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ONC201 for Brain Tumor (ACTION Trial)

Phase 3
Recruiting
Research Sponsored by Chimerix
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from date of randomization up to 44 months
Awards & highlights
Pivotal Trial

Summary

This trial will test if a new drug can improve survival for people with a specific type of brain tumor who have had radiation therapy.

Who is the study for?
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.
What is being tested?
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).
What are the potential side effects?
While specific side effects for ONC201 aren't listed here, common side effects from cancer treatments may include fatigue, nausea, blood count changes increasing infection risk and potential allergic reactions to medication components.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from date of randomization up to 44 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and from date of randomization up to 44 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Overall survival (OS)
Progression free survival (PFS) as assessed by using RANO-HGG criteria
Secondary study objectives
Change from baseline in clinical laboratory parameters
Corticosteroid response
Incidence of adverse events
+2 more

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: ONC201 Twice Weekly GroupExperimental Treatment1 Intervention
Group II: ONC201 Once Weekly GroupExperimental Treatment1 Intervention
Group III: Placebo GroupPlacebo Group1 Intervention

Find a Location

Who is running the clinical trial?

ChimerixLead Sponsor
41 Previous Clinical Trials
3,637 Total Patients Enrolled

Media Library

ONC201 Twice Weekly Group Clinical Trial Eligibility Overview. Trial Name: NCT05580562 — Phase 3
Brain Tumor Research Study Groups: ONC201 Twice Weekly Group, ONC201 Once Weekly Group, Placebo Group
Brain Tumor Clinical Trial 2023: ONC201 Twice Weekly Group Highlights & Side Effects. Trial Name: NCT05580562 — Phase 3
ONC201 Twice Weekly Group 2023 Treatment Timeline for Medical Study. Trial Name: NCT05580562 — Phase 3
~204 spots leftby Aug 2026