A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)
This research study is focused on children and teenagers, aged 2 to 17, who have a condition called generalized Myasthenia Gravis (gMG). This condition causes muscle weakness. The study is testing a new medicine called inebilizumab. The main goals are to learn how this new medicine behaves in a child's body, for example, how much of it gets into the blood and how long it stays there. The study also aims to check if inebilizumab is safe for children and if their bodies can handle it well. By understanding these things, doctors hope to develop better treatments for children living with Myasthenia Gravis.
At a glance
What is this study about?
This study is about a new medicine called inebilizumab for children and teenagers (from 2 up to 17 years old) who have a condition called generalized Myasthenia Gravis, often shortened to gMG. Myasthenia Gravis is a long-term condition that causes muscle weakness. This can affect muscles used for things like eye movement, smiling, talking, swallowing, and even breathing. The 'generalized' part means it can affect muscles all over the body.
The researchers want to learn two main things about inebilizumab in children with gMG. Firstly, they want to understand how the medicine moves through the body, how much of it reaches where it needs to go, and how long it stays active. This helps doctors figure out the best way to give the medicine. Secondly, they want to check if the medicine is safe and if children can take it without too many problems or uncomfortable side effects. This is really important to make sure any new treatment is going to help people.
Finding new and effective treatments for children with gMG is vital because it can greatly improve their quality of life. This study is an important step in researching potential new options to help manage their symptoms and muscle weakness.
Key takeaways
- This study is for children aged 2-17 with generalized Myasthenia Gravis (gMG).
- It's testing a new medicine called inebilizumab.
- The main goals are to understand how the medicine works in the body and to check its safety.
- Children need a confirmed diagnosis of gMG and meet specific medical criteria.
Who may be eligible?
To be part of this study, a child must be between 2 and 17 years old. They need to have a confirmed diagnosis of generalized Myasthenia Gravis (gMG), which means doctors have found specific antibodies in their blood or have seen certain signs of the condition.
Children can't have other conditions that might explain their muscle weakness. Their gMG should be of a certain level of severity, meaning their symptoms are noticeable but not extremely mild or extremely severe based on a specific doctor's classification. Also, if they are already taking certain medicines for their gMG, like steroids or other treatments that lower the immune system, these medications need to have been stable at the same dose for a certain period before joining the study.
Importantly, if a child is too young to give their own permission, their parent or legal guardian must agree for them to take part. The child will also be asked to agree, if they are old enough to understand what's happening, but not old enough to give full legal consent on their own.
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.
- Is my child between 2 and 17 years old?
- Does my child have a confirmed diagnosis of generalized Myasthenia Gravis (gMG)?
- Has my child been on their current gMG medications (if any) at a stable dose for a certain period?
- Do we have a parent/guardian willing to give consent, and is my child willing to agree (assent) if old enough?
What does participation involve?
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Potential risks and benefits
Locations (4)
- Austin Neuromuscular CenterVerified postcodeAustin, United States· Recruiting
- Hopital Armand TrousseauVerified postcodeParis, France· Recruiting
- Hospital Universitari i Politecnic La FeVerified postcodeValencia, Spain· Recruiting
- John Radcliffe HospitalVerified postcodeHeadington, United Kingdom· Not yet recruiting
Common questions
What is Myasthenia Gravis?
Myasthenia Gravis is a condition that causes muscles to become weak and tired easily because of a problem with how nerves communicate with muscles.
What does 'generalized' mean in gMG?
'Generalized' means the muscle weakness can affect many different muscles all over the body, not just in one specific area.
What is inebilizumab?
Inebilizumab is the name of the medicine being studied. Researchers want to see how it works and if it's safe for children with gMG.
Why are they studying this medicine in children?
They are studying it in children to see if it can be a safe and effective treatment option specifically for younger people living with gMG.
What does Phase 2 mean?
Phase 2 means this is an earlier stage of testing where researchers are looking more closely at how effective the medicine is and if it causes side effects in a larger group of people than in a Phase 1 study.
How to find out more
Amgen Call Center
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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