ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study (Chimerix ACTION)

This research study is studying ONC201 as a possible treatment for newly diagnosed H3 K27M-mutant Diffuse Glioma. The purpose of this research is to see if ONC201 is safe and effective for the treatment of brain cancer. The researchers want to confirm the right dose levels of ONC201 with randomizing patients in 3 study treatment groups and find out what effects (good and bad) ONC201 has on people and their condition.

Inova Schar Cancer Institute
A division of Inova Fairfax Hospital
8081 Innovation Park Dr.
Fairfax, VA 22031

• Age 18 years or older
• Body weight ≥ 10 kg at time of randomization
• Histologically diagnosed H3 K27M-mutant diffuse glioma (new diagnosis). Detection of a missense K27M mutation in any histone H3-encoding gene detected by testing of tumor tissue
• Completed standard frontline radiotherapy within 2 to 6 weeks prior to randomization
• Additional eligibility in protocol

• Primary spinal tumor
• Pregnancy/breastfeeding
• Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons
• Evidence of leptomeningeal spread of disease or cerebrospinal fluid dissemination
• Any known concurrent malignancy
• New lesion(s) outside of the radiation field
• Received whole-brain radiotherapy
• Received proton therapy for glioma
• Additional ineligibility in protocol

For additional information, please go to: https://clinicaltrials.gov/ct2/show/NCT05580562