Actively Recruiting
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
Led by University of Alabama at Birmingham · Updated on 2026-03-12
200
Participants Needed
13
Research Sites
366 weeks
Total Duration
On this page
Sponsors
U
University of Alabama at Birmingham
Lead Sponsor
C
Children's Hospital of Philadelphia
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age greater than 1 year and up to 8 years at enrollment
- Diagnosis of Neurofibromatosis Type 1 based on 2021 revised consensus criteria
- No known plexiform neurofibroma before enrollment (may include those with clinical suspicion but no prior MRI)
- Physical exam within 1 year prior to consent
- Written informed consent from legal guardians for participants under 18
- Completed baseline whole-body MRI within 6 weeks before Part 2 enrollment
- Measurable plexiform neurofibroma of 3 mL or more in high-risk locations confirmed by study leadership
- Body surface area of at least 0.55 m2
- Lansky performance status of 70% or higher (wheelchair-bound due to non-PN NF1 conditions considered ambulatory)
- Ability to swallow whole capsules
- Adequate blood counts: neutrophils ≥1200/µL, hemoglobin ≥9 g/dL, platelets ≥100,000/µL without transfusions
- Liver function within 1.5 times upper limit of normal for bilirubin and ≤3 times for AST/ALT
- Kidney function with creatinine clearance ≥60 mL/min/1.73 m2 or normal age-based serum creatinine
- Normal cardiac function with ejection fraction ≥53% and ECG QTc ≤450 msec
- Blood pressure at or below 95th percentile for age, height, and gender, stable if on medication
- Willingness to avoid excessive sun exposure and use sunscreen
- Willingness to avoid grapefruit, Seville oranges, and related products during the study
You will not qualify if you...
- Presence of known symptomatic plexiform neurofibroma
- Previous whole-body MRI before enrollment; regional MRI without PN may be allowed
- Unable or contraindicated to undergo MRI
- Prior treatment with selumetinib or other MEK1/2 inhibitors
- Evidence of optic pathway or other gliomas, malignant peripheral nerve sheath tumor, or other tumors requiring chemotherapy, biologic therapy, or radiation
- Ongoing radiation, chemotherapy, hormonal, immunotherapy, or biologic therapy for tumors
- Prosthesis, orthopedic implants, or dental braces interfering with MRI analysis
- Use of investigational agents within 30 days
- Severe or uncontrolled systemic diseases, active infections, bleeding disorders, or renal transplant; known hepatitis B, C, or HIV
- Inability to comply with safety monitoring
- Severe nausea, vomiting, gastrointestinal diseases, or major bowel resection affecting absorption
- Vitamin E supplementation above 100% daily recommended dose
- Uncontrolled blood pressure despite medication
- Cardiac conditions including inherited coronary disease, symptomatic heart failure, cardiomyopathy, severe valvular heart disease, or history of atrial fibrillation
- Certain eye conditions including central serous retinopathy, retinal vein occlusion, radiation-related eye damage, uncontrolled glaucoma, or other significant ophthalmic abnormalities
- Severe allergy to selumetinib or related compounds
- Recent major surgery within 4 weeks before treatment
- Unresolved chronic toxicities grade 2 or higher from prior therapy except hair loss
- Use of herbal supplements or medications strongly affecting cytochrome P450 enzymes unless discontinued safely before dosing
AI-Screening
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Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 13 locations
1
Childrens of Alabama
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
3
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
4
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
5
University of Chicago
Chicago, Illinois, United States, 63637
Actively Recruiting
6
Riley Hospital for Children/Indiana University
Indianapolis, Indiana, United States, 46202
Actively Recruiting
7
Johns Hopkins University
Baltimore, Maryland, United States, 21231
Actively Recruiting
8
National Cancer Institute/ National Institutes of Health
Bethesda, Maryland, United States, 20892
Actively Recruiting
9
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
10
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
11
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, United States, 45229-
Actively Recruiting
12
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
13
University of Texas, Southwestern
Dallas, Texas, United States, 75390
Actively Recruiting
Research Team
K
Karen Cole-Plourde, BA
CONTACT
J
Juliette Southworth, BS
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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