Active not recruitingPHASE3INTERVENTIONAL

A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis

This research study is testing a new medicine called Efgartigimod PH20 SC for adults who have ocular myasthenia gravis. This condition mainly causes weakness in the eye muscles, leading to problems like drooping eyelids or double vision. The main goals are to find out if this medicine is safe to use and if it can help improve these eye symptoms. The study is split into two parts. In the first part, some participants will receive the active medicine, while others will get a placebo (a dummy medicine with no active ingredients). This helps researchers see the true effect of the new medicine. In the second part, all participants will receive the active medicine. The study will last for about two years and 12 weeks in total.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

argenx

Enrolment target

141

Start

18 Sep 2024

Estimated completion

01 Dec 2027

Myasthenia Gravis, Ocular

What is this study about?

This study is about a new way to treat a condition called ocular myasthenia gravis. If you have this condition, your immune system mistakenly attacks healthy parts of your body, specifically the connections between your nerves and eye muscles. This can lead to symptoms like drooping eyelids or seeing double. The new medicine being tested, Efgartigimod PH20 SC, is given by an injection under the skin using a special pre-filled syringe.

The main aim of the study is to see how well this new medicine works and if it's safe for people with ocular myasthenia gravis. The researchers want to understand if it can help to reduce or improve the eye symptoms that people experience. Knowing if a new treatment is both effective and safe is very important before it can be widely used.

The study is designed in two stages. Initially, some people will get the new medicine, and others will get a placebo, which looks just like the study medicine but doesn’t contain any active ingredients. This comparison helps researchers determine if any improvements are truly due to the medicine. Later on, everyone in the study will have the chance to receive the active medicine. This long-term approach helps track how participants respond over time.

Key takeaways

This study tests a new injection for adults with eye muscle weakness from myasthenia gravis.
It aims to check if the medicine, Efgartigimod, is safe and helps improve eye symptoms.
The study involves a comparison with a dummy medicine (placebo) initially.
Participation lasts for about two years and 12 weeks.
All participants will receive the active medicine in the later part of the study.

Who may be eligible?

To be able to join this study, you must be at least 18 years old.

You also need to have a clear diagnosis of myasthenia gravis, specifically the type that mostly affects the eyes (ocular myasthenia gravis). Doctors will confirm this diagnosis through specific tests, such as blood tests looking for certain antibodies or nerve tests. Your current eye symptoms, such as drooping eyelids or double vision, should also be at a certain level to be included.

There are also some reasons why you might not be able to join. For example, if you have other medical conditions that cause similar eye symptoms, or other serious health problems that might make it unsafe to take part or make it hard to tell if the study medicine is working, you would not be able to participate.

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 at least 18 years old?
Do you have a confirmed diagnosis of myasthenia gravis that mainly affects your eyes?
Are your current eye symptoms at a certain level, such as significant drooping eyelids or double vision?
Do you have any other serious health problems that might complicate your participation?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be involved for about two years and 12 weeks. During this time, you will have regular visits to the study clinic. These visits will involve health checks, assessments of your eye symptoms, and possibly blood tests or other evaluations to see how you are responding to the treatment.

For the first part of the study (about 7 weeks), you will either receive the new medicine, Efgartigimod PH20 SC, or a placebo (a dummy medicine), given by an injection under the skin using a pre-filled syringe. Neither you nor your study doctor will know which one you are receiving during this phase. After this, everyone in the study will receive the active Efgartigimod medicine for the rest of the study period.

Potential risks and benefits

Taking part in a study like this might have potential benefits if the new medicine helps to improve your ocular myasthenia gravis symptoms. However, as with all medicines, there could be risks of side effects, which the study team will monitor closely. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (102)

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

HonorHealth Neurology - Bob Bové Neuroscience Institute
Verified postcode
Scottsdale, United States
Loma Linda University Health
Verified postcode
Loma Linda, United States
USC Roski Eye Institute - Los Angeles
Verified postcode
Los Angeles, United States
University of California San Francisco
Verified postcode
San Francisco, United States
Neurology Offices of South Florida
Verified postcode
Boca Raton, United States
SFM Clinical Research, LLC
Verified postcode
Boca Raton, United States
Neurology Associates PA
Verified postcode
Maitland, United States
University of South Florida
Verified postcode
Tampa, United States
Northwestern Memorial Hospital
Verified postcode
Chicago, United States
University of California Irvine
Verified postcode
Columbia, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
Henry Ford Health System
Verified postcode
Detroit, United States

Common questions

What is ocular myasthenia gravis?

It's a condition where your body's immune system attacks your eye muscles, causing weakness, drooping eyelids, or double vision.

What is a placebo?

A placebo is a 'dummy' medicine that looks like the real treatment but contains no active ingredients. It helps researchers compare the true effects of the new medicine.

How long will the study last if I join?

The study will last for about two years and 12 weeks in total.

Will I know if I'm getting the real medicine or the placebo?

In the first part of the study, neither you nor your study doctor will know. This is a common way to test new medicines fairly.

What does 'Efgartigimod PH20 SC' mean?

Efgartigimod is the name of the new medicine being tested, and 'SC' means it's given by a 'subcutaneous' injection, which is under the skin.