ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
Brain & Spinal Cord Tumor
0 Years and older, Male and Female
ONC201-108 (primary)
NCI-2023-02479
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 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.
Eligibility
- 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.
- Body weight = 10 kg at time of randomization.
- 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): = 10 unstained formalin-fixed paraffin-embedded (FFPE) slides from tumor tissue.]
- At least one, high-quality, contrast-enhanced MRI of the brain obtained prior to starting radiotherapy for submission to sponsor's imaging vendor for central read. For participants who had a surgical resection, this scan must be post-resection; for participants who did not have a resection, this scan may be pre- or post-biopsy.
- At least one, high-quality, contrast-enhanced MRI of the brain obtained 2 to 6 weeks after completion of frontline radiotherapy. If unable to obtain contrast-enhanced imaging due to lack of venous access after multiple attempts, a patient may still be eligible after collection of a nonenhanced MRI of the brain. [Site to also provide all available MRIs completed prior to initiating treatment with study intervention.]
- Received frontline radiotherapy
- Initiated radiotherapy within 12 weeks from the initial diagnosis of H3 K27M-mutant diffuse glioma.
- Completed radiotherapy within 2 to 6 weeks prior to randomization
- Completed standard fractionated radiotherapy (eg. 54 to 60 Gy in 28 to 33 fractions given over approximately 6 weeks or hypofractionated radiotherapy (eg. 40 Gy in 15 fractions given over approximately 3 weeks).
- Karnofsky Performance Status or Lansky Performance Status = 70 at time of randomization.
- Stable or decreasing dose of corticosteroids and anti-seizure medications for 7 days prior to randomization, if applicable. Stable steroid dose is defined as = 2 mg/day increase (based on dexamethasone dose or equivalent dose of an alternative steroid).
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