ONC201 for Brain Tumor

(ACTION Trial)

Recruiting at 157 trial locations

Overseen By Tarapore, PhD

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Chimerix

Must not be taking: Bevacizumab, Temozolomide, DRD2 antagonists, others

Disqualifiers: Spinal tumor, DIPG, Concurrent malignancy, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 44 months

450 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with ONC201 following frontline radiotherapy will extend overall survival and progression-free survival in this population. Eligible participants will have histologically diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.

Will I have to stop taking my current medications?

The trial requires that participants have a stable or decreasing dose of corticosteroids and anti-seizure medications for 7 days before joining. Some medications, like strong CYP3A4 inhibitors and inducers, must be stopped a few days or weeks before starting the trial. The protocol does not specify other medications, so it's best to discuss with the trial team.

Eligibility Criteria

This trial is for patients with a new diagnosis of H3 K27M-mutant diffuse glioma who have finished standard radiotherapy. They must understand the study, weigh at least 10 kg, and have stable health status. Exclusions include recent use of certain drugs like Temozolomide or Bevacizumab, evidence of disease spread beyond the brain, abnormal lab results, pregnancy, or any condition that could affect safety or compliance.

Inclusion Criteria

I finished my radiotherapy 2 to 6 weeks ago.

I have had a detailed MRI scan of my brain before starting radiotherapy.

My weight is at least 10 kg.

Exclusion Criteria

I have new cancer spots not in the area that was radiated.

My cancer has spread to the lining of my brain or spinal cord.

I have received proton therapy for brain cancer.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Radiotherapy

Participants complete standard frontline radiotherapy

6-8 weeks

Treatment

Participants receive ONC201 or placebo following radiotherapy

44 months

Follow-up

Participants are monitored for overall survival and progression-free survival

44 months

Treatment Details

Interventions

• ONC201

Trial Overview The ACTION Study tests whether ONC201 can extend survival after radiotherapy in patients with H3 K27M-mutant diffuse glioma. It's a Phase 3 trial where participants are randomly assigned to receive either ONC201 plus placebo or just placebo in a double-blind manner (neither doctors nor patients know who gets the drug).

Participant Groups

Group I: ONC201 Twice Weekly Group Experimental Treatment 1 Intervention

Group II: ONC201 Once Weekly Group Experimental Treatment 1 Intervention

Group III: Placebo Group Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Chimerix

Lead Sponsor

Trials

42

Recruited

4,100+