Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis
This study is designed to check how safe two different versions of a drug called efgartigimod are over a long period for young people with generalised Myasthenia Gravis (gMG). gMG is a condition that causes muscle weakness. The study will look at efgartigimod given into a vein (IV) and a different form, efgartigimod PH20, given under the skin (SC). All participants will have already taken part in previous studies involving efgartigimod. The aim is to see if these treatments continue to be safe. Participants under 18 can stay in the study until the drug is more widely available. Those 18 and over can stay for up to two years, or until the drug is available commercially, whichever comes first.
At a glance
What is this study about?
This study is focused on understanding the long-term safety of a treatment called efgartigimod for young people affected by generalised Myasthenia Gravis, often shortened to gMG. Myasthenia Gravis is a condition that leads to muscle weakness. Researchers want to ensure that this treatment continues to be safe when used over a longer period.
There are two ways efgartigimod has been given in previous studies: one where it's delivered directly into a vein (this is known as IV) and another where a version called efgartigimod PH20 is given as an injection just under the skin (this is known as SC). This current study will continue to give participants the same way they received it before.
All the children and teenagers taking part in this study have already been involved in earlier trials of efgartigimod (named ARGX-113-2006 and ARGX-113-2207). This study is like a follow-up to those. The main aim isn't to see if the drug works, but purely to monitor its safety over a longer timeframe.
Key takeaways
- This study is about checking the long-term safety of efgartigimod for children and teenagers with gMG.
- Only those who have been part of earlier efgartigimod studies can join.
- Participants will continue to receive the efgartigimod treatment they had before.
- The study aims to observe any effects of the treatment over a longer period.
- Your doctor and the study team will monitor your health carefully.
Who may be eligible?
To be considered for this study, you must have previously completed one of the earlier efgartigimod studies (ARGX-113-2006 or ARGX-113-2207). This means you reached the end of those studies or you were eligible for another dose but couldn't complete it within the original study's timeframe.
Participants, or their legal guardians if they are under 18, must be able to understand what the study involves and agree to take part. They also need to be willing to follow all the study's instructions. The study is open to both boys and girls between the ages of 2 and 18 years.
You cannot join if you stopped taking part in the previous efgartigimod studies early. Also, pregnant or breastfeeding young women, or those planning to become pregnant, cannot participate. If you have a serious ongoing infection that hasn't cleared up, or another serious medical condition that might make it hard to assess your gMG or put you at risk, you would also not be able to join.
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.
- Have I previously completed an efgartigimod study for gMG?
- Am I between 2 and 18 years old?
- Am I (or my legal guardian) able to understand and follow study instructions?
- Am I female and currently not pregnant, breastfeeding, or planning to become pregnant?
- Do I have any serious infections or other health conditions that are not well controlled?
What does participation involve?
If you join this study, you will continue to receive the efgartigimod treatment in the same way and at the same dose you did in your previous study. This means either intravenously (into a vein) or subcutaneously (under the skin).
The main purpose is to keep an eye on your long-term safety, so you'll have regular check-ups and assessments by the study team. They will monitor how you are doing and look for any potential side effects.
If you are under 18 years old, you can stay in the study until efgartigimod becomes generally available in your country, or through another program that allows continued access to the drug for gMG. If you are 18 years or older, you can stay in the study for a maximum of two years, or until the drug becomes widely available, whichever happens first.
Potential risks and benefits
Locations (16)
- Ann and Robert H Lurie Children's Hospital of Chicago - Main HospitalVerified postcodeChicago, United States
- Atrium Health Neurology Specialty CareVerified postcodeCharlotte, United States
- University of Virginia (UVA) Health - Developmental Pediatrics ClinicVerified postcodeCharlottesville, United States
- Universitair Ziekenhuis AntwerpenVerified postcodeEdegem, Belgium
- UZ GentVerified postcodeGhent, Belgium
- Childrens Hospital of Eastern OntarioVerified postcodeOttawa, Canada
- AP-HM- Hôpital de La TimoneVerified postcodeMarseille, France
- Universitätsklinikum EssenVerified postcodeEssen, Germany
- Hadassah Medical Center- Ein KeremVerified postcodeJerusalem, Israel
- Leids Universitair Medisch CentrumVerified postcodeLeiden, Netherlands
- Uniwersyteckie Centrum Kliniczne w GdanskuVerified postcodeGdansk, Poland
- Wielospecjalistyczna Poradnia Lekarska SynapsisVerified postcodeKatowice, Poland
Common questions
What is this study testing?
This study is testing the long-term safety of efgartigimod, a treatment for generalised Myasthenia Gravis (gMG), in young people who have taken it before.
Who can join this study?
This study is for children and teenagers aged 2 to 18 years old who have already completed a previous efgartigimod study for gMG.
What is generalised Myasthenia Gravis (gMG)?
gMG is a condition that causes muscle weakness throughout the body.
How long will I be in the study?
If you're under 18, you can stay until the drug is commercially available. If you're 18 or over, you can stay for up to two years, or until the drug is available.
What happens if I want to stop taking part?
You can withdraw from the study at any time, for any reason, without it affecting your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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