2025
Once-Monthly Maridebart Cafraglutide for the Treatment of Obesity — A Phase 2 Trial
Jastreboff A, Ryan D, Bays H, Ebeling P, Mackowski M, Philipose N, Ross L, Liu Y, Burns C, Abbasi S, Pannacciulli N. Once-Monthly Maridebart Cafraglutide for the Treatment of Obesity — A Phase 2 Trial. New England Journal Of Medicine 2025 PMID: 40549887, DOI: 10.1056/nejmoa2504214.Peer-Reviewed Original ResearchBaseline to weekDose escalationTreatment of obesityTreatment policy estimandType 2 diabetesObese cohortBody weightDose-ranging trialPhase 2 trialGastrointestinal adverse eventsGlycated hemoglobin levelsPeptide-antibody conjugatesGlucagon-like peptide-1 receptor agonismIntention-to-treat approachOnce-monthlyDouble-blindPlacebo groupPlacebo-controlledReceptor antagonismReceptor agonismHemoglobin levelsAdverse eventsPlaceboSafety signalsObesityTwo-year follow-up study (PRIMROSE 3) to assess bone mineral density in subjects with uterine fibroids completing the PRIMROSE 1 and PRIMROSE 2 linzagolix trials
Donnez J, Petraglia F, Taylor H, Becker C, Becker S, Herrera F, Paszkowski M, Bestel E, Hori S, Dolmans M. Two-year follow-up study (PRIMROSE 3) to assess bone mineral density in subjects with uterine fibroids completing the PRIMROSE 1 and PRIMROSE 2 linzagolix trials. Human Reproduction Open 2025, 2025: hoaf025. PMID: 40575397, PMCID: PMC12202088, DOI: 10.1093/hropen/hoaf025.Peer-Reviewed Original ResearchDual-energy X-ray absorptiometryTotal hip bone mineral densityHip bone mineral densityBone mineral densityBaseline to monthAdd-back therapyLumbar spine bone mineral densitySpine bone mineral densityBone mineral density valuesAmerican Society of Reproductive MedicinePlacebo groupClinically relevant impactLumbar spineGedeon RichterOverall bone healthFemoral neckBaseline valuesTreatment groupsPrimary endpointSecondary endpointsL1-L4DEXA scanMineral densityFollow-up studyPre-treatment baseline valuesArginine Therapy for Pain in Sickle Cell Disease: A Phase‐2 Randomized, Placebo‐Controlled Trial
Morris C, Hatabah D, Korman R, Gillespie S, Bakshi N, Brown L, Harris F, Leake D, Rees C, Khemani K, Vichinsky E, Locke A, Wynn B, Griffiths M, Wilkinson H, Kumari P, Sudmeier L, Shiva S, Dampier C. Arginine Therapy for Pain in Sickle Cell Disease: A Phase‐2 Randomized, Placebo‐Controlled Trial. American Journal Of Hematology 2025, 100: 1119-1131. PMID: 40270092, PMCID: PMC12148696, DOI: 10.1002/ajh.27692.Peer-Reviewed Original ResearchConceptsPlacebo-controlled trialPatient-reported outcomesArginine therapyArginine bioavailabilityPain scoresVaso-occlusive pain episodesParenteral opioid useTertiary-care children's hospitalPlasma protein carbonyl levelsDose-dependent increasePost hoc sensitivity analysisOxidative stressSickle cell diseaseMeasures of oxidative stressDecreased oxidative stressOpioid-sparingStandard-dosePlacebo groupPlacebo-controlledPatient 3Primary endpointLoading-doseOpioid usePain episodesClinical outcomesQuinagolide vaginal ring for reduction of endometriotic lesions: Results from the QLARITY trial
Pellicer A, Taylor H, Alberich-Bayarri A, Liu Y, Gamborg M, Barletta K, Pinton P, Heiser P, Bagger Y. Quinagolide vaginal ring for reduction of endometriotic lesions: Results from the QLARITY trial. European Journal Of Obstetrics & Gynecology And Reproductive Biology 2025, 310: 113946. PMID: 40188683, DOI: 10.1016/j.ejogrb.2025.113946.Peer-Reviewed Original ResearchConceptsDeep infiltrating endometriosisVaginal ringNo significant differenceLesion sizeBleeding patternsMenstrual cycleSignificant differenceImaging biomarkersSerum prolactin levelsPhase 2 trialMenstrual bleeding patternsEndpoint of reductionPatient-reported outcomesHigh-resolution MRIInfiltrating endometriosisPlacebo groupDouble-blindPlacebo-controlledAdvanced diseaseEndometriotic lesionsPrimary endpointPain reductionSecondary endpointsProlactin levelsAdverse eventsMolecular residual disease analysis of adjuvant osimertinib in resected EGFR-mutated stage IB–IIIA non-small-cell lung cancer
Herbst R, John T, Grohé C, Goldman J, Kato T, Laktionov K, Bonanno L, Tiseo M, Majem M, Dómine M, Ahn M, Kowalski D, Pérol M, Sriuranpong V, Özgüroğlu M, Bhetariya P, Markovets A, Rukazenkov Y, Muldoon C, Robichaux J, Hartmaier R, Tsuboi M, Wu Y. Molecular residual disease analysis of adjuvant osimertinib in resected EGFR-mutated stage IB–IIIA non-small-cell lung cancer. Nature Medicine 2025, 31: 1958-1968. PMID: 40097663, PMCID: PMC12176615, DOI: 10.1038/s41591-025-03577-y.Peer-Reviewed Original ResearchConceptsMolecular residual diseaseDisease-free survivalNon-small-cell lung cancerDisease-free survival eventsEvent-free rateAdjuvant osimertinibOsimertinib treatmentStage IB-IIIA non-small-cell lung cancerLung cancerOverall survival improvementEvent-free statusPosttreatment follow-upExploratory post hoc analysisYear adjuvant treatmentPost hoc analysisDFS eventsOsimertinib groupResidual diseaseTreatment discontinuationPlacebo groupAdjuvant therapyAdjuvant treatmentSurvival improvementOsimertinibFollow-upPsilocybin-assisted therapy for relapse prevention in alcohol use disorder: a phase 2 randomized clinical trial
Rieser N, Bitar R, Halm S, Rossgoderer C, Gubser L, Thévenaz M, Kreis Y, von Rotz R, Nordt C, Visentini M, Moujaes F, Engeli E, Ort A, Seifritz E, Vollenweider F, Herdener M, Preller K. Psilocybin-assisted therapy for relapse prevention in alcohol use disorder: a phase 2 randomized clinical trial. EClinicalMedicine 2025, 82: 103149. PMID: 40144690, PMCID: PMC11937691, DOI: 10.1016/j.eclinm.2025.103149.Peer-Reviewed Original ResearchAlcohol use disorderDose of psilocybinAlcohol use disorder patientsAlcohol useWithdrawal treatmentUse disorderOral doses of psilocybinTherapeutic effects of psilocybinSingle dose of psilocybinEffects of psilocybinInpatient withdrawal treatmentPsilocybin-assisted therapyDaily alcohol useReducing relapse ratesRelapse ratePsilocybin groupPsychotherapy sessionsReduce cravingFollow-upAbstinence durationRelapse preventionPlacebo groupPsilocybinPrevent relapsePsychotherapyA Randomized Phase 3 Study Evaluating the Efficacy and Safety of Alogliptin in Pediatric Participants with Type 2 Diabetes Mellitus
Peng X, Klingensmith G, Hsia D, Xie Y, Czerniak R, Tamborlane W, Shah A. A Randomized Phase 3 Study Evaluating the Efficacy and Safety of Alogliptin in Pediatric Participants with Type 2 Diabetes Mellitus. Diabetes Therapy 2025, 16: 865-883. PMID: 40032809, PMCID: PMC12006607, DOI: 10.1007/s13300-025-01700-3.Peer-Reviewed Original ResearchType 2 diabetes mellitusAlogliptin treatmentEfficacy endpointPediatric patientsAntihyperglycemic therapyOral dipeptidyl peptidase-4 inhibitorHbA1c levelsRandomized phase 3 studyDipeptidyl peptidase-4 inhibitorsSafety of alogliptinPlacebo-controlled trialPhase 3 studyBaseline to weekPeptidase-4 inhibitorsBody mass indexImprove glycemic controlDouble-blindPlacebo groupSecondary endpointsBackground metforminSchedule APharmacological therapyMass indexGlycosylated hemoglobinInsulin therapySafety and efficacy of nipocalimab in adults with generalised myasthenia gravis (Vivacity-MG3): a phase 3, randomised, double-blind, placebo-controlled study
Antozzi C, Vu T, Ramchandren S, Nowak R, Farmakidis C, Bril V, De Bleecker J, Yang H, Minks E, Park J, Grudniak M, Smilowski M, Sevilla T, Hoffmann S, Sivakumar K, Suzuki Y, Youssef E, Sanga P, Karcher K, Zhu Y, Sheehan J, Sun H, Group T. Safety and efficacy of nipocalimab in adults with generalised myasthenia gravis (Vivacity-MG3): a phase 3, randomised, double-blind, placebo-controlled study. The Lancet Neurology 2025, 24: 105-116. PMID: 39862879, DOI: 10.1016/s1474-4422(24)00498-8.Peer-Reviewed Original ResearchConceptsStandard-of-care therapyLeast-squares mean changeOpen-label extension phaseDouble-blind phasePhase 3 studyPopulation of patientsMyasthenia gravisAdverse eventsAntibody-positivePlacebo groupDouble-blindStudy drugMG-ADLAssociated with dose-dependent reductionsDose of study drugLong-term disease controlMean changeIntention-to-treat populationIncidence of adverse eventsMG-ADL scoresPlacebo-controlled studyGeneralised myasthenia gravisPhase 2 studyBaseline to weekAnti-acetylcholine receptorUtilization of Low‐Dose Phentermine for Weight Loss Prior to Metabolic and Bariatric Surgery: A Prospective, Randomized, and Placebo‐Controlled Trial
Garcia L, Rivas H, Morton J. Utilization of Low‐Dose Phentermine for Weight Loss Prior to Metabolic and Bariatric Surgery: A Prospective, Randomized, and Placebo‐Controlled Trial. Obesity Science & Practice 2025, 11: e70043. PMID: 39830713, PMCID: PMC11739713, DOI: 10.1002/osp4.70043.Peer-Reviewed Original ResearchBody mass indexPreoperative weight lossLow-dose formulationsPlacebo groupWeight lossBariatric surgeryLaparoscopic Roux-en-Y gastric bypassPercentage total weight lossRoux-en-Y gastric bypassPlacebo-controlled trialPreoperative medication useTreatment groupsPost-operative complicationsFollow-up visitImprove weight lossMultivariate regression analysisAverage weight lossSleeve gastrectomyGastric bypassMass indexBariatric patientsTotal weight lossPhenterminePatientsOR timeTransarterial 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-up
2024
Efficacy of antiobesity medications for weight reduction in older adults: a systematic review
Chen A, Hajduk A, Grimshaw A, Fried T, Jastreboff A, Lipska K. Efficacy of antiobesity medications for weight reduction in older adults: a systematic review. Obesity 2024 PMID: 39725567, PMCID: PMC12198421, DOI: 10.1002/oby.24160.Peer-Reviewed Original ResearchOlder adultsAntiobesity medicationsStudy of older adultsOlder adults ageAdult ageCardiovascular diseaseRisk of biasAdverse events associated with useWeight reductionStatistically significant weight reductionFood and Drug AdministrationClinical careSystematic reviewInclusion criteriaLimited evidenceBaseline weightPlacebo groupObservational studySeven medicationsAdverse eventsAdultsDrug AdministrationEvaluation studiesMedicationSignificant weight reductionSix-month randomized, placebo controlled trial of synbiotic supplementation in women with polycystic ovary syndrome undergoing lifestyle modifications
Chudzicka-Strugała I, Kubiak A, Banaszewska B, Wysocka E, Zwozdziak B, Siakowska M, Pawelczyk L, Duleba A. Six-month randomized, placebo controlled trial of synbiotic supplementation in women with polycystic ovary syndrome undergoing lifestyle modifications. Archives Of Gynecology And Obstetrics 2024, 311: 499-506. PMID: 39636391, PMCID: PMC11890239, DOI: 10.1007/s00404-024-07833-3.Peer-Reviewed Original ResearchPolycystic ovary syndromeIntensive lifestyle modificationPlacebo groupLifestyle modificationSynbiotic groupMetabolic aspects of polycystic ovary syndromeSynbiotic supplementationImprovement of hyperandrogenismMarkers of endotoxemiaLow-density lipoprotein cholesterolPlacebo-controlled trialReduction of BMILong-term administrationBody fat percentageLPS-binding proteinAdministration of synbioticsObese subjectsReduced LHTotal testosteronePlaceboLipoprotein cholesterolLaboratory aspectsLipid profileTotal cholesterolSix-monthsTirzepatide for Obesity Treatment and Diabetes Prevention
Jastreboff A, le Roux C, Stefanski A, Aronne L, Halpern B, Wharton S, Wilding J, Perreault L, Zhang S, Battula R, Bunck M, Ahmad N, Jouravskaya I. Tirzepatide for Obesity Treatment and Diabetes Prevention. New England Journal Of Medicine 2024, 392: 958-971. PMID: 39536238, DOI: 10.1056/nejmoa2410819.Peer-Reviewed Original ResearchType 2 diabetesProgression to type 2 diabetesDose escalation periodRisk of progression to type 2 diabetesBody weightDoses of tirzepatideBaseline to weekOnce-weekly dosingOff-treatment periodSustained weight reductionYear of treatmentDiagnosis of type 2 diabetesAssigned doseDouble-blindPlacebo groupAdverse eventsTirzepatideTirzepatide groupsPlaceboCausal precursorsSafety signalsObesity treatmentSustained reductionDelay progression to type 2 diabetesObesityAficamten and Cardiopulmonary Exercise Test Performance
Lee M, Masri A, Nassif M, Barriales-Villa R, Abraham T, Claggett B, Coats C, Gimeno J, Kulac I, Landsteiner I, Ma C, Maron M, Olivotto I, Owens A, Solomon S, Veselka J, Jacoby D, Heitner S, Kupfer S, Malik F, Meng L, Wohltman A, Lewis G, Wang A, Sherrid M, Kelly J, Marian A, Owens A, Wever-Pinzon O, Owens D, Wheeler M, Nagueh S, Rader F, McGrew F, Wong T, O'Neill T, Bach R, Martinez M, Lakdawala N, Collado E, Turer A, Desai Y M, Hussain Z, Tower-Rader A, Hannawi B, Geske J, Saberi S, Phelan D, Kramer C, Sarswat N, Ahmad F, Choudhury L, Markowitz J, Sen S, Bering P, Maron M, Jani S, Brinkley D, Naidu S, Maurer M, Moss N, Bilen O, Silva Enciso J, Fraser R, Akinboboye O, Asher C, Emani S, Sharma A, Fermin D, Lyle M, Raymer D, Darlington A, Resnic F, Nielsen C, Metra M, Musumeci B, Emdin M, Targetti M, Canepa M, Michels M, Knackstedt C, Amin A, Barriales Villa R, Garcia Pavia P, Gimeno Blanes J, Hidalgo Urbano R, Rincon Diaz L, Ripoll Vera T, Garcia Alvarez A, Zemanek D, Jensen M, Mogensen J, Thune J, Bundgaard H, Charron P, Trochu J, Habib G, Lhermusier T, Reant P, Hagege A, Logeart D, Mitrovic V, Edelmann F, Seidler T, Meder B, Schulze P, Stoerk S, Bekfani T, Rassaf T, Merkely B, Arad M, Halabi M, Zwas D, Piltz X, Paz O, Habib M, Dudek D, Oreziak A, Wojakowski W, Toste Batista A, Mesquita Bastos J, Elliott P, Mahmod M, Coats C, Cooper R, Bradlow W, Pantazis A, Tome Esteban M, McGinnis S, Campain J, Cocca-Spofford D, Giverts I, Griskowitz C, Newlands C, Moreno F. Aficamten and Cardiopulmonary Exercise Test Performance. JAMA Cardiology 2024, 9: 990-1000. PMID: 39230885, PMCID: PMC11375526, DOI: 10.1001/jamacardio.2024.2781.Peer-Reviewed Original ResearchObstructive hypertrophic cardiomyopathySymptomatic obstructive hypertrophic cardiomyopathyBaseline to weekHypertrophic cardiomyopathyRandomized clinical trialsExercise performanceVentilatory efficiencyClinical trialsPrimary outcomeExercise testPlacebo-corrected improvementCardiac myosin inhibitorPhase 3 trialClinical measuresImpaired exercise capacityCardiopulmonary exercise testingExercise performance measuresHeart rate reserveSubmaximal exercise performancePeak heart rateClinically meaningful thresholdsPlacebo groupDouble-blindPlacebo-controlledAficamtenAlternative Complement Pathway Inhibition with Iptacopan in IgA Nephropathy
Perkovic V, Barratt J, Rovin B, Kashihara N, Maes B, Zhang H, Trimarchi H, Kollins D, Papachristofi O, Jacinto-Sanders S, Merkel T, Guerard N, Renfurm R, Hach T, Rizk D. Alternative Complement Pathway Inhibition with Iptacopan in IgA Nephropathy. New England Journal Of Medicine 2024, 392: 531-543. PMID: 39453772, DOI: 10.1056/nejmoa2410316.Peer-Reviewed Original ResearchProtein-to-creatinine ratioUrinary protein-to-creatinine ratioIgA nephropathyMonth 9Biopsy-confirmed IgA nephropathySupportive therapyTreatment periodDouble-blind treatment periodSecondary end point analysisTrial populationIncidence of adverse eventsIncreased risk of infectionPlacebo-controlled trialSecondary end pointsComplement pathway inhibitionProportion of patientsPathogenesis of IgA nephropathyKidney replacement therapyClinically meaningful reductionsInterim efficacy analysisRisk of infectionDouble-blindPlacebo groupSafety findingsEfficacy analysisAtrasentan in Patients with IgA Nephropathy
Heerspink H, Jardine M, Kohan D, Lafayette R, Levin A, Liew A, Zhang H, Lodha A, Gray T, Wang Y, Renfurm R, Barratt J. Atrasentan in Patients with IgA Nephropathy. New England Journal Of Medicine 2024, 392: 544-554. PMID: 39460694, DOI: 10.1056/nejmoa2409415.Peer-Reviewed Original ResearchIgA nephropathyAdverse eventsInterim analysisBiopsy-proven IgA nephropathyInterim analysis of dataBaseline to weekBody-surface areaPercentage of patientsProtein-to-creatinine ratioUrinary protein-to-creatinine ratioMean percentage changeCases of cardiac failureRisk of kidney failureClinically meaningful reductionsTrial regimenPlacebo groupAntagonist atrasentanDouble-blindBetween-group differencesCardiac failureReduce proteinuriaFluid retentionSevere proteinuriaAtrasentan groupAtrasentanSafety, 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 miceSafety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial
Allen J, Lin J, Basta I, Dysgaard T, Eggers C, Guptill J, Gwathmey K, Hewamadduma C, Hofman E, Hussain Y, Kuwabara S, Le Masson G, Leypoldt F, Chang T, Lipowska M, Lowe M, Lauria G, Querol L, Simu M, Suresh N, Tse A, Ulrichts P, Van Hoorick B, Yamasaki R, Lewis R, van Doorn P, Grinzinger S, Wanschitz J, Seifert-Held T, Claeys K, Baets J, Remiche G, Bissay V, Dubuisson N, Delstanche S, Tarnev I, Genov K, Tsvetanov P, Milanov I, Zhao C, Bu B, Yu X, Li W, Jiang H, Da Y, Lu Z, Liang H, Guo F, Li Z, Zou Z, Hong D, Yang H, Guo J, Shi J, Tu J, He D, Wang Y, Ding J, Zhang Y, Zhao Y, Xu R, Yue Y, Guo A, Wang Y, Talab R, Harbo T, Sindrup S, De Seze J, Sacconi S, Péréon Y, Echaniz-Laguna A, Magy L, Nicolas G, Taithe F, Cassereau J, Debs R, Shakarishvili R, Tsiskaridze A, Mania M, Janelidze M, Janelidze T, Schroeter M, Skripuletz T, Lee D, Klehmet J, Hotter B, Hoffmann O, Baum P, Zschuentzsch J, Pitarokoili K, Stettner M, Bereczki D, Pánczél G, Abraham A, Dori A, Lampl Y, Manganelli F, Morino S, Padovani A, Siciliano G, Schenone A, Magri F, Mazzeo A, Giannini F, Sorbi S, Chiò A, Kuwahara M, Okuno T, Okamoto T, Kokubun N, Nishiyama K, Kaida K, Bokuda K, Katsuno M, Yabe I, Saji E, Yokota T, Hatanaka Y, Nakahara J, Sugimoto T, Tanaka F, Tomita S, Yamano Y, Hayashi T, Yamazaki H, Tokashiki T, Horiuchi K, Karelis G, Eftimov F, Banach M, Chyrchel-Paszkiewicz U, Kochanowicz J, Selmaj K, Zielinski T, Banaszkiewicz K, Mitrea D, Scutaru-Kadar A, Axelerad A, Stuchevskaya F, Boyko A, Khabirov F, Trushnikova T, Goncharova Z, Suponeva N, Dorogov N, Yakupov E, Raicevic R, Miletic Drakulic S, Cabrera Serrano M, Muñoz Blanco J, Guerrero Sola A, Aguera Morales E, Diaz Marin C, Juntas Morales R, Yeh J, Sung J, Huang H, Tsai N, Guo Y, Chao C, Ro L, Sengun I, Terzi M, Alpaydin Baslo S, Koç F, Necdet Karli H, Shulga O, Smolko D, Doroshenko O, Tomakh N, Kyrychenko A, Seliuk O, Kalbus O, Skrypchenko I, Novakovska O, Carod-Artal F, Rinaldi S, Brennan K, Ellis S, Carr A, Matthews E, Traub R, Mozaffar T, Elliott M, Bhavaraju-Sanka R, Nance C, Levine T, Lisak R, Pasnoor M, Pulley M, Roy B, Govindarajan R, Sahagian G, Khella S, Jacob D, Kushlaf H, Sivakumar K, Melamed I, Sharma K, Quick A, Ubogu E, Lacomis D, Isa A, Brannagan T, Chen S, Katz J, Feinberg M, Pavlakis P, Lange D, Gudesblatt M, Tandan R, Gable K, Rivner M, Barnes B, Luke D, Mahuwala Z, Macwan S, Kwon P, Scott J, Altamimi S, Sabharwal P. Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial. The Lancet Neurology 2024, 23: 1013-1024. PMID: 39304241, DOI: 10.1016/s1474-4422(24)00309-0.Peer-Reviewed Original ResearchConceptsTreatment-emergent adverse eventsChronic inflammatory demyelinating polyradiculoneuropathyRisk of relapseInflammatory demyelinating polyradiculoneuropathyStage BStage ADouble-blindModified intention-to-treat populationIntention-to-treat populationOpen-label phaseInflammatory Neuropathy CauseInteractive response technologyPhase 2 trialPlacebo-controlled trialEvidence of clinical improvementClinically meaningful deteriorationPeripheral nervous systemPartial responseSafety populationPlacebo groupPlacebo-controlledResidual neurological impairmentClinical improvementWeeks treatmentTreatment optionsA Randomized Placebo-Controlled Trial of Leronlimab in Mild-To-Moderate COVID-19
Seethamraju H, Yang O, Loftus R, Ogbuagu O, Sammartino D, Mansour A, Sacha J, Ojha S, Hansen S, Arman A, Lalezari J. A Randomized Placebo-Controlled Trial of Leronlimab in Mild-To-Moderate COVID-19. Clinical Therapeutics 2024, 46: 891-899. PMID: 39353749, DOI: 10.1016/j.clinthera.2024.08.019.Peer-Reviewed Original ResearchMild to moderate COVID-19National Early Warning Score 2SARS-CoV-2 infectionPost hoc analysisSARS-CoV-2Randomized placebo-controlled trialHoc analysisHuman IgG4 monoclonal antibodyModerate-to-severe diseaseViral replicationHIV-1 infectionPlacebo-controlled studyProduction of pro-inflammatory cytokinesExcessive production of pro-inflammatory cytokinesMild-to-moderateAdverse event ratesExploratory post hoc analysisTotal symptom scorePathophysiology of SARS-CoV-2 infectionPro-inflammatory cytokinesHeightened immune responsePathophysiology of COVID-19IgG4 monoclonal antibodyPlacebo groupDouble-blindImpact of Aficamten on Disease and Symptom Burden in Obstructive Hypertrophic Cardiomyopathy Results From SEQUOIA-HCM
Maron M, Masri A, Nassif M, Barriales-Villa R, Abraham T, Arad M, Cardim N, Choudhury L, Claggett B, Coats C, Düngen H, Garcia-Pavia P, Hagège A, Januzzi J, Kulac I, Lee M, Lewis G, Ma C, Michels M, Oreziak A, Owens A, Spertus J, Solomon S, Tfelt-Hansen J, van Sinttruije M, Veselka J, Watkins H, Jacoby D, Heitner S, Kupfer S, Malik F, Meng L, Wohltman A, Olivotto I, Investigators S. Impact of Aficamten on Disease and Symptom Burden in Obstructive Hypertrophic Cardiomyopathy Results From SEQUOIA-HCM. Journal Of The American College Of Cardiology 2024, 84: 1821-1831. PMID: 39352339, DOI: 10.1016/j.jacc.2024.09.003.Peer-Reviewed Original ResearchObstructive hypertrophic cardiomyopathyN-terminal pro-B-type natriuretic peptidePro-B-type natriuretic peptideSeptal reduction therapyHypertrophic cardiomyopathyNatriuretic peptideReduction therapySymptomatic obstructive hypertrophic cardiomyopathyExercise capacityCardiac myosin inhibitorProportion of patientsTreatment of patientsHemodynamic responseAssociated with substantial improvementsOutflow gradientPlacebo groupAficamtenEnhanced exercise capacityClinical efficacyEfficacy measuresClinical impactPlaceboResponder analysisPatientsCardiomyopathy
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