An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
This study is an extension of a previous trial, focusing on children and teenagers who have a condition called generalized myasthenia gravis, which causes muscle weakness. The main goal is to understand the long-term safety and how well people tolerate a medicine called Zilucoplan. Participants will receive this medicine as a shot under the skin once a day for an additional 52 weeks (about a year). This research helps doctors learn more about using Zilucoplan safely and effectively for a longer period in younger patients. It's for people who have already been in the first part of the study and are doing well with the treatment.
At a glance
What is this study about?
This study is about a medicine called Zilucoplan, which is being investigated for children and teenagers who have a medical condition called generalized myasthenia gravis. This condition causes muscles to become weak and tired easily.
The study is called an 'open-label extension' because it follows on from a previous study. If you were in the first study and responded well to Zilucoplan, this extension allows you to continue taking the medicine for another year. The main reason for doing this is to see if taking Zilucoplan for a longer time (an extra 52 weeks) is still safe and if your body continues to cope well with it.
Understanding the long-term safety and how people tolerate a medicine is really important, especially for younger patients. This research helps doctors and scientists gather more information about how Zilucoplan works over time, making sure it's a suitable and safe option for those who need it.
Key takeaways
- This study is for children and teenagers who took part in a previous Zilucoplan study.
- It aims to check the long-term safety and how well Zilucoplan is tolerated.
- Participants will get daily shots under the skin for another year.
- It helps doctors understand the medicine better for younger patients.
- You must meet certain age and health requirements to join.
Who may be eligible?
To join this study, you must have already taken part in the previous Zilucoplan study (called MG0014) and completed it properly. Your doctor must also believe that continuing Zilucoplan treatment would be good for you.
For those in the United States, you need to be at least 12 years old. For participants in other parts of the world, you need to be at least 2 years old. Everyone joining the study must agree to have booster vaccinations against certain infections (like meningitis) if recommended by their local doctor.
You cannot join if you stopped the previous study early because of a serious side effect or if your blood tests show you have a specific type of myasthenia gravis (called muscle-specific kinase positive). Also, if you know you are allergic to any part of the medicine, or if you've had meningococcal disease in the past, you won't be able to take part.
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.
- Did I complete the previous Zilucoplan study (MG0014)?
- Does my doctor think continuing Zilucoplan is good for me?
- Am I at least 12 years old (USA) or 2 years old (Rest of World)?
- Am I willing to get booster vaccinations if my doctor recommends them?
- Have I ever had meningococcal disease?
- Do I know if I'm allergic to any part of the Zilucoplan medicine?
What does participation involve?
If you join this study, you will continue to receive the medicine Zilucoplan. It's given as a shot under the skin every day. The study will last for an extra 52 weeks, which is about one year, after you finish the first study. You will have regular appointments with your study team to check how you are doing, measure your muscle strength, and make sure the medicine is still safe and working well for you. The team will also monitor you for any side effects and may take blood samples. If needed, you might also receive booster vaccinations during the study.
Potential risks and benefits
Locations (7)
- Mg0015 40144Verified postcodeMilan, Italy
- Mg0015 40774Verified postcodeKatowice, Poland
- Mg0015 40218Verified postcodeWarsaw, Poland
- Mg0015 20104Verified postcodeSeoul, South Korea
- Mg0015 20220Verified postcodeSeoul, South Korea
- Mg0015 40735Verified postcodeGlasgow, United Kingdom
- Mg0015 40736Verified postcodeLondon, United Kingdom
Common questions
What is myasthenia gravis?
It's a condition that causes muscles to get weak and tired easily, affecting things like eye movement, speaking, and swallowing.
What is Zilucoplan?
Zilucoplan is a medicine being tested to see if it can help manage the symptoms of myasthenia gravis.
How will I take Zilucoplan?
You will receive it as a daily shot under your skin.
How long will this study last?
This extension study will last for an additional 52 weeks, which is about one year, after you finished the first study.
Can I stop the study if I want to?
Yes, you can choose to leave the study at any time, and it won't affect 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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