exchange), at any time.

Do I have to pay for any part of the trial?

No. The trial medicine, tests, and care are provided free of charge, and travel expenses for hospital visits are often reimbursed.

Can I leave the study early?

Yes. You are free to stop taking part at any time without giving a reason, and your standard NHS medical care will continue as normal.