TABLE A2.

Eligibility Criteria for Patients Included in the Efficacy Analysis

Inclusion Criterion	Exclusion Criterion
Received at least one dose of ONC201 at 625 mg (or scaled by body weight for patients age <18 years)	DIPG and primary spinal tumors, because of imaging characteristics on gadolinium-enhanced MRI
At least age 2 years	Leptomeningeal spread, cerebrospinal fluid dissemination, atypical and nonastrocytic histologies (eg, ependymoma, ganglioma, and pleomorphic xanthoastrocytoma), or pilocytic astrocytoma and subependymal giant cell astrocytoma
Diffuse glioma with a known H3 K27M mutation confirmed by immunohistochemistry or sequencing
Tumor in midline brain structure (thalamus, hypothalamus, basal ganglia, brainstem [non-DIPG], cerebellum, cerebellar peduncle, midline cortex, corpus callosum, pineal region, optic tract, or optic chiasm)
Progressive, measurable disease on contrast-enhanced brain MRI by RANO-HGG criteria
Previous therapy with at least radiation and an interval of at least 90 days from the completion of radiation to the first dose of ONC201
Previous therapy with the following, provided that sufficient washout had elapsed:  Temozolomide (23 days),  Antibodies including bevacizumab (42 days)  Other antitumor therapies (28 days)
KPS/LPS ≥60
Stable or decreasing corticosteroid dose for at least 3 days before baseline scan

Abbreviations: DIPG, diffuse intrinsic pontine glioma; H3, histone 3; KPS, Karnofsky performance score; LPS, Lansky performance score; MRI, magnetic resonance imaging; RANO-HGG, response assessment in neuro-oncology high-grade glioma;