NCT06188741 · University of Alabama at Birmingham
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
(NF114)
What this study 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.
View original scientific 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.
Interventions
DRUG
Selumetinib
Selumetinib (KoselugoTM) at the FDA approved dose of 25 mg/m2/dose PO BID
Primary outcome measures
Progression free survival (PFS)
Time frame: 60 months
Progression free survival (PFS) in the group treated with selumetinib compared to those in the observation group
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 institution within 1 year prior to consent. 5. Written informed consent must be obtained from the legal guardians of all participants \<18 years of age.
Exclusion criteria
- Presence of a known, symptomatic PN with or without previous MRI imaging. 2. 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
Where
- Birmingham, Alabama
- Los Angeles, California
- Palo Alto, California
- Washington D.C., District of Columbia
- Chicago, Illinois
- Indianapolis, Indiana
- Baltimore, Maryland
- Bethesda, Maryland
- Boston, Massachusetts
- Rochester, Minnesota
- St Louis, Missouri
- Cincinnati, Ohio
And 2 more locations — see the full list below.
Collaborators
Children's Hospital of Philadelphia, Congressionally Directed Medical Research Programs
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 29, 2026 · Source of record for eligibility and locations