Phase 3 Study Comparing ONC201 and Everolimus to Treat Diffuse Intrinsic Pontine Glioma and Diffuse Midline Glioma in Patients Older Than 6 Months

What this Trial Is About

Researchers are investigating treatment options for Diffuse Intrinsic Pontine Glioma DIPG and related diffuse midline gliomas with H3K28M mutation or similar molecular features. This phase 3, randomized, open-label trial compares the effects of ONC201 versus everolimus on progression-free survival. The study aims to determine whether ONC201 provides better outcomes than everolimus, focusing on patients including newly diagnosed DIPG and non-DIPG diffuse midline glioma cases. Participants are assigned to one of two treatment groups one takes everolimus tablets daily at a dose capped at 10 mg, and the other takes ONC201 capsules twice a week with dose limits. Both treatments continue until tumor progression, unacceptable side effects, or withdrawal of consent. Patients experiencing disease relapse may switch to the other treatment after a 7-day washout period. All patients also receive radiotherapy over six weeks, starting within weeks of biopsy or surgery, with options for reirradiation at progression. During the study, participants undergo central reviews of disease status, and treatment effects are monitored through clinical, radiological, and pathological assessments. Outcome measures include progression-free survival up to two years after the last patient joins, overall survival, complications from biopsy procedures, and safety profiles of the drugs monitored for up to five years. Participants health status and responses to treatments are regularly evaluated, ensuring careful observation throughout the trial period.

Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0