2025
Glucagon-like Peptide-1 receptor agonists for the prevention and treatment of Parkinson’s disease
Lee S, Yin L, Xiao N, Rhee T, Lo H, Wong S, Fox S, Teopiz K, Lam B, Zheng Y, Le G, Mansur R, Rosenblat J, McIntyre R. Glucagon-like Peptide-1 receptor agonists for the prevention and treatment of Parkinson’s disease. CNS Spectrums 2025, 30: e44. PMID: 40485141, DOI: 10.1017/s109285292510031x.Peer-Reviewed Original ResearchConceptsGlucagon-like peptide-1 receptor agonistsPeptide-1 receptor agonistsGLP-1RAsCentral nervous systemReceptor agonistsEvidence of clinically meaningful benefitFood and Drug Administration (FDA)-approved treatmentsTreatment-emergent adverse eventsParkinson's diseaseEffects of GLP-1RAsEmergent adverse eventsClinically meaningful benefitFDA-approved treatmentClinical trial evidenceNo current therapyTreatment of Parkinson's diseasePreclinical evidenceAdverse eventsCurrent therapiesMeaningful benefitClinical evidenceFeatures of PDTherapeutic benefitTrial evidenceInsulin resistanceLong-Term Safety and Efficacy of Esmethadone in Patients With Major Depressive Disorder: Findings From a 12-Month Open-Label Study.
Fava M, Pani L, De Martin S, Cutler A, Gorodetzky C, Vocci F, Sapienza F, Kosten T, Kröger C, Champasa P, Guidetti C, Comai S, Mattarei A, Folli F, Bushnell D, Traversa S, Inturrisi C, Manfredi P, Pappagallo M. Long-Term Safety and Efficacy of Esmethadone in Patients With Major Depressive Disorder: Findings From a 12-Month Open-Label Study. The Journal Of Clinical Psychiatry 2025, 86 PMID: 39999772, DOI: 10.4088/jcp.24m15438.Peer-Reviewed Original ResearchConceptsDepressive disorderColumbia-Suicide Severity Rating ScaleOpen-label studyDSM-5</i> criteriaFull analysis setMajor depressive disorderMeasures of depressionSeverity Rating ScaleSexual adverse eventsAntidepressant efficacyTreatment-emergent adverse eventsPostbaseline efficacy assessmentAdverse eventsSuicide attemptsUncompetitive antagonistCognitive functionSafety populationEfficacy assessmentRating ScaleEfficacy measuresMDDDouble-blind studyEmergent adverse eventsLong-term treatmentTreatment-related treatment-emergent adverse eventsP-1107. Oral Sulopenem/probenecid for Uncomplicated Urinary Tract Infections (uUTI): Results from the REASSURE Trial
Puttagunta S, Aronin S, Gupta J, Das A, Gupta K, Dunne M. P-1107. Oral Sulopenem/probenecid for Uncomplicated Urinary Tract Infections (uUTI): Results from the REASSURE Trial. Open Forum Infectious Diseases 2025, 12: ofae631.1295. PMCID: PMC11777793, DOI: 10.1093/ofid/ofae631.1295.Peer-Reviewed Original ResearchUncomplicated urinary tract infectionsTreatment-emergent adverse eventsTreatment of uncomplicated urinary tract infectionsSusceptible to amoxicillin/clavulanateMMITT populationAdverse eventsFrequent treatment-emergent adverse eventsTreatment of multidrug-resistant infectionsBaseline urine cultureOral antibiotic optionsEmergent adverse eventsUrinary tract infectionMultidrug-resistant infectionsAdult womenTreatment of adult womenAmoxicillin/clavulanate groupDouble-dummyActive controlled trialAntibiotic optionsUrine cultureOral antibioticsResistant uropathogensDouble-blindStudy drugPrimary endpoint
2024
Standard-of-Care Medication Withdrawal in Patients With Obstructive Hypertrophic Cardiomyopathy Receiving Aficamten in FOREST-HCM
Masri A, Choudhury L, Barriales-Villa R, Elliott P, Maron M, Nassif M, Oreziak A, Owens A, Saberi S, Tower-Rader A, Rader F, Garcia-Pavia P, Olivotto I, Nagueh S, Wang A, Heitner S, Jacoby D, Kupfer S, Malik F, Melloni C, Meng L, Wei J, Sherrid M, Abraham T, Investigators F. Standard-of-Care Medication Withdrawal in Patients With Obstructive Hypertrophic Cardiomyopathy Receiving Aficamten in FOREST-HCM. Journal Of The American College Of Cardiology 2024, 84: 1839-1849. PMID: 39477631, DOI: 10.1016/j.jacc.2024.09.002.Peer-Reviewed Original ResearchConceptsObstructive hypertrophic cardiomyopathyKansas City Cardiomyopathy Questionnaire-Clinical Summary ScoreStandard-of-careSOC therapyAdverse eventsDose reductionTherapy withdrawalHypertrophic cardiomyopathyTreatment of obstructive hypertrophic cardiomyopathyPro-B-type natriuretic peptide levelsN-terminal pro-B-type natriuretic peptide levelsTreatment-emergent adverse eventsHigh-sensitivity troponin IOutflow tract gradientEmergent adverse eventsNYHA functional classFirst-line therapyNatriuretic peptide levelsOff-target side effectsSummary scoreControlled clinical trialsMeasures of efficacyClinical measures of efficacyStable doseNo withdrawalsEfficacy and Safety of Aficamten in Patients with Obstructive Hypertrophic Cardiomyopathy and Very High Left Ventricular Outflow Tract Gradients
Veselka J, Abraham T, Barriales-Villa R, Claggett B, Coats C, Hegde S, Januzzi J, Maron M, Masri A, Miao Z, Nassif M, Olivotto I, Jacoby D, Heitner S, Kupfer S, Malik F, Meng L, Wohltman A, Gimeno J. Efficacy and Safety of Aficamten in Patients with Obstructive Hypertrophic Cardiomyopathy and Very High Left Ventricular Outflow Tract Gradients. Journal Of Cardiac Failure 2024, 30: s9. DOI: 10.1016/j.cardfail.2024.08.014.Peer-Reviewed Original ResearchObstructive hypertrophic cardiomyopathyOutflow tract gradientSecondary endpointsHypertrophic cardiomyopathyCardiac biomarkersPhase 3 placebo-controlled trialsLeft ventricular outflow tract gradientGroup 2Group 1Symptomatic obstructive hypertrophic cardiomyopathyTreatment-emergent adverse eventsExercise capacityEmergent adverse eventsBaseline to weekEffective treatment optionInhibitor therapyNT-proBNPPrimary endpointEchocardiographic dataBetween-group differencesBaseline characteristicsAficamtenAdverse eventsTreatment optionsSymptom reliefEncorafenib and cetuximab versus irinotecan/cetuximab or FOLFIRI/cetuximab in Chinese patients with BRAF V600E mutant metastatic colorectal cancer: The NAUTICAL CRC study.
Wang X, Deng Y, Zhang Y, Liu T, Yuan X, Yang J, Zhang T, Zang A, Liu Y, Huang L, Ye F, Zong H, Ba Y, Klauck I, Vedovato J, Groc M, Guo A, Shen L. Encorafenib and cetuximab versus irinotecan/cetuximab or FOLFIRI/cetuximab in Chinese patients with BRAF V600E mutant metastatic colorectal cancer: The NAUTICAL CRC study. Journal Of Clinical Oncology 2024, 42: lba3559-lba3559. DOI: 10.1200/jco.2024.42.17_suppl.lba3559.Peer-Reviewed Original ResearchMutant metastatic colorectal cancerTreatment-emergent adverse eventsMetastatic colorectal cancerBRAF V600E mutationChinese patientsControl armMetastatic treatmentV600E mutationGrade 3BRAF V600E-mutated mCRCFrequent treatment-emergent adverse eventsColorectal cancerTreatment-related grade 3CRC studyBaseline ECOG performance statusCombination of encorafenibSafety lead-inEmergent adverse eventsData cut-offMedian patient ageECOG performance statusPrimary cancer siteConfirmed ORRMedian OSMedian PFS
2022
O-306 LINZAGOLIX FOR ENDOMETRIOSIS-ASSOCIATED PAIN: SAFETY RESULTS FROM EDELWEISS 3, A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL
Donnez J, Taylor H, Gemzell-Danielsson K, Catherino W, Bestel E, Gotteland J, Humberstone A, Moore L, Garner E. O-306 LINZAGOLIX FOR ENDOMETRIOSIS-ASSOCIATED PAIN: SAFETY RESULTS FROM EDELWEISS 3, A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL. Human Reproduction 2022, 37: deac105.103. DOI: 10.1093/humrep/deac105.103.Peer-Reviewed Original ResearchTreatment-emergent adverse eventsEndometriosis-associated painSevere endometriosis-associated painBone mineral densityMonths of treatmentSafety resultsPlacebo groupTreatment armsSerious treatment-emergent adverse eventsLumbar spine bone mineral densityZ-scoreLS bone mineral densityMulticenter phase 3 trialSpine bone mineral densityEmergent adverse eventsPhase 3 trialActive treatment armsReproductive-aged womenGnRH receptor antagonistTRIAL REGISTRATION NUMBERMean percent changePARTICIPANTS/MATERIALSROLE OF CHANCEDose-dependent mannerAdverse events
2021
Safety, feasibility, and adherence of a daily, in‐home gamma sensory stimulation therapy with the Cognito Sensory Stimulation System in Alzheimer’s subjects
Williams M, Cimenser A, Hempel E, Cotter C, Shukla M, Jiang K, Zhang A, Mrozak H, Konisky A, Kwan K, Hajos M, Malchano Z. Safety, feasibility, and adherence of a daily, in‐home gamma sensory stimulation therapy with the Cognito Sensory Stimulation System in Alzheimer’s subjects. Alzheimer's & Dementia 2021, 17 DOI: 10.1002/alz.056480.Peer-Reviewed Original ResearchMean adherenceAD subjectsSerious treatment-emergent adverse eventsTreatment-emergent adverse eventsSensory stimulationEmergent adverse eventsPreclinical AD modelsSelf-administered therapyAdverse event monitoringΒ-amyloid plaquesLong-term safetyModerate AD subjectsSensory stimulation therapyAdverse eventsPhysical examSham participantsNovel therapiesTau tanglesModerate ADStimulation therapyCognition improvementAdherence dataAD modelTreatment sessionsTreatment studies539 Phase 1 study of mRNA-2752, a lipid nanoparticle encapsulating mRNAs encoding human OX40L/IL-23/IL-36γ, for intratumoral (ITu) injection +/- durvalumab in advanced solid tumors and lymphoma
Patel M, Jimeno A, Wang D, Stemmer S, Bauer T, Sweis R, Geva R, Kummar S, Reagan P, Perets R, LoRusso P, Gupta S, Zacharek S, Laino A, Milberg O, Frederick J, Chen S, Pascarella S, Randolph W, Aanur P, Johansen L, Do K, Meehan R, Sullivan R. 539 Phase 1 study of mRNA-2752, a lipid nanoparticle encapsulating mRNAs encoding human OX40L/IL-23/IL-36γ, for intratumoral (ITu) injection +/- durvalumab in advanced solid tumors and lymphoma. 2021, a569-a569. DOI: 10.1136/jitc-2021-sitc2021.539.Peer-Reviewed Original ResearchIL-23 serum concentrationsTreatment-emergent adverse eventsPro-inflammatory cytokinesIL-23IL-36γArm BSerum concentrationsAST/ALT increasePD-L1 inhibitor durvalumabPost-treatment tumor biopsiesSquamous cell bladder cancerPK/PD modelingEmergent adverse eventsPain/swellingPD-L1 blockadeCytokine release syndromeAdvanced solid tumorsCytokine IL-22PD-L1 levelsPhase 1 studyPK/PD modelT cell infiltrationEthics BoardGood clinical practiceTreatment effects
2019
Phase I study of pembrolizumab in people with HIV and cancer.
Uldrick T, Goncalves P, Abdul Hay M, Claeys A, Emu B, Ernstoff M, Fong L, Kaiser J, Kohrt H, Lacroix A, Lee S, Lundgren L, Lurain K, Parsons C, Peeramsetti S, Ramaswami R, Sharon E, Wang C, Yarchoan R, Cheever M. Phase I study of pembrolizumab in people with HIV and cancer. Journal Of Clinical Oncology 2019, 37: 2500-2500. DOI: 10.1200/jco.2019.37.15_suppl.2500.Peer-Reviewed Original ResearchAdverse eventsNon-Hodgkin lymphomaAntiretroviral therapyViral loadClinical benefitAnti-PD-1/PD-L1 therapyCD4 cells/μL.Elevated AST/ALTMedian age 57 yearsMulticenter phase 1 trialTreatment-emergent adverse eventsEmergent adverse eventsHBV/HCVSquamous cell skinPD-L1 therapyAge 57 yearsHIV viral loadAST/ALTPhase 1 trialCells/Cells/μL.Multicentric Castleman's diseaseAppropriate eligibility criteriaImmuno-oncology studiesNumber of cancers
2018
Randomized trial of lacosamide versus fosphenytoin for nonconvulsive seizures
Husain AM, Lee JW, Kolls BJ, Hirsch LJ, Halford JJ, Gupta PK, Minazad Y, Jones JM, LaRoche SM, Herman ST, Swisher CB, Sinha SR, Palade A, Dombrowski KE, Gallentine WB, Hahn CD, Gerard EE, Bhapkar M, Lokhnygina Y, Westover MB, Consortium F. Randomized trial of lacosamide versus fosphenytoin for nonconvulsive seizures. Annals Of Neurology 2018, 83: 1174-1185. PMID: 29733464, PMCID: PMC6785201, DOI: 10.1002/ana.25249.Peer-Reviewed Original ResearchConceptsTreatment-emergent adverse eventsNonconvulsive seizuresContinuous electroencephalographyConfidence intervalsEmergent adverse eventsAnn NeurolPrimary endpointAdverse eventsAntiseizure drugsIll patientsElectrographic seizuresMean ageTreatment trialsOptimal treatmentRisk ratioRelative riskComparative effectivenessSeizuresFosphenytoinNoninferiorityPatientsTreatmentTrialsEquivalents/Subjects
2013
Safety profile of recombinant poxviral TRICOM vaccines.
Kim J, Marte J, Singh N, Heery C, Madan R, Pazdur M, McMahon S, Rauckhorst M, Schlom J, Kantoff P, Gulley J. Safety profile of recombinant poxviral TRICOM vaccines. Journal Of Clinical Oncology 2013, 31: e16036-e16036. DOI: 10.1200/jco.2013.31.15_suppl.e16036.Peer-Reviewed Original ResearchThrombotic thrombocytopenic purpuraAdverse eventsPoxviral vaccinesSafety profileMetastatic castration-resistant prostate cancerRandomized phase II trialCastration-resistant prostate cancerEmergent adverse eventsMedian overall survivalPhase II trialPhase III trialsTherapeutic cancer vaccinesResistant prostate cancerFavorable safety profileGranulocyte-macrophage colony-stimulating factorProstate-specific antigenRoute of administrationColony-stimulating factorII trialIII trialsOverall survivalVaccine injectionImmune adjuvantsThrombocytopenic purpuraCancer vaccines
2012
A first-in-human, phase I safety and pharmacokinetic study of genz-644282, a non-camptothecin topoisomerase I inhibitor, in patients with advanced solid tumors.
Han H, Tan A, Weiss G, Sullivan D, Strosberg J, Collins S, Moss R, Wu J, Ewesuedo R, LoRusso P. A first-in-human, phase I safety and pharmacokinetic study of genz-644282, a non-camptothecin topoisomerase I inhibitor, in patients with advanced solid tumors. Journal Of Clinical Oncology 2012, 30: 2534-2534. DOI: 10.1200/jco.2012.30.15_suppl.2534.Peer-Reviewed Original ResearchTopoisomerase I inhibitorDay scheduleCell lungTreatment-emergent adverse eventsNon-small cell lungEmergent adverse eventsPhase I safetyAdvanced solid tumorsAccelerated titration designPK-PD relationshipSmall cell lungDistinct clinical profileDose-exposure relationshipPharmacokinetic-pharmacodynamic modelI inhibitorNon-camptothecin topoisomerase I inhibitorsStable diseaseI safetyAdverse eventsPhase I developmentWeekly dosesClinical profileEfficacy dataTitration designSafety data
2007
Phase II study of the efficacy and safety of intravenous (IV) AVE0005 (VEGF Trap) given every 2 weeks in patients (Pts) with platinum- and erlotinib- resistant adenocarcinoma of the lung (NSCLA)
Massarelli E, Miller V, Leighl N, Rosen P, Albain K, Hart L, Melnyk O, Sternas L, Ackerman J, Herbst R. Phase II study of the efficacy and safety of intravenous (IV) AVE0005 (VEGF Trap) given every 2 weeks in patients (Pts) with platinum- and erlotinib- resistant adenocarcinoma of the lung (NSCLA). Journal Of Clinical Oncology 2007, 25: 7627-7627. DOI: 10.1200/jco.2007.25.18_suppl.7627.Peer-Reviewed Original ResearchTreatment-emergent adverse eventsPartial responsePerformance statusGrade 3Prior treatmentSquamous cell lung cancerEmergent adverse eventsSignificant bleeding diathesisECOG performance statusObjective response ratePhase II studyProgression-free survivalSingle-agent activityDuration of responseMulti-center trialMost common reasonsInterim futility analysisVEGF receptor inhibitorQuality of lifeBone painEvaluable ptsFebrile neutropeniaMeasurable diseaseBrain metastasesAdverse events
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