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 diseaseNeoadjuvant immunotherapy of hepatocellular carcinoma: A single-institution experience at Mount Sinai.
Feng D, Crowley F, Hapanowicz O, Venturini N, Lucas N, Wu K, Wilk J, Sadek N, Hamon P, Hennequin C, Devraj V, Thanigaimani P, Uldrick T, Miller E, Lowy I, Doroshow D, Tabrizian P, Schwartz M, Merad M, Marron T. Neoadjuvant immunotherapy of hepatocellular carcinoma: A single-institution experience at Mount Sinai. Journal Of Clinical Oncology 2025, 43 DOI: 10.1200/jco.2025.43.16_suppl.e16320.Peer-Reviewed Original ResearchTreatment-related adverse eventsRelapse-free survivalOverall response rateAnti-PD-1Single-institution experienceResectable hepatocellular carcinomaNeoadjuvant immunotherapyHepatocellular carcinomaAdverse eventsTumor necrosisPathological responseSingle-agent anti-PD-1Early-stage hepatocellular carcinomaEarly-phase clinical trialsGrade 3 hepatitisAdvanced hepatocellular carcinomaDelay of surgerySurvival of patientsCombination immunotherapyNeoadjuvant settingAdjuvant immunotherapyResected tumorMetastatic diseaseImmunotherapy regimensNeoadjuvant nivolumabTirabrutinib for the treatment of relapsed or refractory primary central nervous system lymphoma: Efficacy and safety from the phase II PROSPECT study.
Nayak L, Grommes C, Kallam A, Peereboom D, Ambady P, Mendez J, Aregawi D, Sumrall A, Omuro A, Iwamoto F, Dietrich J, Umemura Y, Munker R, Chukwueke U, Schaff L, Prados S, Takazawa A, Aoi A, Batchelor T. Tirabrutinib for the treatment of relapsed or refractory primary central nervous system lymphoma: Efficacy and safety from the phase II PROSPECT study. Journal Of Clinical Oncology 2025, 43: 2019-2019. DOI: 10.1200/jco.2025.43.16_suppl.2019.Peer-Reviewed Original ResearchPrimary central nervous system lymphomaTreatment-emergent adverse eventsTreatment-related adverse eventsDuration of responseProgression-free survivalTime to responseMedian duration of responseCentral nervous system lymphomaNervous system lymphomaTyrosine kinase inhibitorsSystem lymphomaOverall survivalResponse rateNeutrophil countProspective studyAdverse eventsTreatment optionsRefractory primary central nervous system lymphomaSecond-generation Bruton's tyrosine kinase inhibitorAggressive form of non-Hodgkin lymphomaOpen-label phase II studyDisease progressionMedian progression-free survivalPhase II prospective studyMedian time to responseLenvatinib Plus Pembrolizumab, Pemetrexed, and a Platinum as First-Line Therapy for Metastatic Nonsquamous Non–Small-Cell Lung Cancer: Phase 3 LEAP-006 Study
Herbst R, Cho B, Zhou C, Burotto M, Dols M, Sendur M, Moiseyenko V, Casarini I, Nishio M, Hui R, Pons-Tostivint E, Dudnik J, Ahmed S, Okpara C, Dutcus C, Yin L, Luo Y, Chirovsky D, Bhagwati N, Abreu D. Lenvatinib Plus Pembrolizumab, Pemetrexed, and a Platinum as First-Line Therapy for Metastatic Nonsquamous Non–Small-Cell Lung Cancer: Phase 3 LEAP-006 Study. Journal Of Thoracic Oncology 2025 PMID: 40419140, DOI: 10.1016/j.jtho.2025.05.016.Peer-Reviewed Original ResearchNonsquamous non-small-cell lung cancerProgression-free survivalMetastatic nonsquamous non-small-cell lung cancerNonsquamous NSCLCNon-small-cell lung cancerFirst-line pembrolizumabOverall survivalTargetable genetic alterationsPlacebo armGenetic alterationsLung cancerTreatment-related adverse eventsFirst-line therapyMedian follow-upDouble-blind studyPrimary endpointPembrolizumabLenvatinibAdverse eventsChemotherapyFollow-upDisease progressionSafety signalsPemetrexedMonthsSafety of tarlatamab with 6-8-h outpatient versus 48-h inpatient monitoring during cycle 1: DeLLphi-300 phase 1 substudy
Chiang A, Garcia M, Carlisle J, Dowlati A, Reguart N, Felip E, Jost P, Steeghs N, Stec R, Gadgeel S, Loong H, Jiang W, Hamidi A, Parkes A, Paz-Ares L. Safety of tarlatamab with 6-8-h outpatient versus 48-h inpatient monitoring during cycle 1: DeLLphi-300 phase 1 substudy. ESMO Open 2025, 10: 104538. PMID: 40187110, PMCID: PMC12002761, DOI: 10.1016/j.esmoop.2025.104538.Peer-Reviewed Original ResearchConceptsSmall-cell lung cancerCytokine release syndromeInpatient monitoringRate of treatment-related adverse eventsRisk of cytokine release syndromeTreated small-cell lung cancerT cell-engaging immunotherapiesTreatment-related adverse eventsDelta-like ligand 3First-in-human studyPrimary endpointSurvival outcomesMedian timeAdverse eventsLung cancerEmergency room visitsClinical trialsQ2WOutpatient monitoringSafety outcomesInpatient groupHospitalization ratesCycle 1Room visitsPatientsPhase II Trial of Chemotherapy, Cetuximab, and Erlotinib in Patients With Metastatic or Recurrent Squamous Cell Carcinoma of the Head and Neck
Bhatia A, Mehra R, Bauman J, Khan S, Wei W, Neumeister V, Sandoval‐Schaefer T, Alpaugh R, Lango M, Rimm D, Ridge J, Burtness B. Phase II Trial of Chemotherapy, Cetuximab, and Erlotinib in Patients With Metastatic or Recurrent Squamous Cell Carcinoma of the Head and Neck. Head & Neck 2025 PMID: 40166973, DOI: 10.1002/hed.28152.Peer-Reviewed Original ResearchHead and neck squamous cell cancerProgression-free survivalEpidermal growth factor receptorTyrosine kinase inhibitorsOverall survivalPhase II trial of chemotherapyMedian progression-free survivalRecurrent squamous cell carcinomaTreatment-related adverse eventsDual epidermal growth factor receptorTreated with carboplatinPhase 2 trialPhase II trialSecondary end pointsSquamous cell cancerNuclear translocation of epidermal growth factor receptorSquamous cell carcinomaTrials of chemotherapyHead and neckSmall patient sampleTranslocation of epidermal growth factor receptorGrowth factor receptorInhibited nuclear translocationEGFR blockadeChemotherapy backboneA Phase I, First-in-Human, Dose-Escalation, Expansion Trial of Cytokine-Encoding Synthetic mRNA Mixture Alone or with Cemiplimab in Advanced Solid Tumors
Bechter O, Loquai C, Champiat S, Baurain J, Grob J, Utikal J, Rottey S, Berrocal A, Hassel J, Arance A, Sanmamed M, Boers-Sonderen M, Gastman B, Gebhardt C, Delafontaine B, Sahin U, Türeci Ö, Brueck P, Abbadessa G, Marpadga R, Lee H, Yang Y, Buday B, Di Genova G, Wang H, Xia B, Lee J, Lebbe C. A Phase I, First-in-Human, Dose-Escalation, Expansion Trial of Cytokine-Encoding Synthetic mRNA Mixture Alone or with Cemiplimab in Advanced Solid Tumors. Clinical Cancer Research 2025, 31: 2358-2369. PMID: 40152791, PMCID: PMC12163594, DOI: 10.1158/1078-0432.ccr-24-1983.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overAntibodies, Monoclonal, HumanizedAntineoplastic Combined Chemotherapy ProtocolsCytokinesFemaleGranulocyte-Macrophage Colony-Stimulating FactorHumansInterferon alpha-2Interleukin-12Interleukin-15MaleMaximum Tolerated DoseMiddle AgedNeoplasmsRNA, MessengerTreatment OutcomeConceptsAdvanced solid tumorsMaximum administered doseSolid tumorsCombination therapyEscalation phaseTreated with anti-PD-1 therapyTreated with anticancer therapyAnti-PD-1 therapyTreatment-related adverse eventsDose level 8Predefined dose levelsInjection-site painFirst-in-humanPlasma cytokine concentrationsDose escalationAntitumor responsePartial responseIntratumoral administrationExpansion trialIL-15IFN-gCemiplimabAdverse eventsCytokine concentrationsDose levelsPembrolizumab Plus Docetaxel Versus Docetaxel for Previously Treated Metastatic Castration-Resistant Prostate Cancer: The Randomized, Double-Blind, Phase III KEYNOTE-921 Trial
Petrylak D, Ratta R, Matsubara N, Korbenfeld E, Gafanov R, Mourey L, Todenhöfer T, Gurney H, Kramer G, Bergman A, Zalewski P, De Santis M, Armstrong A, Gerritsen W, Pachynski R, Byun S, Retz M, Levesque E, McDermott R, Bracarda S, Manneh R, Levartovsky M, Li X, Schloss C, Poehlein C, Fizazi K. Pembrolizumab Plus Docetaxel Versus Docetaxel for Previously Treated Metastatic Castration-Resistant Prostate Cancer: The Randomized, Double-Blind, Phase III KEYNOTE-921 Trial. Journal Of Clinical Oncology 2025, 43: 1638-1649. PMID: 40043230, PMCID: PMC12058370, DOI: 10.1200/jco-24-01283.Peer-Reviewed Original ResearchMetastatic castration-resistant prostate cancerTreat metastatic castration-resistant prostate cancerRadiographic progression-free survivalAndrogen receptor pathway inhibitorsCastration-resistant prostate cancerTreatment-related adverse eventsStandard of careAdverse eventsOverall survivalDouble-blindProstate cancerMedian radiographic progression-free survivalDual primary end pointsImmune-mediated adverse eventsEnd pointsBlinded independent central reviewData cutoff dateSafety of pembrolizumabAndrogen deprivation therapyProgression-free survivalIndependent central reviewSecondary end pointsPrimary end pointConcomitant prednisoneMedian OSEfficacy and safety of larotrectinib in patients with TRK fusion gastrointestinal cancer
Qi C, Shen L, Andre T, Chung H, Cannon T, Garralda E, Italiano A, Rieke D, Liu T, Burcoveanu D, Neu N, Mussi C, Xu R, Hong D, Drilon A, Berlin J. Efficacy and safety of larotrectinib in patients with TRK fusion gastrointestinal cancer. European Journal Of Cancer 2025, 220: 115338. PMID: 40068370, DOI: 10.1016/j.ejca.2025.115338.Peer-Reviewed Original ResearchTreatment-related adverse eventsSafety of larotrectinibMicrosatellite instability-highGI cancersMedian duration of responseNext-generation sequencing testMedian overall survivalProgression-free survivalDuration of responseNTRK gene fusionsOverall response rateFirst-in-classOverall survivalMedian durationTRK inhibitorsSolid tumorsTumor typesAdverse eventsExtended survivalLarotrectinibGastrointestinal cancerPatientsHepatic cancerResponse rateCancerSafety and efficacy of immune checkpoint therapy for the treatment of patients with cardiac metastasis: a multicenter international retrospective study
Nassar A, Alaiwi S, Zarif T, Denu R, Macaron W, Abdel-Wahab N, Freeman D, Vasbinder A, Hayeck S, Anderson E, Goodman R, Johnson D, Grynberg S, Shapira R, Kwan J, Woodford R, Long G, Haykal T, Dent S, Kojima Y, Yonemor K, Tandon A, Trevino A, Akhter N, Yang E, Hui G, Drakaki A, El-Am E, Kozaily E, Al-Hader A, Farhat E, Babu P, Mittra A, Li M, Jones N, Baena J, Herrera M, Foderaro S, Nana F, Kim C, Sackstein P, Parikh K, Desai A, Smith C, Cortellini A, Pinato D, Korolewicz J, Lopetegui-Lia N, Funchain P, Choudhary A, Asnani A, Navani V, Meyers D, Stukalin I, Gallegos J, Trent J, Nusrat S, Malvar C, McKay R, Neilan T, Choueiri T, Naqash A. Safety and efficacy of immune checkpoint therapy for the treatment of patients with cardiac metastasis: a multicenter international retrospective study. Journal For ImmunoTherapy Of Cancer 2025, 13: e009364. PMID: 40032601, PMCID: PMC11877189, DOI: 10.1136/jitc-2024-009364.Peer-Reviewed Original ResearchConceptsImmune checkpoint inhibitor initiationImmune checkpoint inhibitorsTreatment-related adverse eventsObjective response rateProgression-free survivalCardiac metastasisOverall survivalRetrospective studyAnti-cytotoxic T-lymphocyte antigen 4Dose of immune checkpoint inhibitorsEfficacy of immune checkpoint therapyAnti-programmed death-1International multicenter retrospective studyMulticenter international retrospective studyT-lymphocyte antigen-4Non-small cell lung cancerMedian follow-up timeClinical outcomes of patientsICI-based regimensMultiple cardiac metastasesSolid Tumors V.1.1Immune checkpoint therapyResponse Evaluation CriteriaInternational retrospective studyMulticenter retrospective studyCLASSIFICATION OF ADVERSE EVENTS AFTER PROSTATE CANCER HYDROGEL PERIRECTAL SPACER INSERTION USING LARGE LANGUAGE MODELS
Sohoni N, Sohoni N, Sutherland R, Sundaresan V, Olivieri J, Leapman M. CLASSIFICATION OF ADVERSE EVENTS AFTER PROSTATE CANCER HYDROGEL PERIRECTAL SPACER INSERTION USING LARGE LANGUAGE MODELS. Urologic Oncology Seminars And Original Investigations 2025, 43: 62-63. DOI: 10.1016/j.urolonc.2024.12.157.Peer-Reviewed Original ResearchAdverse eventsRadiation treatment of prostate cancerTreatment-related adverse eventsTreatment of prostate cancerGold standardPerirectal hydrogel spacerTreatment-related toxicitySecondary adverse eventsPrimary adverse eventsPresence of symptomsBoard-certified urologistsHydrogel spacerProstate cancerReduced inter-rater variabilitySafety profileInter-rater variabilityRadiation treatmentSeverity gradeInterpretation of adverse eventsMAUDE databaseSpacer insertionKappa scoreClinical researchTrained reviewersCabozantinib (cabo) and nivolumab (nivo) with or without CBM588 in patients with metastatic renal cell carcinoma: Updated clinical outcomes of a phase I study.
Ebrahimi H, Meza L, Dizman N, Barragan-Carrillo R, Li X, Llamas-Quitiquit M, Hsu J, Zengin Z, Castro D, Mercier B, Zugman M, Jaime-Casas S, Chehrazi-Raffle A, Trent J, Lee P, Takahashi M, Dorff T, Caporaso G, Lee K, Pal S. Cabozantinib (cabo) and nivolumab (nivo) with or without CBM588 in patients with metastatic renal cell carcinoma: Updated clinical outcomes of a phase I study. Journal Of Clinical Oncology 2025, 43: 543-543. DOI: 10.1200/jco.2025.43.5_suppl.543.Peer-Reviewed Original ResearchProgression-free survivalTreatment-related adverse eventsClinical benefitControl armTreatment armsFollow-upImproving progression-free survivalMedian progression-free survivalMetastatic renal cell carcinomaTime of data cutoffTarget lesion sizeTreatment naive patientsMedian follow-upPhase I studyKaplan-Meier methodSecondary clinical endpointsRenal cell carcinomaKarnofsky performance statusFisher's exact testMedian OSSarcomatoid featuresStable diseaseComplete responseData cutoffPapillary histologyAvelumab first-line maintenance (1LM) in patients (pts) with advanced urothelial carcinoma (aUC) with or without diabetes mellitus (DM): Long-term outcomes from JAVELIN Bladder 100.
Gupta S, Grivas P, Park S, Petrylak D, Tyroller K, Hoffman J, Bellmunt J. Avelumab first-line maintenance (1LM) in patients (pts) with advanced urothelial carcinoma (aUC) with or without diabetes mellitus (DM): Long-term outcomes from JAVELIN Bladder 100. Journal Of Clinical Oncology 2025, 43: 869-869. DOI: 10.1200/jco.2025.43.5_suppl.869.Peer-Reviewed Original ResearchProgression-free survivalAdvanced urothelial carcinomaBest supportive carePlatinum-based chemotherapyOverall survivalDiabetes mellitusAdverse eventsAssociated with long-term efficacyImmune-related adverse eventsTreatment-related adverse eventsFirst-line maintenanceAssociated with reduced efficacyEfficacy of immunotherapyMedian follow-upProlonged overall survivalLong-term efficacyPresence of DMLong-term outcomesPost hoc exploratory analysisJAVELIN BladderMedian OSMetastatic UCUrothelial carcinomaEligible ptsPrimary endpointTransarterial chemoembolisation combined with lenvatinib plus pembrolizumab versus dual placebo for unresectable, non-metastatic hepatocellular carcinoma (LEAP-012): a multicentre, randomised, double-blind, phase 3 study
Kudo M, Ren Z, Guo Y, Han G, Lin H, Zheng J, Ogasawara S, Kim J, Zhao H, Li C, Madoff D, Ghobrial R, Kawaoka T, Gerolami R, Ikeda M, Kumada H, El-Khoueiry A, Vogel A, Peng X, Mody K, Dutcus C, Dubrovsky L, Siegel A, Finn R, Llovet J, investigators L. Transarterial chemoembolisation combined with lenvatinib plus pembrolizumab versus dual placebo for unresectable, non-metastatic hepatocellular carcinoma (LEAP-012): a multicentre, randomised, double-blind, phase 3 study. The Lancet 2025, 405: 203-215. PMID: 39798578, DOI: 10.1016/s0140-6736(24)02575-3.Peer-Reviewed Original ResearchConceptsEastern Cooperative Oncology GroupNon-metastatic hepatocellular carcinomaProgression-free survivalTreatment-related adverse eventsPembrolizumab groupPhase 3 studyTransarterial chemoembolisationPlacebo groupHepatocellular carcinomaIntention-to-treatOverall survivalDouble-blindPerformance statusAdverse eventsFollow-upEastern Cooperative Oncology Group performance statusChild-Pugh class A diseaseMedian progression-free survivalSolid Tumors version 1.1Blinded independent central reviewA-fetoprotein levelAlbumin-bilirubin gradeResponse Evaluation CriteriaAs-treated populationMedian follow-upPhase 1 trial of hypofractionated stereotactic re-irradiation in combination with nivolumab, ipilimumab, and bevacizumab for recurrent high-grade gliomas
Sahebjam S, Raval R, Forsyth P, Enderling H, Tran N, Arrington J, Macaulay R, Perlow H, Palmer J, Ghose J, Rajappa P, Giglio P, Li Z, Etame A, Mokhtari S, Cruz-Chamorro R, Bhandari M, Thapa R, Robinson T, Chen D, Yu H. Phase 1 trial of hypofractionated stereotactic re-irradiation in combination with nivolumab, ipilimumab, and bevacizumab for recurrent high-grade gliomas. Neuro-Oncology Advances 2025, 7: vdaf033. PMID: 40134851, PMCID: PMC11934552, DOI: 10.1093/noajnl/vdaf033.Peer-Reviewed Original ResearchTreatment-related adverse eventsStereotactic re-irradiationRecurrent high-grade gliomaHigh-grade gliomasRe-irradiationPD-1PD-1 blockadeProgression-free survivalPhase I studySecondary end pointsPhase 1 trialRecurrent HGGCheckpoint immunotherapyOverall survivalRecurrent glioblastomaAnaplastic astrocytomaBevacizumabNivolumabIpilimumabGrade 3Adverse eventsClinical investigationImmune responseEnd pointsPatientsFamitinib plus camrelizumab in patients with advanced colorectal cancer: Data from a multicenter, basket study
Ai L, Li Q, Zhang S, Dong Y, Yang M, Li J, Pan Y, Yuan Y, Yi S, Wang J, Cheng Y, Feng J, Gao S, Wang X, Qu S, Zhang X, Lu J, Xiu P, Wang S, Yang X, Yu Y, Liu T. Famitinib plus camrelizumab in patients with advanced colorectal cancer: Data from a multicenter, basket study. The Innovation 2025, 6: 100745. PMID: 39872476, PMCID: PMC11763884, DOI: 10.1016/j.xinn.2024.100745.Peer-Reviewed Original ResearchProgression-free survivalDuration of responseAdvanced colorectal cancerOverall survivalColorectal cancerMedian duration of responseMedian progression-free survivalMetastatic colorectal cancer patientsTreatment-related adverse eventsMedian follow-up timeMedian overall survivalMetastatic solid tumorsPD-1 antagonistsFollow-up timeCohort of patientsAnti-angiogenic agentsColorectal cancer patientsInhibition of angiogenesisPD-1Immune checkpointsMetastatic diseaseBasket studyMedian durationPrimary endpointSystemic treatment
2024
Nivolumab plus relatlimab and nivolumab plus ipilimumab for patients with advanced renal cell carcinoma: results from the open-label, randomised, phase II FRACTION-RCC trial
Choueiri T, Kuzel T, Tykodi S, Verzoni E, Kluger H, Nair S, Perets R, George S, Gurney H, Pachynski R, Folefac E, Castonguay V, Lee C, Vaishampayan U, Miller W, Bhagavatheeswaran P, Wang Y, Gupta S, DeSilva H, Lee C, Escudier B, Motzer R. Nivolumab plus relatlimab and nivolumab plus ipilimumab for patients with advanced renal cell carcinoma: results from the open-label, randomised, phase II FRACTION-RCC trial. ESMO Open 2024, 9: 104073. PMID: 39642635, PMCID: PMC11667034, DOI: 10.1016/j.esmoop.2024.104073.Peer-Reviewed Original ResearchConceptsAdvanced renal cell carcinomaNivolumab + ipilimumabProgression-free survivalDuration of responseMedian duration of responseProgression-free survival ratesProgrammed death-ligand 1Renal cell carcinomaImmuno-oncologyOpen-labelCell carcinomaPatients treated with nivolumabTyrosine kinase inhibitor therapyTreatment-related adverse eventsLymphocyte activation gene-3Death-ligand 1Kinase inhibitor therapyAssessment of combination therapyEffective combination regimenImmuno-oncology studiesCombination regimenInhibitor therapyLAG-3Combination therapySecondary endpointsPhase I Study of Ruxolitinib in Combination with Abemaciclib for Patients with Primary or Post-Polycythemia Vera/Essential Thrombocythemia Myelofibrosis
Bewersdorf J, Derkach A, Zeidan A, Stein E, Mauro M, Podoltsev N, Rampal R. Phase I Study of Ruxolitinib in Combination with Abemaciclib for Patients with Primary or Post-Polycythemia Vera/Essential Thrombocythemia Myelofibrosis. Blood 2024, 144: 6659-6659. DOI: 10.1182/blood-2024-194918.Peer-Reviewed Original ResearchDose-limiting toxicityCancer Institute Common Terminology Criteria for Adverse EventsTreatment-related adverse eventsAdverse eventsDose levelsCombination therapySpleen volumeInhibitor abemaciclibGrade 3Patients discontinued treatment due to adverse eventsNational Cancer Institute Common Terminology Criteria for Adverse EventsPhase I dose-escalation trialTreatment due to adverse eventsCommon Terminology Criteria for Adverse EventsDisease progressionRecommended phase II doseMulticenter Phase IPlanned dose levelsGrade 3 thrombocytopeniaMedian overall survivalPhase II doseBone marrow blastsBone marrow fibrosisClinically significant bleedingData cut-offSafety, tolerability, pharmacokinetics and pharmacodynamics of a single intravenous dose of SHR-1707 in healthy adult subjects: two randomized, double-blind, single-ascending-dose, phase 1 studies
Yang Y, Qiu H, Fan Y, Zhang Q, Qin H, Wu J, Zhang X, Liu Y, Zhou R, Zhang Q, Ye Z, Ma J, Xu Y, Feng S, Fei Y, Li N, Cui X, Dong F, Wang Q, Shen K, Shakib S, Williams J, Hu W. Safety, tolerability, pharmacokinetics and pharmacodynamics of a single intravenous dose of SHR-1707 in healthy adult subjects: two randomized, double-blind, single-ascending-dose, phase 1 studies. Alzheimer's Research & Therapy 2024, 16: 218. PMID: 39390616, PMCID: PMC11465679, DOI: 10.1186/s13195-024-01584-8.Peer-Reviewed Original ResearchConceptsTreatment-related adverse eventsPhase 1 studySingle-ascending-doseIntravenous doseHealthy adult subjectsDouble-blindElderly subjectsHealthy young adultsAdult subjectsTransient laboratory abnormalitiesPD profilesDose-proportional mannerSingle intravenous dosesYoung adultsDose-dependent increaseIgG1 monoclonal antibodyDose cohortsPlacebo groupLaboratory abnormalitiesPreclinical studiesAdverse eventsClinical developmentDose levelsNo ethnic differencesTransgenic mice12429 Overnight Metyrapone Therapy In Patients With Mild Autonomous Cortisol Secretion (MACS): An Open Label Phase 2 Interventional Study
Ferreira E, Saini J, Larson J, Atkinson E, Powell C, Nevin S, Fell V, Bancos I. 12429 Overnight Metyrapone Therapy In Patients With Mild Autonomous Cortisol Secretion (MACS): An Open Label Phase 2 Interventional Study. Journal Of The Endocrine Society 2024, 8: bvae163.251. PMCID: PMC11453951, DOI: 10.1210/jendso/bvae163.251.Peer-Reviewed Original ResearchMild autonomous cortisol secretionAutonomous cortisol secretionMacronodular adrenal hyperplasiaOvernight metyraponeManagement of patientsAdrenal adenomaMetyrapone therapyAdrenal hyperplasiaCortisol secretionAdverse eventsDehydroepiandrosterone-sulfateIncidence of treatment related adverse eventsSeverity scoreTreatment-related adverse eventsTreatment related adverse eventsIncidence of cardiovascular morbidityPatients discontinued therapyBilateral adrenal adenomasClinical severity scoreSenescence biomarkersSingle-center studyEffective medical therapyAssociated with increased prevalenceRelated adverse eventsPost-dexamethasone cortisol
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