NCT02525692

Oral ONC201 in Adult Recurrent Glioblastoma

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 84 (actual)
• Sponsor: Jazz Pharmaceuticals · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

This was a Phase 2, open-label, 6-arm, multi-center study of dordaviprone (ONC201) in patients with recurrent glioblastoma (Arms A, B, and C), H3 K27M-mutant diffuse glioma (Arm D), or diffuse midline glioma (Arms E and F). The primary objective of this study was the assessment of dordaivprone (ONC201) anti-tumor activity through progression-free survival at 6 months using Response Assessment in Neuro-Oncology (RANO) criteria for high-grade glioma (HGG).

Detailed description

This study included 6 arms: * Patients in Arm A received 625 mg oral dordaviprone (ONC201) every 3 weeks. * Patients in Arms B, C, D, E, and F received 625 mg oral dordaviprone (ONC201) every 1 week (Days 1, 8 and 15 of each cycle). * Patients in Arms C and E received salvage surgical resection of their brain tumor 1 day after the second (or more) dose of dordaviprone (ONC201). All patients underwent clinical evaluation after each cycle (defined as every 3 weeks). Neuroimaging studies (contrast-enhanced brain magnetic resonance imaging or computed tomography for patients unable to undergo MRI) were performed at baseline, 8 weeks from treatment initiation, and then every 8 weeks thereafter. Assessments of dordaviprone (ONC201) anti-tumor activity were assessed through progression-free survival at 6 months using RANO-HGG criteria. Safety was assessed through the reporting of adverse events, measurement of vital signs, electrocardiograms, 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 84 patients were enrolled and received at least 1 dose of dordaviprone (ONC201).

Conditions

• Glioblastoma
• Diffuse Midline Glioma
• H3 K27M Glioma
• Thalamic Glioma
• Infratentorial Glioma
• Basal Ganglia Glioma

Interventions

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

Timeline

• Start date: 2016-01-01
• Primary completion: 2023-04-17
• Completion: 2023-04-17
• First posted: 2015-08-17
• Last updated: 2024-12-24
• Results posted: 2024-12-24

Locations

5 sites across 1 country: United States