Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

To join this study, children need to be between 1 and 6 years old when the parents or guardians agree to take part. They must have a diagnosis of Neurofibromatosis Type 1 (NF1) and have a specific type of tumour called a Plexiform Neurofibroma (PN) that causes symptoms and can't be operated on.

Their PN must be large enough to be measured on scans, and the child's overall health and ability to do daily activities must be at a certain level. Parents or guardians will need to give their full permission for their child to join the study.

Children cannot join if they have had certain types of cancer recently, or if they have serious stomach or gut problems that might stop them from absorbing the medicine properly. Children with certain severe brain tumours are also not able to take part.

If your child joins the study, they will receive the selumetinib medicine in a granule (liquid) form. The study will start by carefully finding the right dose for children in different age groups (4 to 6 years old first, then 1 to 3 years old). They will take the medicine for a total of 25 cycles, with each cycle lasting 28 days, unless they need to stop earlier.

During this time, your child will have regular check-ups, probably including blood tests to see how the medicine is working in their body, and scans (like MRI) to check the size of their tumour. Doctors will also carefully monitor your child for any side effects and to make sure the medicine is safe. The total duration of participation for each child will depend on how they respond to the treatment, but they are planned to receive the medicine for almost two years if they continue through all 25 cycles.