Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
Status:
Not yet recruiting
Trial end date:
2031-08-01
Target enrollment:
Participant gender:
Summary
Plexiform neurofibromas (PN) are known to cause significant morbidity in children with NF1.
The recent FDA approval for selumetinib in children 2 years and older with inoperable
symptomatic PN was based on the finding that selumetinib shrinks the majority of PN in
children with NF1 and results in clinically meaningful benefit such as improvement in pain or
range of motion. However, many morbidities, such as blindness or nerve damage, cannot be
fully reversed with PN shrinkage. Therefore, there remains a critical need in this patient
population to determine if young participants with PN in high-risk locations may benefit from
early medical intervention prior to the development of clinical problems. This study will
determine whether participants with asymptomatic PN in high-risk locations can potentially
benefit from early treatment with selumetinib.
Phase:
Phase 2
Details
Lead Sponsor:
University of Alabama at Birmingham
Collaborators:
Children's Hospital of Philadelphia Congressionally Directed Medical Research Programs