Brain Tumours

Investigator-assessed Best Confirmed Response a by Tumor Type Tumor type High-grade glioma n = 8 Low-grade glioma n = 15 LCH n = 2 Other solid tumor n = 2 Total N = 27 Best response, n Complete response (CR) 3 (2AA, 1 APXA) 2 5 Partial response (PR) 2 (GBM, AGG) 5 (2 PA, PMA, GG, LGG) 7 Stable disease (SD) 7 (3 PA, GG, PXA, 2 LGG) 1 (PTC) 5 Progressive disease 3 (GBM, AG, AA) 1 (PA) 1 (NB) 5 Not evaluable 2 (GG b, GG c ) 5 Response rate, n (%) CR+PR 5 (63) 5 (33) 2 (100) 0 12 (44) a Extracranial solid tumors assessed by RECIST (Response Evaluation Criteria In Solid Tumors); brain tumors assessed by RANO (Response Assessment in Neuro-Oncology); b missing data in eCRF: PI reports SD weeks 8-16; c missing data in eCRF: PI reports PR weeks 16-24. AA , anaplastic astrocytoma; AG , anaplastic glioma; AGG , anaplastic ganglioglioma; APXA , anaplastic pleomorphic xanthoastrocytoma ; GBM , gliobastoma multiforme; GG , ganglioglioma; LGG , low grade glioma; NB , neuroblastoma; PA , pilocytic astorcytoma; PMA , pilomyxoid astrocytoma; PTC , papillary thyroid carcinoma; PXA , pleomorphic xanthoastrocytoma. 114