ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
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 dordaviprone (ONC201) following frontline radiotherapy will extend overall survival and progression-free survival in this p
| Condition(s) | H3 K27M, Glioma |
|---|---|
| Status | Recruiting |
| Phase | Phase 3 |
| Study type | Interventional |
| Summary | 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 dordaviprone (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. |
| Who can participate | Inclusion Criteria: 1. Able to understand the study procedures and agree to participate in the study by providing written informed consent (by participant or legally authorized representative), and assent when applicable. 2. Body weight ≥ 10 kg at time of randomization. 3. 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 (immunohistochemistry \[IHC\] or next-generation sequencing \[NGS\] in a Clinical Laboratory Improvement Amendments \[CLIA\]-certified or equivalent laboratory). \[Site to provide (as available): ≥ 11 unstained formalin-fixed paraffin-embedded (FFPE) slides from tumor tissue.\] 4. At least one, high-quality, contrast-enhanced MRI of the brain obt |
| Sex | All |
| Lead sponsor | Jazz Pharmaceuticals |
| Locations | Phoenix, Arizona, United States; Phoenix, Arizona, United States; Phoenix, Arizona, United States; Phoenix, Arizona, United States; La Jolla, California, United States; Los Angeles, California, United States (+156 more sites) |
| Start date | 2023-01-23 |
| NCT ID | NCT05580562 |
| Official listing | https://clinicaltrials.gov/study/NCT05580562 |