CONNECT 1903 - Larotrectinib for Children with Newly-Diagnosed HGG with NTRK Fusion

What is the Purpose of this Study?

We want to find out if a study drug called larotrectinib is an effective treatment for brain tumors in children and young adults. We want to learn how well it works to shrink brain tumors when used alone or when given with standard chemotherapy or after radiation therapy.

What is the Condition Being Studied?

High-grade Glioma (HGG) with a certain abnormal gene called NTRK

Who Can Participate in the Study?

Children and young adults ages 1-21 who:

  • Have a newly diagnosed high-grade glioma, including diffuse intrinsic pontine gliomas (DIPG), that has been tested to confirm the presence of an abnormal NTRK gene in the tumor

For more information about who can participate in this study, please contact the study team at 919-684-5301.

Age Group

What is Involved?

If you choose to join the study, you or your child will:

  • Take the study drug (larotrectinib) by mouth as either a pill or liquid
  • Be evaluated by the study doctor after 2 cycles of the study drug (one cycle equals 28 days) to determine the next course of treatment
  • Continue to take the study drug on its own, or combined with standard chemotherapy, or on its own following radiation treatment based on the study doctor's evaluation after the first 2 cycles

The length of treatment in the study will vary and depends on how well you or your child are doing and which treatment the doctor thinks is best for you or your child. You or your child will visit our clinic at least every 28 days to see the study doctor. Visits might be more frequent depending on treatment and how you/your child are feeling.

There is also a surgical cohort of the study where tumor samples and blood samples will be collected and sent to researchers to determine if larotrectinib is found in the tumor and how much is in the blood.

Study Details

Full Title
A Pilot and Surgical Study of Larotrectinib for Treatment of Children with Newly-Diagnosed High-Grade Glioma with NTRK Fusion
Principal Investigator
Rory David Deutsch Distinguished Professor of Neuro-Oncology
Protocol Number
IRB: PRO00104689
NCT: NCT04655404
Phase Pilot
Enrollment Status