The purpose of this study is to learn if a new drug, ONC201 can make tumors become smaller or
go away completely. Investigators also want to learn if ONC201 can prevent new deposits of
cancer from appearing in new places in participants (metastases). A phase 2 study of ONC201
in PC-PG (pheochromocytoma-paraganglioma) and other neuroendocrine tumors will determine
whether inhibition of DRD2 (a member of the dopamine receptor family) is safe in
unresectable, recurrent, locally advanced, refractory, or metastatic neuroendocrine cancers
including PC-PG, desmoplastic small round cell tumor (DSRCT), Ewing sarcoma (PNET) or any
other neuroendicrine tumor with a catecholamine or dopamine biomarker or autocrine or
paracrine dependence on dopamine including cholangiocarcinoma and adrenal cortical carcinoma.
ONC201 is an investigational (experimental) agent and has a favorable safety profile in phase
1 and early phase 2 clinical trials in advanced cancers. This study design has been chosen to
see whether ONC201 is associated with reduction of anti-hypertension medications, safety and
significant efficacy against neuroendocrine tumors, especially PC-PG.