This study is investigating whether the medication selumetinib (KoselugoTM) can prevent the growth of plexiform neurofibromas (PN) in young children with Neurofibromatosis type 1 (NF1). PN are tumors that can cause serious health problems. Selumetinib is already approved for children aged 2 and older with inoperable PN, but this study aims to see if starting treatment earlier, before problems develop, can be beneficial. The study is looking for children aged 1 to 8 years old who have NF1 and no known PN at the start of the study. Researchers will measure how long participants live without their PN growing (progression-free survival) over 60 months. The study plans to enroll 200 participants and its current status is unclear.

Study design

This is an interventional study planning to enroll 200 participants. It is not specified if it is randomized or blinded.

What's involved

Participants will receive selumetinib twice daily by mouth. The participant duration in the study is 5 years.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for progression-free survival for 60 months (5 years).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.