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.
Disclaimer
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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Study contacts
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Contact
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Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20010, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Childrens of Alabama
RECRUITINGBirmingham, Alabama, 35233, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Childrens Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229-, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Johns Hopkins University
RECRUITINGBaltimore, Maryland, 21231, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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National Cancer Institute/ National Institutes of Health
RECRUITINGBethesda, Maryland, 20892, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Riley Hospital for Children/Indiana University
RECRUITINGIndianapolis, Indiana, 46202, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Stanford University
RECRUITINGPalo Alto, California, 94304, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Chicago
RECRUITINGChicago, Illinois, 63637, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Texas, Southwestern
RECRUITINGDallas, Texas, 75390, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Washington University - St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact