1568-1569 / 2437
Stratum 1: Recurrent/Refractory
BRAF Aberrant Pilocytic Astrocytoma
• N=25 eligible and evaluable.
– Expansion beyond first 16 justified based on response and statistical design
– 18 BRAF duplications and 7 BRAF V600e mutations
• Best response (regardless of timeframe):
– 8 PR, 10 SD, 7 PD during treatment
– Of the 8 PRs, 6 had BRAF duplication and 2 had BRAF V600e mutation
• 5 patients still on treatment.
• 5 patients progressed while off treatment and after completing all 26 courses.