Expanded Access to ONC201 for Patients with H3 K27M-mutant and/or Midline Gliomas

Brief description of study

The purpose of this research study is to provide ONC201 to patients with specific forms of brain cancer who cannot access ONC201 by participating in a clinical trial. ONC201 is a newly discovered compound that may stop cancer cells from growing. This drug has been shown in laboratory experiments to use a new mechanism that induces a stress response to kill brain tumor cells but not normal cells. Information from those other research studies suggests that this agent may help to stop cancer cells from growing. ONC201 is “investigational”, which means that the FDA (the U.S. Food and Drug Administration) has not approved ONC201 as a treatment for this condition, but the FDA has given its permission to test this agent in the current trial.


Clinical Study Identifier: s20-01692
ClinicalTrials.gov Identifier: NCT04617002
Principal Investigator: Sharon L Gardner.


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