Can a cancer drug stop nerve tumors before they cause harm?
NCT ID NCT06188741
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests whether giving selumetinib early to children with NF1, before nerve tumors cause symptoms, can prevent tumor growth and related problems. About 200 children aged 1 to 8 years with no known tumors will either receive the drug or be observed. The goal is to see if early treatment improves progression-free survival.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Selumetinib (Koselugo)
- What this could lead to
- If successful, this could show that starting treatment early prevents serious complications like blindness or nerve damage in children with NF1.
- What could go wrong
- This is a Phase 2 trial, so it is still early. The drug may not prevent tumor growth better than observation, and side effects like nausea or skin rash are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Children's Hospital of Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Childrens of Alabama
RECRUITINGBirmingham, Alabama, 35233, United States
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Cincinnati Childrens Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229-, United States
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Johns Hopkins University
RECRUITINGBaltimore, Maryland, 21231, United States
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Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
Contact Email: •••••@•••••
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National Cancer Institute/ National Institutes of Health
RECRUITINGBethesda, Maryland, 20892, United States
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Riley Hospital for Children/Indiana University
RECRUITINGIndianapolis, Indiana, 46202, United States
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Stanford University
RECRUITINGPalo Alto, California, 94304, United States
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University of Chicago
RECRUITINGChicago, Illinois, 63637, United States
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University of Texas, Southwestern
RECRUITINGDallas, Texas, 75390, United States
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Washington University - St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Light-Activated cream could slow growth of NF1 skin tumors
- Real-World study tracks Selumetinib's impact on NF1 tumors in children
- Antioxidant drug shows promise for kids with NF1
- New drug combo shows promise for rare nerve tumors
- Could a cancer drug shrink Children's brain tumors?
- Could a common supplement help kids with NF1 learn and move better?