Phase 2 Trial of Selumetinib for the Prevention of Plexiform Neurofibroma Growth and Morbidity in Neurofibromatosis Type 1
University of Alabama at Birmingham
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.
Description
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…
Eligibility
- Age range
- 1–8 years
- Sex
- All
- Healthy volunteers
- No
PART 1: Inclusion Criteria: 1. Age: \> 1 (\>12 months) and ≤8 years of age at the time of study enrollment. 2. Diagnosis: Participants with a diagnosis of NF1 based on the 2021 revised consensus criteria \[52\] and 3. No known PN (prior to enrollment on Part 1). Participants 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. 4. Physical exam at your instituti…
Interventions
- DrugSelumetinib
Selumetinib (KoselugoTM) at the FDA approved dose of 25 mg/m2/dose PO BID
Locations (13)
- Childrens of AlabamaBirmingham, Alabama
- Children's Hospital of Los AngelesLos Angeles, California
- Children's National HospitalWashington D.C., District of Columbia
- Lurie Children's Hospital of ChicagoChicago, Illinois
- University of ChicagoChicago, Illinois
- Riley Hospital for Children/Indiana UniversityIndianapolis, Indiana