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Primary Purpose : Treatment
Allocation : N/A
Interventional Model : Single Group Assignment
Masking : None (Open Label)
Primary outcome measures:
Safety, Dose-finding, ORR
Selumetinib is active in recurrent, refractory, or progressive pilocytic astrocytoma harbouring common BRAF aberrations and NF1-associated paediatric low-grade glioma. These results show that selumetinib could be an alternative to standard chemotherapy for these subgroups of patients, and have directly led to the development of two Children's Oncology Group phase 3 studies comparing standard chemotherapy to selumetinib in patients with newly diagnosed paediatric low-grade glioma both with and without NF1
Fangusaro et al. 2019 Lancet Oncology 20: 1011-1022
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