RecruitingPHASE2, PHASE3INTERVENTIONAL

Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

This research study is investigating a new treatment, efgartigimod, given intravenously (into a vein) for children and teenagers between 2 and 18 years old who have a muscle weakness condition called generalized myasthenia gravis (gMG). The main goals are to understand how the medication behaves in the body, how it affects the condition, and if it is safe for this age group. Participants will be in the study for about 28 weeks in total, with treatment lasting 8 weeks in the first part and 18 weeks in the second part. The study aims to gather important information to help children and young people living with gMG.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

argenx

Enrolment target

Start

26 Oct 2021

Estimated completion

01 Mar 2027

Generalized Myasthenia Gravis

What is this study about?

This study is being conducted to learn more about a new treatment, efgartigimod, for children and teenagers with a condition called generalized myasthenia gravis (gMG). gMG is a long-term condition that causes muscle weakness. Efgartigimod is given directly into a vein (intravenously).

The main aim of the study is to understand how the body processes efgartigimod (this is called pharmacokinetics), how it affects the symptoms of gMG (pharmacodynamics), and most importantly, to check if it's safe for children and teenagers. Researchers also want to see if the treatment can improve the symptoms of gMG in this age group.

By gathering this information, doctors hope to find better ways to manage gMG in younger patients, potentially leading to more effective and safer treatment options in the future. The study is divided into two main parts, with different treatment durations, to carefully assess the medication's effects.

Key takeaways

This study is for children and teenagers with generalized myasthenia gravis (gMG).
It's testing a new treatment called efgartigimod, given into a vein.
The main goals are to check safety and how the treatment works.
Participation lasts about 28 weeks, including treatment and monitoring.
It's for those whose current gMG treatments haven't been fully effective.
You can withdraw from the study at any time.

Who may be eligible?

This study is looking for children and teenagers with generalized myasthenia gravis (gMG) who are between 2 and 18 years old. Participants must have received a formal diagnosis of gMG and meet specific clinical criteria related to the severity of their condition.

To be considered, participants should have found their current treatments (like certain immune-suppressing medicines, steroids, or acetylcholinesterase inhibitors) haven't worked well enough, or have caused difficult side effects. They should also be on a stable dose of their current gMG treatment for a certain period before joining the study.

Additionally, participants must have a positive blood test for acetylcholine receptor (anti-AChR) antibodies, which helps confirm the gMG diagnosis. For very young children weighing less than 15kg, previous test results might be used instead.

Quick self-check

Is your child between 2 and 18 years old?
Does your child have a confirmed diagnosis of generalized myasthenia gravis (gMG)?
Has your child found their current gMG treatments aren't working well enough?
Has your child had a positive blood test for specific gMG antibodies?
Are you able to attend regular clinic visits for your child?

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

What does participation involve?

If you decide to take part, the total study duration for each child will be around 28 weeks. This period includes regular visits to the clinic for assessments and to receive the study treatment.

The treatment itself will be given over two main periods: an initial 8-week phase to help determine the right dose, followed by an 18-week phase to confirm how well the treatment works. During these periods, efgartigimod will be given directly into a vein.

Throughout the study, there will be various tests and checks to monitor your child's health, how the treatment is affecting their gMG, and to ensure their safety. This will involve physical examinations, blood tests, and discussions about any symptoms or side effects.

Potential risks and benefits

Participating in a study like this may offer potential benefits, such as access to a new treatment that might improve your child's gMG symptoms. However, it's also important to be aware of potential risks, including possible side effects from the medication or discomfort from procedures like blood tests. The study team will carefully monitor your child's health throughout and explain all known and potential risks in detail. Remember, your child can withdraw from the study at any time and for any reason, without it affecting their ongoing healthcare.

Locations (25)

Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, United States· Recruiting
University of North Carolina at Chapel Hill
Chapel Hill, United States· Recruiting
University of Virginia (UVA) Health - Developmental Pediatrics Clinic
Charlottesville, United States· Recruiting
Medizinische Universitat Wien
Vienna, Austria· Completed
Universitair Ziekenhuis Antwerpen
Antwerp, Belgium· Recruiting
Alberta Childrens Hospital
Calgary, Canada· Recruiting
British Columbia Children's Hospital
Vancouver, Canada· Recruiting
AP-HM - Hopital de la Timone
Marseille, France· Recruiting
Assistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades
Paris, France· Recruiting
Vian - M. Iashvili Children's Central Hospital
Tbilisi, Georgia· Completed
Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic
Tbilisi, Georgia· Completed
Charite Universitaetsmedizin Berlin - Campus Virchow-Klinikum - Sozialpadiatrisches Zentrum
Berlin, Germany· Completed

+13 more sites — see the official record for the full list.

Common questions

What is generalized myasthenia gravis (gMG)?

gMG is a condition that causes muscles to weaken, leading to problems with movement, speaking, and breathing.

What is efgartigimod?

Efgartigimod is a potential new treatment being tested to help manage the symptoms of gMG.

How will the treatment be given?

The treatment will be given directly into a vein, which is called an intravenous (IV) infusion.

How long will my child be in the study?

Each child will participate for approximately 28 weeks in total.

Can my child stop participating at any time?

Yes, your child can stop participating in the study at any point without it affecting their normal medical care.

How to find out more

Sabine Coppieters, MD

ClinicalTrials@argenx.com 857-350-4834 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.