Targeted drug shows promise for rare childhood brain tumors
NCT ID NCT04655404
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial is testing the drug larotrectinib in 15 children (up to age 21) with newly diagnosed high-grade glioma or DIPG that have a specific genetic change called NTRK fusion. The study aims to see if the drug can control the disease after two cycles, and how safe it is when combined with chemotherapy or radiation. Some children will also have surgery to measure drug levels in their tumor.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Larotrectinib (a targeted cancer drug)
What this could lead to
If successful, this could point toward a new treatment option for children with hard-to-treat brain tumors that have a specific genetic change (NTRK fusion).
What could go wrong
This is a very early (Phase 1) pilot study with only 15 children, so results are preliminary. The drug may not shrink tumors or improve survival, and side effects are still being studied.
Disclaimer
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This is a summary of
the original study
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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
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
WITHDRAWNChicago, Illinois, 60611, United States
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's National Medical Center
RECRUITINGWashington D.C., District of Columbia, 20010, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Dana-Farber Cancer Institute
RECRUITINGBoston, Massachusetts, 02215, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Duke University Health System
RECRUITINGDurham, North Carolina, 27708, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hopp Children's Cancer Center at NCT Heidelberg (KiTZ)
RECRUITINGHeidelberg, Baden-Wurttemberg, 69120, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Montreal Children's Hospital
RECRUITINGMontreal, Quebec, H4A3J1, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43235, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Perth Children's Hospital
RECRUITINGPerth, Western Australia, 6000, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Queensland Children's Hospital
RECRUITINGSouth Brisbane, Queensland, 4101, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Starship Children's Hospital
RECRUITINGAuckland, Grafton, 1023, New Zealand
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Sydney Children's Hospital
RECRUITINGRandwick, New South Wales, 2031, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Hospital for Sick Children (SickKids)
RECRUITINGToronto, Ontario, M5G1X8, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University Hospital Berlin
RECRUITINGBerlin, Germany
Contact Email: •••••@•••••
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University Hospital Koln
RECRUITINGCologne, Germany
Contact Email: •••••@•••••
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University Medical Center Augsburg
RECRUITINGAugsburg, Germany
Contact Email: •••••@•••••
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University Medical Center Gottingen
RECRUITINGGöttingen, Germany
Contact Phone: •••-•••-••••