2023
Trial in Progress: An Open-Label Expansion Trial Evaluating the Safety, PK/PD, and Clinical Activity of Emavusertib (CA-4948) + Ibrutinib in R/R Primary CNS Lymphoma
Grommes C, Nowakowski G, Rosenthal A, Lunning M, Ramchandren R, Regales L, Fowle M, Lane M, Wang C, Omuro A, Leslie L, Soussain C, Dabrowska-Iwanicka A, Ferreri A, Tun H. Trial in Progress: An Open-Label Expansion Trial Evaluating the Safety, PK/PD, and Clinical Activity of Emavusertib (CA-4948) + Ibrutinib in R/R Primary CNS Lymphoma. Blood 2023, 142: 3143. DOI: 10.1182/blood-2023-190391.Peer-Reviewed Original ResearchPrimary central nervous system lymphomaInterleukin-1 receptor-associated kinase 4FMS-like tyrosine kinase 3Toll-like receptorsExpansion cohortPCNSL patientsDisease progressionRefractory primary central nervous system lymphomaDiagnosis of PCNSLCentral nervous system lymphomaPathogenesis of PCNSLSafety/tolerabilityOpen-label trialBlood-brain barrier penetrationPrimary CNS lymphomaSufficient blood-brain barrier penetrationNervous system lymphomaInitial clinical dataKey inclusion criteriaPotent oral inhibitorCentral nervous systemBrain barrier penetrationPK/PDTyrosine kinase 3Further preclinical studiesRandomized controlled trial of urinE chemiStry guided aCute heArt faiLure treATmEnt (ESCALATE): Rationale and design
Cox Z, Siddiqi H, Stevenson L, Bales B, Han J, Hart K, Imhoff B, Ivey-Miranda J, Jenkins C, Lindenfeld J, Shotwell M, Miller K, Ooi H, Rao V, Schlendorf K, Self W, Siew E, Storrow A, Walsh R, Wrenn J, Testani J, Collins S. Randomized controlled trial of urinE chemiStry guided aCute heArt faiLure treATmEnt (ESCALATE): Rationale and design. American Heart Journal 2023, 265: 121-131. PMID: 37544492, PMCID: PMC10592235, DOI: 10.1016/j.ahj.2023.07.014.Peer-Reviewed Original ResearchAcute decompensated heart failureUrine chemistryDiuretic strategyIntravenous diuresisUsual careAcute heartHeart failureClinical trialsGlobal clinical statusKey exclusion criteriaUsual care strategyAcute heart failureDecompensated heart failureRandomized clinical trialsKey inclusion criteriaMultiple clinical trialsPaucity of evidenceDiuretic dosingDiuretic therapyPrimary outcomePatient's symptomsClinical statusTreatment trialsValvular stenosisClinical stateInitial efficacy and safety results from ENGOT-ov60/GOG-3052/RAMP 201: A phase 2 study of avutometinib (VS-6766) ± defactinib in recurrent low-grade serous ovarian cancer (LGSOC).
Banerjee S, Ring K, Van Nieuwenhuysen E, Fabbro M, Aghajanian C, Oaknin A, Colombo N, Santin A, Clamp A, Moore K, Rose P, O'Malley D, Chon H, Salinas E, Prendergast E, Lustgarten S, Rodrigues M, Gennigens C, Monk B, Grisham R. Initial efficacy and safety results from ENGOT-ov60/GOG-3052/RAMP 201: A phase 2 study of avutometinib (VS-6766) ± defactinib in recurrent low-grade serous ovarian cancer (LGSOC). Journal Of Clinical Oncology 2023, 41: 5515-5515. DOI: 10.1200/jco.2023.41.16_suppl.5515.Peer-Reviewed Original ResearchRecurrent low-grade serous ovarian cancerLow-grade serous ovarian cancerOptimal regimenPartial responseKRAS statusOvarian cancerHigher disease control rateMost AEsMEK inhibitorsBlinded independent central reviewRAF/MEK inhibitionDisease control ratePrior systemic regimensSimilar AE profilesNew safety signalsPhase 2 studyIndependent central reviewKey inclusion criteriaSerous ovarian cancerSynergistic antitumor activityMajority of treatmentsDermatitis acneiformEvaluable patientsCombination armSystemic regimens
2022
A phase 1/2 study of the highly selective EGFR inhibitor, BLU-701, in patients with EGFR-mutant non–small cell lung cancer (NSCLC).
Spira A, Spigel D, Camidge D, De Langen A, Kim T, Goto K, Elamin Y, Shum E, Reckamp K, Rotow J, Goldberg S, Gadgeel S, Leal T, Albayya F, Fitzpatrick S, Louie-Gao M, Parepally J, Zalutskaya A, Yu H. A phase 1/2 study of the highly selective EGFR inhibitor, BLU-701, in patients with EGFR-mutant non–small cell lung cancer (NSCLC). Journal Of Clinical Oncology 2022, 40: tps9142-tps9142. DOI: 10.1200/jco.2022.40.16_suppl.tps9142.Peer-Reviewed Original ResearchNon-small cell lung cancerEGFRm non-small cell lung cancerTyrosine kinase inhibitorsOverall response ratePrimary endpointEastern Cooperative Oncology Group performance status 0EGFR-mutant non-small cell lung cancerPhase 1 dose escalationPhase 2 primary endpointOral tyrosine kinase inhibitorGeneration tyrosine kinase inhibitorsResistance mutationsEGFR T790M mutationDisease control rateKey exclusion criteriaPerformance status 0Phase 1/2 studyPhase 2 doseProgression-free survivalPlatinum-based chemotherapyCell lung cancerDuration of responseCentral nervous system activityKey inclusion criteriaPK/pharmacodynamicsCambridge hybrid closed-loop algorithm in children and adolescents with type 1 diabetes: a multicentre 6-month randomised controlled trial
Ware J, Boughton CK, Allen JM, Wilinska ME, Tauschmann M, Denvir L, Thankamony A, Campbell FM, Wadwa RP, Buckingham BA, Davis N, DiMeglio LA, Mauras N, Besser REJ, Ghatak A, Weinzimer SA, Hood KK, Fox DS, Kanapka L, Kollman C, Sibayan J, Beck RW, Hovorka R, Consortium D, Hovorka R, Acerini C, Thankamony A, Allen J, Boughton C, Dovc K, Dunger D, Ware J, Musolino G, Tauschmann M, Wilinska M, Hayes J, Hartnell S, Slegtenhorst S, Ruan Y, Haydock M, Mangat J, Denvir L, Kanthagnany S, Law J, Randell T, Sachdev P, Saxton M, Coupe A, Stafford S, Ball A, Keeton R, Cresswell R, Crate L, Cripps H, Fazackerley H, Looby L, Navarra H, Saddington C, Smith V, Verhoeven V, Bratt S, Khan N, Moyes L, Sandhu K, West C, Wadwa R, Alonso G, Forlenza G, Slover R, Towers L, Berget C, Coakley A, Escobar E, Jost E, Lange S, Messer L, Thivener K, Campbell F, Yong J, Metcalfe E, Allen M, Ambler S, Waheed S, Exall J, Tulip J, Buckingham B, Ekhlaspour L, Maahs D, Norlander L, Jacobson T, Twon M, Weir C, Leverenz B, Keller J, Davis N, Kumaran A, Trevelyan N, Dewar H, Price G, Crouch G, Ensom R, Haskell L, Lueddeke L, Mauras N, Benson M, Bird K, Englert K, Permuy J, Ponthieux K, Marrero-Hernandez J, DiMeglio L, Ismail H, Jolivette H, Sanchez J, Woerner S, Kirchner M, Mullen M, Tebbe M, Besser R, Basu S, London R, Makaya T, Ryan F, Megson C, Bowen-Morris J, Haest J, Law R, Stamford I, Ghatak A, Deakin M, Phelan K, Thornborough K, Shakeshaft J, Weinzimer S, Cengiz E, Sherr J, Van Name M, Weyman K, Carria L, Steffen A, Zgorski M, Sibayan J, Beck R, Borgman S, Davis J, Rusnak J, Hellman A, Cheng P, Kanapka L, Kollman C, McCarthy C, Chalasani S, Hood K, Hanes S, Viana J, Lanning M, Fox D, Arreaza-Rubin G, Eggerman T, Green N, Janicek R, Gabrielson D, Belle S, Castle J, Green J, Legault L, Willi S, Wysham C. Cambridge hybrid closed-loop algorithm in children and adolescents with type 1 diabetes: a multicentre 6-month randomised controlled trial. The Lancet Digital Health 2022, 4: e245-e255. PMID: 35272971, DOI: 10.1016/s2589-7500(22)00020-6.Peer-Reviewed Original ResearchConceptsClosed-loop groupInsulin pump therapyType 1 diabetesControl groupBaseline HbAUsual carePump therapyClosed-loop insulin deliverySuboptimal glucose controlPediatric diabetes centersKey inclusion criteriaClosed-loop insulin delivery systemPrimary endpointBlock randomisationDiabetes CenterGlucose controlInsulin delivery systemsInclusion criteriaPrimary analysisTherapyInsulin deliveryMonthsG pumpHbADiabetesInhibition of 11β‐Hydroxysteroid dehydrogenase‐1 with AZD4017 in patients with nonalcoholic steatohepatitis or nonalcoholic fatty liver disease: A randomized, double‐blind, placebo‐controlled, phase II study
Yadav Y, Dunagan K, Khot R, Venkatesh S, Port J, Galderisi A, Cobelli C, Wegner C, Basu A, Carter R, Basu R. Inhibition of 11β‐Hydroxysteroid dehydrogenase‐1 with AZD4017 in patients with nonalcoholic steatohepatitis or nonalcoholic fatty liver disease: A randomized, double‐blind, placebo‐controlled, phase II study. Diabetes Obesity And Metabolism 2022, 24: 881-890. PMID: 35014156, PMCID: PMC9135169, DOI: 10.1111/dom.14646.Peer-Reviewed Original ResearchConceptsNonalcoholic fatty liver diseaseLiver fat fractionFatty liver diseaseMagnetic resonance imagingMean changePlacebo groupPrimary outcomeLiver diseasePresence of NAFLDSignificant between-group differencesPhase II studyPhase 2 studyKey inclusion criteriaType 2 diabetesBetween-group differencesShort-term treatmentBiopsy positiveII studyNonalcoholic steatohepatitisWeek 12Liver steatosisHepatic fatInsulin sensitivityAZD4017Liver enzymes
2020
Carfilzomib or bortezomib in combination with lenalidomide and dexamethasone for patients with newly diagnosed multiple myeloma without intention for immediate autologous stem-cell transplantation (ENDURANCE): a multicentre, open-label, phase 3, randomised, controlled trial
Kumar SK, Jacobus SJ, Cohen AD, Weiss M, Callander N, Singh AK, Parker TL, Menter A, Yang X, Parsons B, Kumar P, Kapoor P, Rosenberg A, Zonder JA, Faber E, Lonial S, Anderson KC, Richardson PG, Orlowski RZ, Wagner LI, Rajkumar SV. Carfilzomib or bortezomib in combination with lenalidomide and dexamethasone for patients with newly diagnosed multiple myeloma without intention for immediate autologous stem-cell transplantation (ENDURANCE): a multicentre, open-label, phase 3, randomised, controlled trial. The Lancet Oncology 2020, 21: 1317-1330. PMID: 32866432, PMCID: PMC7591827, DOI: 10.1016/s1470-2045(20)30452-6.Peer-Reviewed Original ResearchConceptsAutologous stem cell transplantationProgression-free survivalStem cell transplantationVRd regimenInduction therapyMultiple myelomaVRd groupDay 1Oral lenalidomideOral dexamethasoneOverall survivalEastern Cooperative Oncology Group performance statusMedian progression-free survivalNon-haematological adverse eventsSecond planned interim analysisNext-generation proteasome inhibitorsHigh-risk multiple myelomaCommon grade 3Treatment-related deathsMedian overall survivalPhase 2 trialCommunity oncology practicesStandard of careKey inclusion criteriaPhase 3Multicenter Study of Metastatic Lung Tumors Targeted by Interventional Cryoablation Evaluation (SOLSTICE)
Callstrom MR, Woodrum DA, Nichols FC, Palussiere J, Buy X, Suh RD, Abtin FG, Pua BB, Madoff DC, Bagla SL, Papadouris DC, Fernando HC, Dupuy DE, Healey TT, Moore WH, Bilfinger TV, Solomon SB, Yarmohammadi H, Krebs HJ, Fulp CJ, Hakime A, Tselikas L, de Baere T. Multicenter Study of Metastatic Lung Tumors Targeted by Interventional Cryoablation Evaluation (SOLSTICE). Journal Of Thoracic Oncology 2020, 15: 1200-1209. PMID: 32151777, PMCID: PMC9201766, DOI: 10.1016/j.jtho.2020.02.022.Peer-Reviewed Original ResearchConceptsOverall survival ratePulmonary metastasesPercutaneous cryoablationSurvival rateLocal recurrence-free survivalGrade 4 eventsPleural catheter placementMetastatic lung tumorsPhase 2 studyKaplan-Meier methodRecurrence-free survivalRate of pneumothoraxKaplan-Meier estimatesKey inclusion criteriaQuality of lifeComplication eventsExtrapulmonary cancersMetastatic diseasePerformance statusInitial treatmentRecurrent tumorsCatheter placementLung metastasesMulticenter studyCryoablation treatment
2019
Lenvatinib (len) plus pembrolizumab (pembro) in patients (pts) with advanced melanoma previously exposed to anti–PD-1/PD-L1 agents: Phase 2 LEAP-004 study.
Arance Fernandez A, Ascierto P, Carlino M, Daud A, Eggermont A, Hauschild A, Kluger H, Taylor M, Smith A, Chen K, Krepler C, Diede S, O'Day S. Lenvatinib (len) plus pembrolizumab (pembro) in patients (pts) with advanced melanoma previously exposed to anti–PD-1/PD-L1 agents: Phase 2 LEAP-004 study. Journal Of Clinical Oncology 2019, 37: tps9594-tps9594. DOI: 10.1200/jco.2019.37.15_suppl.tps9594.Peer-Reviewed Original ResearchPD-1/PD-L1 inhibitor therapyPD-L1 inhibitor therapyPD-1 inhibitorsRECIST v1.1Advanced melanomaLast doseInhibitor therapyAnti-PD-1/PD-L1 agentsKey secondary end pointPrimary efficacy end pointEnd pointActive autoimmune diseaseAdequate organ functionBaseline tumor samplesECOG PS 0/1Exploratory biomarker analysisNCI CTCAE v4.0PD-L1 agentsUnresectable stage IIIAntitumor activityEfficacy end pointSecondary end pointsWeeks of therapyPhase 2 trialKey inclusion criteriaEfficacy and safety of burosumab in children aged 1–4 years with X-linked hypophosphataemia: a multicentre, open-label, phase 2 trial
Whyte MP, Carpenter TO, Gottesman GS, Mao M, Skrinar A, San Martin J, Imel EA. Efficacy and safety of burosumab in children aged 1–4 years with X-linked hypophosphataemia: a multicentre, open-label, phase 2 trial. The Lancet Diabetes & Endocrinology 2019, 7: 189-199. PMID: 30638856, DOI: 10.1016/s2213-8587(18)30338-3.Peer-Reviewed Original ResearchConceptsSerum phosphorus concentrationPhase 2 trialWeeks of treatmentAdverse eventsWeek 40Week 64Serum phosphorusPhosphatonin fibroblast growth factor 23Treatment-related adverse eventsFibroblast growth factor 23History of toothRickets Severity ScoreSafety of burosumabSerious adverse eventsInjection site reactionsKey secondary outcomesFavorable safety profileGrowth factor 23Severe food allergyHeight z-scoreKey inclusion criteriaRenal phosphate wastingHuman monoclonal antibodyRadiographic Global ImpressionYoung children
2016
HERMIONE: a randomized Phase 2 trial of MM-302 plus trastuzumab versus chemotherapy of physician’s choice plus trastuzumab in patients with previously treated, anthracycline-naïve, HER2-positive, locally advanced/metastatic breast cancer
Miller K, Cortes J, Hurvitz SA, Krop IE, Tripathy D, Verma S, Riahi K, Reynolds JG, Wickham TJ, Molnar I, Yardley DA. HERMIONE: a randomized Phase 2 trial of MM-302 plus trastuzumab versus chemotherapy of physician’s choice plus trastuzumab in patients with previously treated, anthracycline-naïve, HER2-positive, locally advanced/metastatic breast cancer. BMC Cancer 2016, 16: 352. PMID: 27259714, PMCID: PMC4893300, DOI: 10.1186/s12885-016-2385-z.Peer-Reviewed Original ResearchConceptsHER2-positive breast cancerProgression-free survivalPhase 2 trialMetastatic breast cancerBreast cancerDisease progressionOverall survivalMM-302Physician's choiceInvestigator-assessed progression-free survivalMetastatic HER2-positive breast cancerRandomized phase 2 trialEpidermal growth factor receptor 2Anthracycline-naïve patientsBackgroundHuman epidermal growth factor receptor 2Objective response rateGrowth factor receptor 2Positive breast cancerAdo-trastuzumab emtansineKey inclusion criteriaFactor receptor 2Quality of lifeOS ratesRECIST v1.1Refractory HER2
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