The TiNT trial is studying whether a medicine called trametinib can help children and young adults with Neurofibromatosis Type 1 (NF1) who have tumours in the nerves or brain. These tumours can cause pain, vision problems, and difficulties with daily life, and current treatments don’t always work well. The trial is testing whether trametinib can safely shrink or slow tumour growth and improve symptoms and quality of life for people aged 3 months to 25 years with NF1‑related plexiform neurofibromas or optic pathway gliomas.

There is also a sub-study occurring within this study to find out if trametinib can improve learning, behaviour, pain and well-being for those with NF1. For this part of the study, an untreated control group will be enrolled to compare to the trametinib-treated groups. The untreated control group will be children, teenagers and young adults with NF1 who do not have a tumour that requires trametinib treatment.

It is hoped this research will help determine if trametinib has an effect on tumours and if there are any additional impacts to cognitive function, behaviour and quality of life.