A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
This research study is looking into a new medicine called Zilucoplan for young people diagnosed with generalised myasthenia gravis, a condition that causes muscle weakness. The main goal is to find out more about how Zilucoplan behaves in the body of children and teenagers, including how much of it reaches different parts of the body and how long it stays there. We also want to see if the medicine is safe and if it causes any unwanted side effects. Importantly, the study will also help us understand if Zilucoplan can improve the symptoms of gMG in young patients. This information is key to deciding if this new treatment could be a good option for future care.
At a glance
What is this study about?
This study is about a new medicine called Zilucoplan, and it's for young people who have a condition called generalised myasthenia gravis, or gMG. Myasthenia gravis is a long-term condition that causes muscles to become weak and tired easily. This can affect things like how you move your eyes, swallow, speak, or even move your arms and legs. We don't have many treatment options specifically for children and teenagers with gMG, so finding new ones is really important.
In this study, doctors want to learn more about how Zilucoplan works in the bodies of young people. They will be looking at things like how quickly the medicine gets into the body, how it affects certain body processes, and if it's generally safe for children and teenagers to use. They'll also be carefully checking for any side effects and seeing if the medicine helps improve the muscle weakness that comes with gMG. The study is currently in Phase 2 and 3, which means it has already been tested in some people and is now being studied more widely to see how well it works compared to existing treatments, if any, and its overall safety.
The information gathered from this study will help us understand if Zilucoplan could be a helpful new treatment option for young people with gMG in the future. Finding effective and safe treatments is crucial for improving the quality of life for those living with this condition.
Key takeaways
- Tests a new medicine, Zilucoplan, for young people with gMG.
- Aimed at improving muscle weakness and overall health.
- Involves regular check-ups, blood tests, and symptom tracking.
- Free to participate and can withdraw at any time.
- Helps scientists learn more about gMG treatments for the future.
Who may be eligible?
This study is looking for young people with generalised myasthenia gravis. In the UK and most other countries outside of the USA, children aged 2 to under 18 years old can take part. For those in the USA, participants must be between 12 and under 18 years old. All participants must have a clear diagnosis of generalised myasthenia gravis that has been confirmed by a specific blood test for antibodies.
Your gMG symptoms must be of a certain type and severity, for example, affecting your daily activities with a score of 6 or more on a special scale if you are aged 12 or over. For younger children (2 to under 12 years old), you must have documented weakness in at least one limb, neck, or swallowing muscle. It's also really important that you have had your meningococcal vaccinations or are able to take antibiotics to prevent infection if you haven't been fully vaccinated.
However, some things might mean you can't join the study. For example, if you have a different type of myasthenia gravis (like MUSK positive), or have recently had a serious infection, or have had an operation to remove your thymus gland in the last 6 months, you wouldn't be able to participate. Your study doctor will discuss all these details with you and your family to see if this study is right.
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 between 2 and under 18 years old (outside USA)?
- Have you been diagnosed with generalised myasthenia gravis (gMG) at your hospital?
- Do you have muscle weakness that affects your daily activities?
- Are your meningococcal vaccinations up to date, or can you take antibiotics?
- Have you *not* had your thymus gland removed in the last 6 months?
- Have you *not* had a serious infection recently that needed strong antibiotics?
What does participation involve?
If you decide to take part, you'll receive the study medicine, Zilucoplan, and will have regular check-ups with the study team. These check-ups will involve things like physical examinations, blood tests to see how the medicine is affecting you and to check for side effects, and questions about your symptoms and how you're feeling. You might also have specific tests to measure your muscle strength. The study will involve several visits to the clinic over a period, and you'll be carefully monitored throughout. At each visit, the team will ensure you are comfortable and understand everything that is happening. The full duration of your involvement will be clearly explained by the study staff.
Potential risks and benefits
Locations (9)
- Mg0014 50168Verified postcodeChicago, United States· Withdrawn
- Mg0014 50574Verified postcodeDenton, United States· Withdrawn
- Mg0014 40144Verified postcodeMilan, Italy· Recruiting
- Mg0014 40774Verified postcodeKatowice, Poland· Recruiting
- Mg0014 40218Verified postcodeWarsaw, Poland· Active not recruiting
- Mg0014 20104Verified postcodeSeoul, South Korea· Recruiting
- Mg0014 20220Verified postcodeSeoul, South Korea· Recruiting
- Mg0014 40735Verified postcodeGlasgow, United Kingdom· Recruiting
- Mg0014 40736Verified postcodeLondon, United Kingdom· Recruiting
Common questions
What is generalised myasthenia gravis (gMG)?
gMG is a long-term condition that causes muscle weakness and tiredness, affecting muscles that control movement, eyes, speech, and swallowing.
What is Zilucoplan?
Zilucoplan is a new medicine being tested to see if it can help improve symptoms in young people with gMG.
Who is this study for?
This study is for children and teenagers (aged 2 to under 18, or 12 to under 18 in the USA) who have been diagnosed with generalised myasthenia gravis.
Will I have to pay to be in the study?
No, all study-related treatments and tests are provided at no cost to you or your family.
Can I leave the study at any time?
Yes, you or your parent/guardian can decide to leave the study at any point, and it won't affect your regular medical care.
How to find out more
UCB Cares
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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