Clinical Trials

Evaluating potential of investigational medicines

  • ONC201: Phase 3 ACTION Study

    The ACTION study is enrolling newly diagnosed diffuse glioma patients whose tumor harbors the H3 K27M-mutation. The study is a world wide randomized, double-blind, placebo-controlled Phase 3 clinical trial. Treatment with ONC201 begins shortly after completion of radiation therapy. The primary endpoint of the study is overall survival (OS). The study will take place at up to 120 sites in North America, Europe and Asia Pacific. 

    If you would like more information about the ACTION study, please contact us at [email protected]

  • ONC206

    ONC206 is second generation imipridone that has demonstrated anti-cancer activity in pre-clinical models of various central nervous system (CNS) tumors and other malignancies. ONC206 is a ClpP agonist and DRD antagonist with enhanced in vitro potency relative to ONC201. ONC206 exhibits a distinct gene expression profile as well as single agent and combinatorial efficacy with ONC201 in cells with acquired resistance to ONC201. ONC206 is effective in preclinical models of difficult-to-treat neuroendocrine tumors and high-grade gliomas. It affects some of the same downstream pathways as ONC201, including activation of the integrated stress response and inhibition of Ras signaling, leading to selective killing of tumor cells.

    The first-in-human clinical trial for adults with recurrent primary CNS tumors is ongoing at the National Institute of Health and in pediatric CNS tumors with the Pacific Pediatric Neuro-Oncology Consortium.

For Clinical Trial Options please visit