A Phase II Trial of the MEK Inhibitor Selumetinib for the Prevention of Plexiform Neurofibroma Growth and Morbidity

GOALS

To determine if selumetinib treatment prevents PN growth in young patients with asymptomatic PN in high-risk locations. 

DRUGS

Selumetinib (Koselugo™) at the FDA approved dose of 25mg/m²/dose PO BID.

ELIGIBILITY

Participants that are ≤8 years old with NF1

• Age: > 1 (>12 months) and ≤8 years of age at the time of study enrollment.
• Diagnosis: Patients with a diagnosis of NF1 based on the 2021 revised consensus criteria [52] and
• No known PN (prior to enrollment on Part 1). Patients for whom there is clinical suspicion for a PN (e.g., subtle facial asymmetry or large overlying hyperpigmented area) may be included in the study after discussion with the Study Chair so long as they have not previously had an MRI of the region of concern and are otherwise asymptomatic.
• Physical exam at your institution within 1 year prior to consent.
• Written informed consent must be obtained from the legal guardians of all participants <18 years of age.

PROTOCOL

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 patients with PN in high-risk locations may benefit from early medical intervention prior to the development of clinical problems. This study will determine whether patients with asymptomatic PN in high-risk locations can potentially benefit from early treatment with selumetinib  

  PROTOCOL CHAIRS

• Andrea Gross, MD

CURRENT STATUS

 Currently enrolling