The ACTION Study: ONC201 for H3 K27M-mutant Diffuse Glioma

This study, called the ACTION Study, is testing a drug called dordaviprone (ONC201) in people with a type of brain tumor called H3 K27M-mutant diffuse glioma. This is a new diagnosis of glioma, and you must have already completed standard radiation therapy. Researchers want to see if dordaviprone (ONC201) can help people live longer after their initial treatment. Some participants will receive dordaviprone (ONC201), and others will receive a placebo (an inactive substance that looks like the drug). The study is looking at how long people live overall. This is an international study planning to enroll 510 participants.

Study design
This is a randomized, double-blind (meaning neither you nor your doctor will know if you're getting the drug or placebo), placebo-controlled study with 510 planned participants.
What's involved
Participants will receive dordaviprone (ONC201) or placebo capsules on specific dosing days. The dose will be adjusted based on body weight.
Compensation
Not stated in the trial record.
Follow-up
Your overall survival will be assessed from the date you are assigned to a group until your death, for up to approximately 44 months.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05580562

ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)

Recruiting
PHASE3All AgesInterventionalTreatment
Jazz Pharmaceuticals
~510 participants
Updated 2026-04-16 on ClinicalTrials.gov
What's tested:Dordaviprone (ONC201)Dordaviprone (ONC201) + PlaceboPlacebo

At a glance

Recruiting sites
94 of 162 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall survival (OS)
Measured over From date of randomization until date of death from any cause, assessed up to approximately 44 months
H3 K27M
Glioma
162 sites across 78 states
California9
Spain8
New York7
Texas6
Israel6
Japan6
Florida5
Arizona4
Clinical Trial Disclosure & Transparency
Email the study team

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  • Overall survival (OS)From date of randomization until date of death from any cause, assessed up to approximately 44 months

    Overall Survival is defined as the time from randomization to death due to any cause.