Actively Recruiting

Early Phase 1
Age: 0 - 21Years
All Genders
NCT04655404

A Pilot Study of Larotrectinib for Newly-Diagnosed High-Grade Glioma With NTRK Fusion

Led by Nationwide Children's Hospital · Updated on 2026-03-18

15

Participants Needed

21

Research Sites

816 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This is a pilot study that will evaluate disease status in children that have been newly diagnosed high-grade glioma with TRK fusion. The evaluation will occur after 2 cycles of the medication (Larotrectinib) have been given. The study will also evaluate the safety of larotrectinib when given with chemotherapy in your children; as well as the safety larotrectinib when given post-focal radiation therapy.

CONDITIONS

Official Title

A Pilot Study of Larotrectinib for Newly-Diagnosed High-Grade Glioma With NTRK Fusion

Who Can Participate

Age: 0 - 21Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients must be 21 years old or younger at enrollment.
  • Newly diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) with tumor confirmed to have NTRK fusion by certified lab testing.
  • Histologically verified high-grade glioma such as anaplastic astrocytoma, glioblastoma, or H3 K27-mutant diffuse midline glioma.
  • Patients with disseminated DIPG or HGG eligible only if receiving chemotherapy without craniospinal radiation.
  • Patients with primary spinal tumors eligible if receiving chemotherapy or focal radiation without craniospinal radiation.
  • Surgical cohort: patients with prior biopsy and planned definitive surgery, excluding DIPG.
  • Karnofsky performance score ≥50% for patients older than 16 years; Lansky score ≥50 for patients 16 years or younger.
  • No prior anti-cancer chemotherapy; prior corticosteroid use allowed.
  • Adequate bone marrow, renal, liver, pulmonary, and neurologic function as defined.
  • Written informed consent/assent obtained per institutional guidelines.
Not Eligible

You will not qualify if you...

  • Pregnant or breast-feeding women; pregnancy tests required for post-menarchal girls.
  • Use of effective contraception required for males and females of reproductive potential.
  • Prior or current use of other investigational drugs.
  • Prior or current treatment with other anti-cancer agents including chemotherapy, immunotherapy, monoclonal antibodies, biologics, or targeted therapy.
  • Active, uncontrolled systemic bacterial, viral, or fungal infection.
  • Prior solid organ transplantation.
  • Malabsorption syndrome or conditions affecting oral drug absorption.
  • Treatment with strong CYP3A4 inhibitors or inducers from 7 days before enrollment through study end.
  • Inability to comply with safety monitoring requirements as judged by the investigator.

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 21 locations

1

Children's Hospital Colorado

Aurora, Colorado, United States, 80045

Actively Recruiting

2

Children's National Medical Center

Washington D.C., District of Columbia, United States, 20010

Actively Recruiting

3

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

Chicago, Illinois, United States, 60611

Withdrawn

4

Dana-Farber Cancer Institute

Boston, Massachusetts, United States, 02215

Actively Recruiting

5

Duke University Health System

Durham, North Carolina, United States, 27708

Actively Recruiting

6

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Actively Recruiting

7

Nationwide Children's Hospital

Columbus, Ohio, United States, 43235

Actively Recruiting

8

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

9

Texas Children's Hospital

Houston, Texas, United States, 77030

Actively Recruiting

10

Seattle Children's Hospital

Seattle, Washington, United States, 98105

Actively Recruiting

11

Sydney Children's Hospital

Randwick, New South Wales, Australia, 2031

Actively Recruiting

12

Queensland Children's Hospital

South Brisbane, Queensland, Australia, 4101

Actively Recruiting

13

Perth Children's Hospital

Perth, Western Australia, Australia, 6000

Actively Recruiting

14

The Hospital for Sick Children (SickKids)

Toronto, Ontario, Canada, M5G1X8

Actively Recruiting

15

Montreal Children's Hospital

Montreal, Quebec, Canada, H4A3J1

Actively Recruiting

16

Hopp Children's Cancer Center at NCT Heidelberg (KiTZ)

Heidelberg, Baden-Wurttemberg, Germany, 69120

Actively Recruiting

17

University Medical Center Augsburg

Augsburg, Germany

Actively Recruiting

18

University Hospital Berlin

Berlin, Germany

Actively Recruiting

19

University Hospital Koln

Cologne, Germany

Actively Recruiting

20

University Medical Center Gottingen

Göttingen, Germany

Actively Recruiting

21

Starship Children's Hospital

Auckland, Grafton, New Zealand, 1023

Actively Recruiting

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Research Team

K

Kelsey H Troyer, PhD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

2

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