Akt/ERK Inhibitor ONC201 in Treating Patients with Neuroendocrine Tumors That Are Locally Advanced, Metastatic, Recurrent, Refractory, or Cannot Be Removed by Surgery

Inclusion Criteria

• Subjects must have a unresectable, recurrent, locally advanced, refractory, or metastatic neuroendocrine tumor including pheochromocytoma-paraganglioma (PC-PG), desmoplastic small round cell tumor (DSRCT), Ewing sarcoma or primitive neuroectodermal tumor (PNET), or any neuroendocrine tumor with a catecholamine or dopamine biomarker or autocrine or paracrine dependence on dopamine including cholangiocarcinoma and adrenal cortical carcinoma (ACC). N=12
• There is no limit on number of prior therapies
• Hemoglobin >= 10.0 g/dl (within 3 weeks prior to enrollment)
• Leukocytes >= 1500/mcL (within 3 weeks prior to enrollment)
• Absolute neutrophil count >= 1,000/mcL (within 3 weeks prior to enrollment)
• Platelet count >= 75000/mcL (within 3 weeks prior to enrollment)
• Total bilirubin within 1.5 x normal institutional limits (within 3 weeks prior to enrollment)
• Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT]) =< 5 x institutional upper limit of normal (within 3 weeks prior to enrollment)
• Alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 5 x institutional upper limit of normal (within 3 weeks prior to enrollment)
• Serum creatinine < 3.0 mg/dL (within 3 weeks prior to enrollment)
• >= 1 lesion detectable on CT, MRI, fludeoxyglucose F-18 (18FDG) PET-CT, or PET-MRI
• Subjects must have the ability to understand and the willingness to sign a written informed consent document
• Karnofsky or if < 16 years old Lansky Play performance status >= 60%

Exclusion Criteria

• Subjects not able to take oral drugs
• Subjects receiving any other investigational agents
• Subjects receiving cytotoxic chemotherapy
• Subjects with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, severe, uncontrolled hypertension (systolic > 150/diastolic > 100 mmHg) or other symptoms of catecholamine excess after efforts to achieve adequate alpha blockade then beta blockade
• Psychiatric illness/social situations that would limit compliance with study requirements including returning for scans, taking oral medication, home monitoring of blood pressure and heart rate, recording side effects in a self-report diary, or becoming pregnant while on study drug
• Pregnant and breast-feeding subjects
• Patients with prolactinomas