Targeted drug shows promise for rare childhood brain tumors

NCT ID NCT04655404

Recruiting now Disease control Sponsor: Nationwide Children's Hospital Source: ClinicalTrials.gov ↗

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.

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.

anaplastic astrocytoma diffuse intrinsic pontine glioma diffuse midline glioma, H3 K27M-mutant glioblastoma glioma malignant glioma

As listed by the trial registrant

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

DIFFUSE INTRINSIC PONTINE GLIOMA HIGH GRADE GLIOMA

Contacts and locations

Locations

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WITHDRAWN

Chicago, Illinois, 60611, United States
Children's Hospital Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's National Medical Center
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Dana-Farber Cancer Institute
RECRUITING

Boston, Massachusetts, 02215, United States
Duke University Health System
RECRUITING

Durham, North Carolina, 27708, United States
Hopp Children's Cancer Center at NCT Heidelberg (KiTZ)
RECRUITING

Heidelberg, Baden-Wurttemberg, 69120, Germany
Montreal Children's Hospital
RECRUITING

Montreal, Quebec, H4A3J1, Canada
Nationwide Children's Hospital
RECRUITING

Columbus, Ohio, 43235, United States
Perth Children's Hospital
RECRUITING

Perth, Western Australia, 6000, Australia
Queensland Children's Hospital
RECRUITING

South Brisbane, Queensland, 4101, Australia
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
Starship Children's Hospital
RECRUITING

Auckland, Grafton, 1023, New Zealand
Sydney Children's Hospital
RECRUITING

Randwick, New South Wales, 2031, Australia
Texas Children's Hospital
RECRUITING

Houston, Texas, 77030, United States
The Hospital for Sick Children (SickKids)
RECRUITING

Toronto, Ontario, M5G1X8, Canada
University Hospital Berlin
RECRUITING

Berlin, Germany
University Hospital Koln
RECRUITING

Cologne, Germany
University Medical Center Augsburg
RECRUITING

Augsburg, Germany
University Medical Center Gottingen
RECRUITING

Göttingen, Germany

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