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.
Parallel
Tovorafenib
DrugTovorafenib will be given orally once weekly (QW) throughout each cycle.
Progression Free Survival Stratum A
Estimate the progression-free survival (PFS) distribution for pediatric and young adult patients (age: ≥12 months and ≤39 years) with newly-diagnosed BRAFV600 mutant HGG (Stratum A) who receive tovorafenib monotherapy post RT compared to molecularly-stratified, matched historical controls.
Overall Survival Stratum A
Estimate the overall survival (OS) distribution for pediatric and young adult patients with newly-diagnosed BRAFV600 mutant HGG (Stratum A) who receive tovorafenib post RT compared to molecularly-stratified, matched historical controls.
OS Stratum B
Describe OS for pediatric and young adult patients newly diagnosed with DIPG or DMG harboring MAPK pathway alterations who receive tovorafenib post RT (Stratum B).
PFS Stratum B
Describe PFS for pediatric and young adult patients newly diagnosed with HGG harboring MAPK alterations not included in Stratum A who receive tovorafenib post RT (Stratum B).
OS Stratum B
Describe the OS for pediatric and young adult patients newly diagnosed with primary spinal cord HGG harboring a MAPK alteration who receive tovorafenib post RT (Stratum B).
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Nationwide Children's Hospital
Lead sponsor
Day One Biopharmaceuticals, Inc.
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
