Pilot and Surgical Study of Larotrectinib to Treat Children and Young Adults with Newly Diagnosed High-Grade Glioma Featuring NTRK Fusion

What this Trial Is About

This research aims to evaluate disease status in children newly diagnosed with high-grade glioma that have a TRK fusion. It is a pilot study focusing on children up to 21 years old, assessing disease control and survival rates. The study also examines the safety of larotrectinib given with chemotherapy or after focal radiation therapy in this group. Participants will receive larotrectinib orally twice daily at a dose based on body surface area, in 28-day cycles. After 2 cycles of larotrectinib alone, patients showing a complete or continued complete response will continue larotrectinib as maintenance therapy for up to 12 cycles, with possible extension to 24 cycles if beneficial. Those with partial response or stable disease may receive additional chemotherapy or focal radiation depending on age and clinical factors. A surgical cohort will receive larotrectinib for 3-5 days before surgery followed by larotrectinib treatment. During the study, participants will be closely monitored for disease control using imaging and for safety by tracking side effects and adverse events. Researchers will measure drug levels in blood and tumor tissue in the surgical group. The main outcomes include disease control after 2 cycles and treatment-related side effects, with survival rates assessed over time. Participation involves regular visits, treatment cycles, and follow-up evaluations for up to several years.

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