The goal of this study is to determine the efficacy of the study drugs tovorafenib to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (MAPK) that this drug targets.
The main question the study aims to answer is whether tovorafenib can prolong the life of patients diagnosed with HGG, including DIPG.
Patient must have previously enrolled on TarGeT-SCR.
Age Patients must be ≥12 months and ≤39 years of age at the time of enrollment on TarGeT-SCR.
Body Surface Area (BSA) Patients must have a BSA >0.3m2.
Patients with a newly-diagnosed HGG, including DIPG, which harbor alterations in the MAPK pathway are eligible. All patients must have tumor tissue from diagnostic biopsy or resection. The diagnosis of HGG, including DIPG, must have been confirmed through TarGeT-SCR.
Pregnancy or breastfeeding.
Other Exclusion Criteria
Concomitant Medications • Corticosteroids: Patients receiving corticosteroids are eligible, but the use of corticosteroids must be reported.
Patients who are receiving duloxetine, alosetron, or theophylline (CYP1A2 inhibitors) are not eligible.
Nationwide Children's Hospital
Lead sponsor
Day One Biopharmaceuticals, Inc.
Collaborator