ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors

This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors. ONC206 is a recently discovered compound that may stop cancer cells from growing. This drug has been shown in laboratory experiments to kill brain tumor cells by causing a so called "stress response" in tumor cells. This stress response causes cancer cells to die, but without affecting normal cells. ONC206 alone or in combination with radiation therapy may be effective in treating newly diagnosed or recurrent diffuse midline gliomas and other recurrent primary malignant CNS tumors.

NCT Number	NCT04732065
Lead Sponsor	Sabine Mueller, MD, PhD Collaborators: Jazz Pharmaceuticals, Mithil Prasad Foundation, Storm the Heavens Fund, The ChadTough Defeat DIPG Foundation, National Cancer Institute (NCI), Dana-Farber Cancer Institute
Conditions	Diffuse Midline Glioma (DMG), Glioblastoma, Recurrent Ependymoma, Recurrent Malignant Central Nervous System Neoplasm, Spinal Cord Glioma, World Health Organization (WHO) Grade III Glioma, CNS Tumor, Central Nervous System Tumor
Enrollment	208 participants
Start Date	2021-08-23
Primary Completion	2027-05-31 (estimated)
Study Completion	2027-07-31 (estimated)
Updated on ClinicalTrials.gov	2026-06-11