Active not recruitingPHASE3INTERVENTIONAL

A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

This study is looking into a new treatment called efgartigimod for a specific type of Myasthenia Gravis (MG). This type of MG is called "AChR-Ab seronegative generalized Myasthenia Gravis." The main goal is to see if efgartigimod helps improve symptoms and if it has any side effects, compared to a placebo (a dummy medicine with no active ingredients). Participants will first be randomly given either efgartigimod or the placebo. After this initial part, all participants will have the chance to receive efgartigimod openly. The study will last for about two and a half years. It's for adults aged 18 and over, and both men and women can take part. This research aims to find more effective and safer ways to manage MG.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

argenx

Enrolment target

119

Start

16 Apr 2024

Estimated completion

01 Jun 2027

Generalized Myasthenia Gravis gMG Myasthenia Gravis, Generalized Myasthenia Gravis

What is this study about?

Myasthenia Gravis (MG) is a long-term condition that causes muscle weakness. It can affect how well you move your eyes, face, throat, and limbs. This particular study is for people with a specific type of MG called 'AChR-Ab seronegative generalized Myasthenia Gravis'. In simpler terms, that means you have MG that affects many parts of your body, but you don't have a specific antibody (a protein in the blood) that doctors often look for to diagnose the condition.

The research aims to test a new medicine called efgartigimod. Doctors want to know if this medicine can help manage the symptoms of this type of MG and if it's safe to use. Currently, there are treatments for MG, but doctors are always looking for new options that might work better or have fewer side effects for different people.

Taking part in a study like this helps advance our understanding of MG and could lead to new treatments becoming available in the future. It's an important step in finding better ways to support people living with this condition.

Key takeaways

This study is testing a new medicine (efgartigimod) for a specific type of Myasthenia Gravis.
It's compared against a placebo initially, then all participants get the new medicine.
The study will last for about 2.5 years.
It's for adults aged 18 and older with diagnosed generalized MG.
Taking part can help advance understanding and treatment of MG, but it involves regular clinic visits.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you need to be at least 18 years old and legally able to agree to take part. You must have been diagnosed with generalized Myasthenia Gravis (MG) after the age of 16, and not have had muscle weakness since childhood. Your diagnosis of MG should also be supported by specific medical tests showing problems with your nerve signals to muscles, or you have anti-MuSK antibodies.

Also, you should have either had a positive edrophonium test in the past, or you've shown improvement in your MG symptoms with treatments like certain pills, plasma exchange, or IVIg. You must also be on a steady dose of your current MG medications, which could include acetylcholinesterase inhibitors, steroids, or other immune-suppressing drugs, either alone or in combination.

You cannot join if you have other known autoimmune diseases, certain cancers (unless fully cured for at least three years, with some exceptions like certain skin cancers), or any medical condition that might make it hard to properly check your MG symptoms or put your health at risk during the study.

Quick self-check

Are you 18 years old or older?
Do you have generalized Myasthenia Gravis (MG) that started after age 16?
Have you had specific tests or shown improvement with certain MG treatments?
Are you on a stable dose of your current MG medications?
Do you not have other serious autoimmune diseases or certain cancers?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you'll be involved for about two and a half years. Initially, you'll be randomly assigned to receive either the new medicine, efgartigimod, or a placebo (a dummy medicine) through a drip into your arm (intravenous, or IV). Neither you nor your study doctor will know which one you're receiving at this point. After this first part of the study, everyone who completes it will then get efgartigimod through IV, openly, meaning everyone will know you are receiving the active drug.

Throughout the study, you'll have regular visits to the clinic where you'll have assessments to check your symptoms, your general health, and any side effects. This will involve physical examinations, blood tests, and possibly questionnaires. The specific number and timing of visits will be explained by the study team, but they are designed to carefully monitor your progress and safety.

Potential risks and benefits

Taking part in a clinical trial might offer potential benefits, such as access to a new treatment before it's widely available and closer monitoring of your health by medical professionals. However, there are also potential risks, including side effects from the study medication or the placebo, and the inconvenience of extra clinic visits and tests. The research medicine might not work for you, or it could cause unexpected reactions. Your safety is a top priority, and the research team will explain all known risks and monitor you closely. Remember, participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (91)

HonorHealth Neurology - Bob Bove Neuroscience Institute
Scottsdale, United States
Loma Linda University Health
Fresno, United States
First Choice Neurology Boca Raton
Boca Raton, United States
SFM Clinical Research LLC
Boca Raton, United States
The Neurology Institute / Healthcare Innovations Institute - Coral Springs
Coral Springs, United States
Neurology Associates PA
Maitland, United States
Desai Sethi Medical Center
Miami, United States
Medsol Clinical Research Center Inc
Port Charlotte, United States
BayCare - St. Anthony's Hospital
St. Petersburg, United States
University of South Florida (USF) Health - Morsani Center for Advanced Healthcare
Tampa, United States
Wellstar - Augusta University Medical Center
Augusta, United States
Kansas University Medical Center - Kansas City
Fairway, United States

+79 more sites — see the official record for the full list.

Common questions

What is 'AChR-Ab seronegative gMG'?

It's a type of Myasthenia Gravis that affects muscles all over the body, but specific antibodies often found in other MG patients are not present in your blood.

What is a placebo?

A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active ingredients. It helps doctors see if the new medicine is truly making a difference.

How long will I be in the study?

The study will last for approximately two and a half years from start to finish.

Will I definitely get the new medicine?

In the first part of the study, you'll have a 50/50 chance of getting the new medicine or the placebo. In the second part, everyone who finishes the first part will receive the new medicine.

Can I stop participating if I want to?

Yes, you can leave the study at any time, for any reason, without impacting your regular medical care.