ONC201 for Brain Tumor
(ACTION Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called ONC201 for individuals with a specific type of brain tumor known as H3 K27M-mutant diffuse glioma. The researchers aim to determine if ONC201 can extend patients' lives and delay disease progression after completing their initial radiation therapy. Participants will receive either ONC201 once or twice a week or a placebo (a non-active treatment) for comparison. Those recently diagnosed with this tumor type and who have completed their first radiation therapy may be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.
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.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that ONC201 is generally safe for people. Studies have found that patients with brain tumors similar to the one under study have tolerated it well. Earlier studies reported only a few serious side effects, with most being mild, such as fatigue or nausea. Additionally, the FDA has approved ONC201 for treating certain brain tumors, indicating its safety has been reviewed for similar conditions. While ongoing research remains important, current evidence supports its safety for patients.12345
Why do researchers think this study treatment might be promising for brain tumors?
Researchers are excited about ONC201 for brain tumors because it offers a novel approach compared to current treatments like surgery, radiation, and chemotherapy. Unlike traditional therapies that often have broad effects on the body, ONC201 specifically targets a pathway responsible for tumor growth, potentially leading to fewer side effects. It also has a unique mechanism of action that selectively induces cell death in tumor cells without harming healthy ones. This targeted approach not only promises better efficacy but also aims to improve the quality of life for patients by minimizing the harsh side effects associated with conventional treatments.
What evidence suggests that ONC201 might be an effective treatment for brain tumors?
Research has shown that ONC201 may help treat H3 K27M-mutant glioma, a type of brain tumor. Studies have found that ONC201 can slow tumor growth and might extend the time patients live without disease progression. Specifically, one study reported that patients taking ONC201 lived about 9 months without their cancer worsening. Another study found that ONC201 was effective in various types of cancer, suggesting potential benefits for gliomas as well. In this trial, participants will be assigned to different groups, with some receiving ONC201 either once or twice weekly, while others receive a placebo. Overall, these findings suggest that ONC201 could be a helpful treatment for this aggressive brain tumor.12367
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Radiotherapy
Participants complete standard frontline radiotherapy
Treatment
Participants receive ONC201 or placebo following radiotherapy
Follow-up
Participants are monitored for overall survival and progression-free survival
What Are the Treatments Tested in This Trial?
Interventions
- ONC201
Find a Clinic Near You
Who Is Running the Clinical Trial?
Jazz Pharmaceuticals
Lead Sponsor
Bruce C. Cozadd
Jazz Pharmaceuticals
Chief Executive Officer since 2009
BA in Economics from Yale University, MBA from Stanford University
Dr. Austin
Jazz Pharmaceuticals
Chief Medical Officer since 2023
MD from the Royal College of Surgeons in Ireland
Chimerix
Lead Sponsor