2025
Efficacy and safety of dordaviprone (ONC201) in prospective clinical trials of adult and pediatric recurrent H3 K27M-mutant diffuse glioma patients.
Sumrall A, Allen J, Bagley S, Brundage T, Butowski N, Clymer J, Haggiagi A, Koschmann C, Kurz S, MacDonald T, Majd N, Mueller S, Ramage S, Tarapore R, Thomas R, Umemura Y, Zaky W, Odia Y. Efficacy and safety of dordaviprone (ONC201) in prospective clinical trials of adult and pediatric recurrent H3 K27M-mutant diffuse glioma patients. Journal Of Clinical Oncology 2025, 43: 10017-10017. DOI: 10.1200/jco.2025.43.16_suppl.10017.Peer-Reviewed Original ResearchTreatment-related adverse eventsTime to responseProspective clinical trialPartial responseRANO criteriaArm BTumor regressionDiffuse gliomasH3 K27M-mutant diffuse midline gliomaClinical trialsH3 K27M-mutant gliomasIntegrated analysis of patientsLansky performance statusTreatment-related deathsDiffuse midline gliomaAnalysis of patientsDiffuse glioma patientsClinical trial armsInvestigator-assessedPFS ratesOS ratesRadiographic responseSpinal tumorsCSF disseminationLeptomeningeal disease
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 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
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-upONC201Single agent ONC201 in adult recurrent H3 K27M-mutant glioma.
Arrillaga I, Kurz S, Sumrall A, Butowski N, Harrison R, De Groot J, Shonka N, Lieberman F, Odia Y, Tarapore R, Merdinger K, Allen J, Oster W, Mehta M, Cloughesy T, Chi A, Lassman A, Batchelor T, Wen P. Single agent ONC201 in adult recurrent H3 K27M-mutant glioma. Journal Of Clinical Oncology 2019, 37: 3005-3005. DOI: 10.1200/jco.2019.37.15_suppl.3005.Peer-Reviewed Original ResearchH3 K27M-mutant gliomasResponse of stable diseaseStable diseaseMedian followTumor regressionTreatment discontinuation due to toxicitySingle agentDiscontinued due to toxicityMedian prior linesDose-limiting toxicityTreated with single agentsCompassionate use protocolProgression-freePrior radiationPartial responseConfirmatory scanDRD2 antagonistsMedian ageAssociated with improvementsProgressive diseaseTumor lesionsOn-treatmentAdult patientsPoor prognosisNeurological symptoms
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