Study on the Long-term Safety of Efgartigimod for Children with Generalized Myasthenia Gravis

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What is this study about?

This clinical trial is focused on studying the long-term safety of a treatment for Generalized Myasthenia Gravis, a chronic autoimmune disease that causes muscle weakness. The treatment being tested is called efgartigimod, which is administered in two forms: intravenously (through a vein) and subcutaneously (under the skin). The study aims to evaluate how safe and well-tolerated these forms of efgartigimod are when given to children with this condition.

Participants in the study will receive either the intravenous or subcutaneous form of efgartigimod over a period of time. The study will monitor the occurrence and severity of any side effects, as well as changes in laboratory test results, vital signs, and other health indicators. The study will also look at the presence of any antibodies that might develop against efgartigimod or the enzyme used in the subcutaneous form, known as recombinant human hyaluronidase PH20.

The purpose of this study is to ensure that the treatment is safe for long-term use in children with Generalized Myasthenia Gravis. By carefully observing the effects of the treatment, researchers hope to gather important information that could lead to better management of this condition in the future.

1 joining the study

Participation begins after completing a previous study, either ARGX-113-2006 or ARGX-113-2207. Agreement to join this study is required.

Understanding the study requirements and providing written consent is necessary. This includes a willingness to follow the study procedures.

2 initial assessment

An initial assessment is conducted to ensure eligibility. This includes a negative pregnancy test for female participants of childbearing potential.

3 treatment administration

The medication efgartigimod is administered in two forms: intravenous infusion and subcutaneous injection.

The intravenous form is known as Vyvgart 20 mg/mL concentrate for solution for infusion.

The subcutaneous form is known as Vyvgart 1 000 mg solution for injection.

4 monitoring and follow-up

Regular monitoring is conducted to evaluate the safety and tolerability of the treatment.

This includes checking for any adverse events, changes in laboratory test results, vital signs, height and weight, and electrocardiogram (ECG) results.

5 antibody testing

Testing for the presence of antidrug antibodies against efgartigimod and antibodies against recombinant human hyaluronidase PH20 is performed.

6 completion of the study

The study is estimated to end by September 30, 2028.

Upon completion, a final assessment is conducted to ensure participant safety and gather final data.

Who Can Join the Study?

  • The participant must have completed a previous study called ARGX-113-2006 or ARGX-113-2207. This means they either finished the study and agreed to join this new study, or they qualify for more treatment but couldn’t finish the previous study within its time limits.
  • The participant or their legally authorized representative must understand what the study involves and agree to it in writing. They should also be willing and able to follow the study’s procedures.
  • If the participant is sexually active and can have children, they must use birth control as required by local rules for clinical studies. This applies to both males and females who are biologically capable of having children. Females must have a negative pregnancy test at the start of the study.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
IRCCS Istituto Giannina Gaslini Genoa Italy
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Universitair Ziekenhuis Gent Gent Belgium
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego Warsaw Poland
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Brno Brno Czechia
University Hospital Ostrava Ostrava Czechia
Umblumjjkgpiskkeywwav Eqybe Amu Essen Germany
Lkghc Ummxggjoiyzm Mclcfja Cmsuehi (uhwyx Leiden The Netherlands
Aldlyukmhs Pjcyzios Hcyepvmd Dh Mdwmziqhj Marseille France
Ugktrxmaoexpkm Caoswas Krolxcaad Gdansk Poland
Uvedefovze Or Aechbfb Edegem Belgium

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
01.08.2022
Belgium Belgium
Recruiting
01.08.2022
Czechia Czechia
Not yet recruiting
01.08.2022
France France
Recruiting
01.08.2022
Germany Germany
Recruiting
01.08.2022
Italy Italy
Not yet recruiting
01.08.2022
Poland Poland
Recruiting
01.08.2022
Spain Spain
Recruiting
01.08.2022
The Netherlands The Netherlands
Recruiting
01.08.2022

Trial locations

Investigated drugs:

Efgartigimod: This medication is being tested to see if it is safe and well-tolerated when given to children with generalized myasthenia gravis. It is administered intravenously, which means it is given directly into a vein through an IV.

Efgartigimod PH20: This is a form of efgartigimod that is administered subcutaneously, meaning it is injected under the skin. The trial is evaluating its safety and how well children with generalized myasthenia gravis tolerate this method of administration.

Investigated diseases:

Generalized Myasthenia Gravis – Generalized Myasthenia Gravis is a chronic autoimmune disorder that affects the communication between nerves and muscles, leading to muscle weakness. It occurs when the immune system mistakenly attacks the body’s own tissues, specifically targeting the receptors that receive nerve signals. This results in fluctuating muscle weakness and fatigue, which can affect various muscle groups, including those responsible for eye movement, facial expression, and swallowing. The severity of symptoms can vary widely among individuals and may worsen with physical activity or stress. Over time, the condition can lead to more persistent muscle weakness, impacting daily activities and quality of life.

Trial ID:
2023-507379-23-00
Protocol code:
ARGX-113-2008
NCT ID:
NCT05374590
Trial Phase:
Therapeutic use (Phase IV)

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