New pill shows promise against childhood brain tumor

NCT ID NCT05465174

Recruiting now Disease control Sponsor: Sabine Mueller, MD, PhD Source: ClinicalTrials.gov ↗

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.

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.

craniopharyngioma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CRANIOPHARYNGIOMA CRANIOPHARYNGIOMA, CHILD RECURRENT CRANIOPHARYNGIOMA

Contacts and locations

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Locations

Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Children's Cancer Centre, Monash Children's Hospital
RECRUITING

Clayton, VIC 3168, Australia
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19103, United States
Children's Minnesota
RECRUITING

Minneapolis, Minnesota, 55404, United States
Dana-Farber/Boston Children's Harvard Medical School
RECRUITING

Boston, Massachusetts, 02215, United States
Duke University Medical Center
RECRUITING

Durham, North Carolina, 27710, United States
John Hopkins University
RECRUITING

Baltimore, Maryland, 21218, United States
John Hunter Children's Hospital
RECRUITING

New Lambton Heights, New South Wales, 2310, Australia
NYU Langone Health
RECRUITING

New York, New York, 10016, United States
Perth Children's Hospital
RECRUITING

Nedlands, Western Australia, 6009, Australia
Rady Children's Hospital/University of California, San Diego
RECRUITING

San Diego, California, 92037, United States
Riley Hospital for Children at Indiana University Health
RECRUITING

Indianapolis, Indiana, 46202, United States
Royal Children's Hospital, Childrens' Cancer Centre
RECRUITING

Parkville, Victoria, 3052, Australia
Royal Hobart Hospital
RECRUITING

Hobart, Tasmania, 7000, Australia
St. Louis Children's Hospital Washington University
RECRUITING

St Louis, Missouri, 63110, United States
University of Alabama at Birmingham
RECRUITING

Birmingham, Alabama, 35294, United States
University of California, San Francisco
RECRUITING

San Francisco, California, 94143, United States

Contact Email: •••••@•••••
University of Florida
RECRUITING

Gainesville, Florida, 32611, United States
University of Michigan
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Utah
RECRUITING

Salt Lake City, Utah, 84112, United States

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