A Phase 1B, Open-Label Study of Mirdametinib in Infants and Toddlers with Neurofibromatosis Type 1 associated Plexiform Neurofibromas
This study is testing a new medicine called Mirdametinib for very young children, specifically babies and toddlers, with a condition known as Neurofibromatosis Type 1 (NF1). A common problem in NF1 can be the growth of benign (non-cancerous) tumours called plexiform neurofibromas. Doctors want to see if Mirdametinib can help with these tumours. The main goal of this first phase of the study is to carefully check if the medicine is safe for these young children. They will be looking for any side effects and how the medicine works in their bodies. They will also look to see if the tumours shrink and how the children's quality of life and pain levels are affected.
At a glance
What is this study about?
This study is about a medical condition called Neurofibromatosis Type 1, often shortened to NF1. NF1 is a genetic condition that causes tumours to grow on nerves. In this study, we're focusing on a specific type of tumour called plexiform neurofibromas, which are non-cancerous but can cause problems. The study is for babies and toddlers who have NF1 and these particular tumours.
The main aim of this research is to see if a new medicine, called Mirdametinib, is safe for these young children. Because it's a new medicine being tested in this age group, doctors need to be very careful to observe how the children's bodies react to it. They will closely monitor for any side effects and understand how the medicine moves through and is used by the body. This is a very important first step before later studies can look at how well the medicine works.
Beyond just safety, the study also wants to see if Mirdametinib can help shrink the plexiform neurofibromas. They will use MRI scans to measure any changes in the size of the tumours. Researchers will also look at how the children's daily lives and comfort are affected, asking about their quality of life and any pain they might be experiencing.
Key takeaways
- This study is for babies and toddlers with Neurofibromatosis Type 1 (NF1) and plexiform neurofibromas.
- It's testing a new medicine called Mirdametinib to see if it's safe and how it affects the tumours.
- Participation involves regular clinic visits for medical checks, blood tests, and scans.
- The study also looks at how the medicine affects a child's quality of life and pain.
- It's an early-stage study, mainly focused on safety and understanding the medicine's effects.
Who may be eligible?
This study is open to babies and toddlers. Boys and girls are both welcome to take part. Since the study is looking at a specific condition, the children taking part would need to have been diagnosed with Neurofibromatosis Type 1 (NF1) and have plexiform neurofibromas.
There might be other specific health requirements for a child to join, which the study doctors would review very carefully. If you are interested, your child's doctor or the study team would be able to check if they meet all the necessary criteria.
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 your child a baby or toddler?
- Does your child have a diagnosis of Neurofibromatosis Type 1 (NF1)?
- Does your child have plexiform neurofibromas?
- Are you able to attend regular clinic appointments with your child?
What does participation involve?
If your child takes part in this study, they will receive the medicine Mirdametinib. This is an 'open-label' study, which means everyone involved (you, your child, and the medical team) will know that your child is receiving the active drug.
Your child will have regular visits to the clinic for various checks. These will include blood tests, checking their heart's activity with an ECG (a simple heart tracing) and an echocardiogram (a heart scan), eye exams, and thorough physical examinations. Doctors will also measure their weight, height, and other vital signs like temperature and breathing. These tests are to make sure the medicine is safe and to see how your child is responding. The team will also ask about your child's quality of life and any pain they might have using special age-appropriate questionnaires. The total duration of participation and how often these visits occur would be explained in detail by the study team.
Potential risks and benefits
Locations (2)
- —UnverifiedSpain
- —UnverifiedGermany
Common questions
What is Neurofibromatosis Type 1 (NF1)?
NF1 is a genetic condition that causes non-cancerous tumours to grow on nerves, often starting in childhood. It can also cause other symptoms like skin spots.
What are plexiform neurofibromas?
These are a specific type of benign (non-cancerous) tumour that can grow in children with NF1. They can sometimes cause problems depending on where they are located.
What does 'Phase 1B' study mean?
This is an early stage of testing a new medicine in people. It mainly focuses on checking if the medicine is safe and how much to give, rather than how well it works.
Will my child receive a placebo (dummy medicine)?
No, this is an 'open-label' study, meaning all children taking part will receive the active medicine, Mirdametinib. There is no placebo group in this study.
What happens if we decide not to participate?
That's perfectly fine. Your child will continue to receive their standard medical care, and your decision will not affect their treatment in any way.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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