ONC201 in Adults With Recurrent H3 K27M-mutant Glioma

Phase 2 Terminated 73 enrolled Jazz Pharmaceuticals Oncoceutics, Inc. · collab

Non-randomizedSingle-groupOpen-labelTreatment

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).

Timeline

Start: 2017-10-30
Primary completion: 2022-12-31
Completion: 2023-07-19

Outcome

Outcome not reported

Stopped (Enrollment): “The Sponsor terminated the study to prioritize enrollment in a randomized Phase 3 trial of ONC201 in an earlier setting. This decision was unrelated to any safety concerns with dordaviprone (ONC201).”

Publications

Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE, Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot J, Dixit KS, Graber JJ, Haggiagi AM, Harrison RA, Kheradpour A, Kilburn LB, Kurz SC, Lu G, MacDonald TJ, Mehta M, Melemed AS, Nghiemphu PL, Ramage SC, Shonka N, Sumrall A, Tarapore RS, Taylor L, Umemura Y, Wen PY. ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma. J Clin Oncol. 2024 May 1;42(13):1542-1552. doi: 10.1200/JCO.23.01134. Epub 2024 Feb 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Dordaviprone	Small molecule	625 mg	Oral