Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms

Required: H3F3A (H3 K27M) K27M

H3 K27M-mutant diffuse gliomas are excluded unless the primary tumor is located in the pons or spinal cord, or the patient has completed front line radiation or received ONC201 therapy prior to 01 January 2023

Must have received: standard of care treatment — disease-specific

after having received established standard of care treatment for their disease and have no standard treatment options available as determined by the investigators

Cannot have received: cytotoxic chemotherapy

must not have received prior investigational or approved cytotoxic chemotherapy within 28 days prior to the first dose of study drug (Cycle 1, Day 1)

Cannot have received: nitrosourea

42 days in the case of nitrosoureas

Cannot have received: bevacizumab (bevacizumab)

42 days in the case of bevacizumab

Cannot have received: molecularly targeted agent

28 days or 5 half-lives (whichever is less; but not less than 14 days) in case of investigational or approved molecularly targeted agent

Cannot have received: radiation therapy

14 days in the case of radiotherapy

Cannot have received: hematopoietic colony-stimulating growth factors (granulocyte-CSF, granulocyte-macrophage-CSF)

Exception: Erythropoietin or darbepoetin therapy, if initiated at least 2 weeks prior to enrollment, may be continued.

Patients who have been treated with any hematopoietic colony-stimulating growth factors (CSFs) (e.g., granulocyte-CSF, granulocyte-macrophage-CSF) ≤2 weeks prior to starting study drug. Erythropoietin or darbepoetin therapy, if initiated at least 2 weeks prior to enrollment, may be continued.

Cannot have received: ONC201 or blinded study drug from an ONC201 clinical study (ONC201, placebo)

Previous receipt of ONC201, placebo, or blinded study drug from an ONC201 clinical study, or from any other source for H3 K27M-mutant diffuse glioma on or after 01 January 2023.