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
CTNI-37. EFFICACY OF ONC201 IN PATIENTS WITH ONC201 FOR 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, Taylor L, Graber J, Kilburn L, Dixit K, Lu G, Allen J, Batchelor T, Lassman A, Wen P. CTNI-37. EFFICACY OF ONC201 IN PATIENTS WITH ONC201 FOR RECURRENT H3 K27M-MUTANT DIFFUSE MIDLINE GLIOMA. Neuro-Oncology 2020, 22: ii50-ii51. PMCID: PMC7651099, DOI: 10.1093/neuonc/noaa215.204.Peer-Reviewed Original ResearchH3 K27M-mutant diffuse midline gliomaDiffuse midline gliomaRANO criteriaMidline gliomaFrequent drug-related adverse eventsDrug-related adverse eventsCentral radiology reviewContrast-enhancing diseaseObjective response rateProgression-free survivalCerebrospinal fluid disseminationGrade nauseaRadiographic regressionPrior radiationOverall survivalDismal prognosisDRD2 antagonistsMedian agePrimary lesionRadiological reviewMedian timeAdverse eventsT1 contrast enhancementClpP agonistsClinical studiesSingle-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 radiationClinical efficacy of ONC201 in thalamic H3 K27M-mutant glioma.
Kawakibi A, Tarapore R, Gardner S, Kurz S, Wen P, Arrillaga-Romany I, Batchelor T, Butowski N, Sumrall A, Shonka N, Harrison R, De Groot J, Mehta M, Odia Y, Hall M, Cloughesy T, Ellingson B, Umemura Y, Allen J, Koschmann C. Clinical efficacy of ONC201 in thalamic H3 K27M-mutant glioma. Journal Of Clinical Oncology 2020, 38: 3617-3617. DOI: 10.1200/jco.2020.38.15_suppl.3617.Peer-Reviewed Original ResearchH3 K27M-mutant gliomasNon-recurrent patientsGlioma patientsRecurrent patientsClinical efficacyTreatment discontinuation due to toxicityDiscontinued due to toxicityMedian duration of responseH3 K27M-mutantDose-limiting toxicityDuration of responseDiffuse midline gliomaExpression of DRD2Wild-type gliomasAntitumor responseMedian followMidline gliomaPrimary tumorRecurrent diseaseDRD2 expressionMedian durationDRD2 antagonistsMedian ageTumor DNAMedian time