Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

Recruiting now · Phase 2

Conditions studied: Neurofibromatosis 1, Plexiform Neurofibroma

In brief

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.

Key facts

Study ID: NCT06188741
Run by: University of Alabama at Birmingham
People needed: 200
Starts: 2025-08-27
Expected to finish: 2032-09-01
Last updated by the study team: 2026-05-29

Who can join

Age: 1 and older, up to 8. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Age: > 1 (>12 months) and ≤8 years of age at the time of study enrollment.
Diagnosis: Participants with a diagnosis of NF1 based on the 2021 revised consensus criteria [52] and
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.
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.

You may not qualify if…

Presence of a known, symptomatic PN with or without previous MRI imaging.
Patients who have had previous whole-body MRI (WBMRI) are excluded from the study. However, patients who have had regional MRI(s) for an indication other than a PN and did not have a PN identified on previous MRI may still be eligible for the study.
Inability to undergo MRI and/or contraindication for MRI examinations following the MRI protocol.
Prior treatment with selumetinib or another specific MEK1/2 inhibitor.
Evidence of an optic pathway or other low-grade glioma, high grade glioma, malignant peripheral nerve sheath tumor, or other cancer/tumor requiring treatment with chemotherapy, biologic therapy or radiation therapy.
Ongoing radiation therapy, chemotherapy, hormonal therapy directed at a tumor, immunotherapy, or biologic therapy.
Clinical judgement by the investigator that the patient should not participate in the study.
PART 2:
Inclusion Criteria:
Enrolled on Part 1 of this study and completed baseline WBMRI within 6 weeks of planned enrollment on Part 2.
A measurable (≥3 mL) PN in a high-risk location as defined below (this must be confirmed by Study Chair or a member of the Study Committee prior to enrollment on Part 2).
In the head or neck (with the exception of isolated scalp lesions) OR
Within the brachial or lumbosacral plexus OR
Adjacent to high-risk structure(s), defined as:
Major ("named") blood vessel OR
Major ("named") airway OR
Hollow viscus OR
Spinal cord and foramina OR
Vital Organs (including heart, lungs, liver, spleen, etc.)
Body Surface Area (BSA): BSA ≥ 0.55 m2 [pending availability of granule formulation].
Performance status: Lansky performance ≥70%. Participants who are wheelchair bound because of paralysis or immobility secondary to a non-PN related manifestation of NF1 (such as tibial pseudarthrosis or severe scoliosis) should be considered ambulatory when they are in their wheelchair.
Able to swallow whole capsules [Pending availability of granule formulation].
Hematologic Function: Absolute neutrophil count ≥1200/µL, hemoglobin ≥9g/dL, and platelets ≥100,000/µL (without transfusions).
Hepatic Function: Bilirubin within 1.5 x the upper limit of normal for age, with the exception of those with Gilbert syndrome, and AST/ALT within ≤ 3 x upper limit of normal.
Renal Function: Creatinine clearance or radioisotope GFR ≥60ml/min/1.73 m2 or a normal serum creatinine based on age, described in the table below.

Where it is running

Childrens of Alabama — Birmingham, Alabama, United States (enrolling)
Children's Hospital of Los Angeles — Los Angeles, California, United States (enrolling)
Stanford University — Palo Alto, California, United States (enrolling)
Children's National Hospital — Washington D.C., District of Columbia, United States (enrolling)
Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
University of Chicago — Chicago, Illinois, United States (enrolling)
Riley Hospital for Children/Indiana University — Indianapolis, Indiana, United States (enrolling)
Johns Hopkins University — Baltimore, Maryland, United States (enrolling)
National Cancer Institute/ National Institutes of Health — Bethesda, Maryland, United States (enrolling)
Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
Mayo Clinic — Rochester, Minnesota, United States (enrolling)
Washington University - St. Louis — St Louis, Missouri, United States (enrolling)
Cincinnati Childrens Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
University of Texas, Southwestern — Dallas, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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