New pill shows promise against childhood brain tumor
NCT ID NCT05465174
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing a drug called Tovorafenib for children and young adults with craniopharyngioma, a rare, non-cancerous brain tumor that can cause serious health problems. The drug is taken by mouth and works by blocking a protein that helps the tumor grow. The trial aims to see if the drug can stop the tumor from growing and improve patients' quality of life. About 57 participants with newly diagnosed or recurrent tumors will be enrolled.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Tovorafenib (a targeted drug taken orally)
What this could lead to
If successful, this could provide a new, less invasive treatment option for craniopharyngioma, potentially delaying or avoiding surgery and radiation in children and young adults.
What could go wrong
This is a small, early-phase (Phase 2) trial with only 57 participants. The drug may not work for everyone, and side effects are possible. It is not a cure, and long-term disease control is uncertain.
Disclaimer
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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.
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
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Cancer Centre, Monash Children's Hospital
RECRUITINGClayton, VIC 3168, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19103, United States
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Children's Minnesota
RECRUITINGMinneapolis, Minnesota, 55404, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Dana-Farber/Boston Children's Harvard Medical School
RECRUITINGBoston, Massachusetts, 02215, United States
Contact Email: •••••@•••••
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Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
Contact Email: •••••@•••••
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John Hopkins University
RECRUITINGBaltimore, Maryland, 21218, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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John Hunter Children's Hospital
RECRUITINGNew Lambton Heights, New South Wales, 2310, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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NYU Langone Health
RECRUITINGNew York, New York, 10016, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Perth Children's Hospital
RECRUITINGNedlands, Western Australia, 6009, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rady Children's Hospital/University of California, San Diego
RECRUITINGSan Diego, California, 92037, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Riley Hospital for Children at Indiana University Health
RECRUITINGIndianapolis, Indiana, 46202, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Children's Hospital, Childrens' Cancer Centre
RECRUITINGParkville, Victoria, 3052, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Hobart Hospital
RECRUITINGHobart, Tasmania, 7000, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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St. Louis Children's Hospital Washington University
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35294, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California, San Francisco
RECRUITINGSan Francisco, California, 94143, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Email: •••••@•••••
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University of Florida
RECRUITINGGainesville, Florida, 32611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Utah
RECRUITINGSalt Lake City, Utah, 84112, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••