NCT03295396

ONC201 in Adults With Recurrent H3 K27M-mutant Glioma

A Phase II, Open-label Study of ONC201 in Adults With Recurrent High-grade Glioma

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 73 (actual)
• Sponsor: Jazz Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This was a Phase 2, open-label, 2-arm study of dordaviprone (ONC201) in patients with recurrent H3 K27M- mutant glioma. The primary assessment of dordaviprone (ONC201) involved evaluating its anti-tumor activity through the overall response rate according to the Response Assessment in Neuro-Oncology (RANO) criteria for high-grade glioma (HGG).

Detailed description

This study included 2 arms: * Arm A included patients with recurrent H3 K27M-mutant glioma. * Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following: * Primary malignant lesion located in the pons or spinal cord. * Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA). * Prior bevacizumab treatment of \>4 doses of \>7.5 mg/kg Patients received dordaviprone (ONC201) 625 mg once weekly. The primary assessment of dordaviprone (ONC201) involved evaluating its anti-tumor activity through the overall response rate according to the Response Assessment in Neuro-Oncology (RANO) criteria for high-grade glioma (HGG). Safety was also assessed, with evaluations including the reporting of adverse events, as well as measurements of vital signs and clinical laboratory results. This study was terminated by an administrative protocol amendment (17 January 2023). The decision to terminate the study was not related to any safety concerns with dordaviprone (ONC201). Before the study was terminated, a total of 73 patients were enrolled and received at least 1 dose of dordaviprone (ONC201).

Conditions

• Glioma

Interventions

Type	Name	Description
DRUG	Dordaviprone (ONC201)	Dordaviprone (ONC201) is a central nervous system (CNS)-penetrant, small-molecule imipridone that acts as a mitochondrial caseinolytic protease P (ClpP) agonist and a dopamine receptor D2 (DRD2) antagonist.

Timeline

• Start date: 2017-10-30
• Primary completion: 2022-12-31
• Completion: 2023-07-19
• First posted: 2017-09-27
• Last updated: 2025-07-28
• Results posted: 2025-07-28

Locations

9 sites across 1 country: United States

Regulatory

• FDA-regulated drug study