2024
ONC201 (Dordaviprone) in Recurrent H3 K27M–Mutant Diffuse Midline Glioma
Arrillaga-Romany I, Gardner S, Odia Y, Aguilera D, Allen J, Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot J, Dixit K, Graber J, Haggiagi A, Harrison R, Kheradpour A, Kilburn L, Kurz S, Lu G, MacDonald T, Mehta M, Melemed A, Nghiemphu P, Ramage S, Shonka N, Sumrall A, Tarapore R, Taylor L, Umemura Y, Wen P. ONC201 (Dordaviprone) in Recurrent H3 K27M–Mutant Diffuse Midline Glioma. Journal Of Clinical Oncology 2024, 42: 1542-1552. PMID: 38335473, PMCID: PMC11095894, DOI: 10.1200/jco.23.01134.Peer-Reviewed Original ResearchConceptsH3 K27M-mutant diffuse midline gliomaDiffuse midline gliomaDuration of responseTime to responseHigh-grade gliomasLow-grade gliomasMidline gliomaMedian duration of responseMedian time to responseTreatment-emergent adverse eventsEnd pointsBlinded independent central reviewCorticosteroid dose reductionIndependent central reviewSecondary end pointsClinically meaningful efficacyRadiographic end pointsSpinal tumorsDose reductionDismal prognosisCentral reviewPerformance scoresCorticosteroid responseResponse assessmentAdverse events
2020
Single-agent ONC201 in recurrent H3 K27M-mutant diffuse midline glioma.
Arrillaga-Romany I, Kurz S, Tarapore R, Sumrall A, Butowski N, Harrison R, De Groot J, Chi A, Shonka N, Umemura Y, Odia Y, Mehta M, Nghiemphu P, Cloughesy T, Lu G, Oster W, Allen J, Batchelor T, Lassman A, Wen P. Single-agent ONC201 in recurrent H3 K27M-mutant diffuse midline glioma. Journal Of Clinical Oncology 2020, 38: 3615-3615. DOI: 10.1200/jco.2020.38.15_suppl.3615.Peer-Reviewed Original ResearchH3 K27M-mutant diffuse midline gliomaBlinded independent central reviewPhase II clinical trialDiffuse midline gliomaII clinical trialsMidline gliomaTreatment discontinuation due to toxicityClinical trialsDiffuse midline glioma patientsDiscontinued due to toxicityH3 K27M-mutant gliomasNon-contrast-enhancing regionsGlioma patientsObjective response rateDuration of responseDopamine receptor expressionIndependent central reviewContrast-enhancing lesionsH3 K27MFirst-in-classT1 post-contrastAnti-cancer small moleculesStable diseaseData cutoffPrior radiation
2019
ACTR-34. SINGLE AGENT ONC201 IN PREVIOUSLY-TREATED, PROGRESSIVE ADULT H3 K27M-MUTANT GLIOMA
Arrillaga-Romany I, Kurz S, Sumrall A, Butowski N, Harrison R, DeGroot J, Chi A, Sulman E, Shonka N, Umemura Y, Odia Y, Mehta M, Iwamoto F, Nghiemphu P, Cloughesy T, Tarapore R, Merdinger K, Oster W, Allen J, Batchelor T, Lassman A, Wen P. ACTR-34. SINGLE AGENT ONC201 IN PREVIOUSLY-TREATED, PROGRESSIVE ADULT H3 K27M-MUTANT GLIOMA. Neuro-Oncology 2019, 21: vi20-vi21. PMCID: PMC6847973, DOI: 10.1093/neuonc/noz175.077.Peer-Reviewed Original ResearchH3 K27M-mutant gliomasPhase II trialMidline gliomaII trialMedian duration of responseBlinded independent central reviewPhase II clinical trialDuration of responseMedian follow-upDopamine receptor expressionIndependent central reviewII clinical trialsIn vitro studiesRANO criteriaPrior radiationRadiographic responseDismal prognosisMedian durationMedian onsetCentral reviewPrimary endpointReceptor expressionClinical outcomesFollow-upONC201