Phase 1 Study of Oral ONC206 for Treatment of Recurrent and Rare Primary Central Nervous System Tumors

What this Trial Is About

Researchers are evaluating the safety and tolerability of oral ONC206 in patients with recurrent, primary central nervous system CNS tumors. This Phase 1, open-label study aims to find the maximum tolerated dose MTD and observe dose-limiting toxicities DLTs in patients who have no standard treatment options left. The trial includes various rare and recurrent CNS neoplasms such as glioblastoma, anaplastic astrocytoma, ependymoma, and medulloblastoma among others. Participants receive ONC206 orally in escalating dose levels following a 33 design over 28-day cycles. Dose adjustments and frequency changes may be made based on safety and pharmacokinetic data. The study includes up to 11 dose levels and approximately 46 patients to determine the MTD. Toxicities are monitored closely during each 28-day treatment cycle, with safety evaluations after each cohort to guide dose escalation decisions. During the study, participants undergo physical exams, vital sign checks, ECGs, blood tests, and adverse event monitoring at every visit. Tumor response is measured using established criteria, and patients provide tumor tissue samples for biomarker analysis. Study staff assess safety, tolerability, and potential toxic effects throughout treatment and for 30 days after the last dose. Total participation duration varies based on dose escalation and individual patient response.

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