Expanded Access Program for Tovorafenib (DAY101) in Patients with RAF-Altered, Relapsed or Hard-to-Treat Low-Grade Glioma

Inclusion Criteria

• Patients aged 6 months to 25 years with low-grade glioma that has returned or is not responding to treatment.
• The tumor must have a specific genetic change known as a BRAF mutation or RAF fusion.
• Must have been diagnosed with glioma or glioneuronal tumor (Grade 1 or 2) at any point.
• Must have tried at least one previous treatment and shown tumor growth on scans.
• Must have finished any previous cancer treatments at least 4 weeks before starting this program.
• Any side effects from past treatments must be stable and mild.
• Must have recovered from any surgeries.
• Must have good blood health, including:
  • Enough white blood cells and platelets.
  • Hemoglobin levels of at least 10.0 g/dL.
• Must have good liver and kidney function.
• Thyroid function must be stable.
• Must be able to follow the treatment plan and attend clinic visits.
• Must agree to use birth control if of childbearing potential.
• Must be able to swallow tablets or take liquid medicine, or use a feeding tube.
• Parents or guardians must understand and agree to the program, and patients must agree if they are old enough.

Exclusion Criteria

• Tumor has other genetic changes like histone mutation or IDH1/2 mutations.
• Known or suspected neurofibromatosis type 1 (NF-1).
• History of major diseases that could interfere with treatment.
• Major surgery within 2 weeks before starting the program.
• Significant heart disease or recent heart attack.
• Active infections.
• Digestive issues that prevent absorbing the medication.
• Taking certain medications that interact with tovorafenib.
• Pregnant or breastfeeding, or planning to become pregnant soon.
• Severe skin issues that could worsen with treatment.
• Any other reasons deemed unsuitable by the healthcare provider.
• High levels of certain enzymes in the blood.