2024
Efficacy and Safety of aficamten in Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy: Results From the REDWOOD-HCM Trial, Cohort 4
Masri A, Sherrid M, Abraham T, Choudhury L, Garcia-Pavia P, Kramer C, Barriales-Villa R, Owens A, Rader F, Nagueh S, Olivotto I, Saberi S, Tower-Rader A, Wong T, Coats C, Watkins H, Fifer M, Solomon S, Heitner S, Jacoby D, Kupfer S, Malik F, Meng L, Sohn R, Wohltman A, Maron M, Investigators R. Efficacy and Safety of aficamten in Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy: Results From the REDWOOD-HCM Trial, Cohort 4. Journal Of Cardiac Failure 2024 PMID: 38493832, DOI: 10.1016/j.cardfail.2024.02.020.Peer-Reviewed Original ResearchNonobstructive hypertrophic cardiomyopathyHypertrophic cardiomyopathyKansas City Cardiomyopathy Questionnaire Clinical Summary ScoreOpen-label phase 2 trialNew York Heart Association classHistory of aborted sudden cardiac deathNon-obstructive hypertrophic cardiomyopathyHigh-sensitivity cardiac troponin IAborted sudden cardiac deathBlood levels of biomarkersNT-proBNP levelsPhase 2 trialPlacebo-controlled studyHeart failure symptomsCardiac myosin inhibitorClinical summary scoreSudden cardiac deathClinically relevant improvementWeeks of washoutLevels of biomarkersCardiac troponin IHeart muscle cellsAsymptomatic reductionNT-proBNPAssociated with improvementsLong-term Safety And Tolerability Of Acoramidis (AG10) In Symptomatic Transthyretin Amyloid Cardiomyopathy: 4-year Update From An Ongoing, Phase 2, Open-label Extension Study
Masri A, Aras M, Falk R, Grogan M, Jacoby D, Maurer M, Shah S, Witteles R, Wong P, Ji A, Du J, Siddhanti S, Sinha U, Fox J, Judge D. Long-term Safety And Tolerability Of Acoramidis (AG10) In Symptomatic Transthyretin Amyloid Cardiomyopathy: 4-year Update From An Ongoing, Phase 2, Open-label Extension Study. Journal Of Cardiac Failure 2024, 30: 221. DOI: 10.1016/j.cardfail.2023.10.251.Peer-Reviewed Original ResearchOpen-label extensionATTR-CMAmyloid cardiomyopathyPhase 3 randomized clinical trialMedian NT-proBNP levelOpen-label extension studyNYHA class IIMedian follow-upNT-proBNP levelsPhase 2 studyPhase 2 trialTransthyretin amyloid cardiomyopathyNT-pro-BNPOpen-label studyLong-term treatmentLong-term safetyEx vivo assaysDouble-blindPlacebo-controlledNT-proAdverse eventsFollow-upClinical trialsConcurrent illnessDay 1
2019
Mavacamten Treatment for Obstructive Hypertrophic Cardiomyopathy: A Clinical Trial.
Heitner SB, Jacoby D, Lester SJ, Owens A, Wang A, Zhang D, Lambing J, Lee J, Semigran M, Sehnert AJ. Mavacamten Treatment for Obstructive Hypertrophic Cardiomyopathy: A Clinical Trial. Annals Of Internal Medicine 2019, 170: 741-748. PMID: 31035291, DOI: 10.7326/m18-3016.Peer-Reviewed Original ResearchMeSH KeywordsAdministration, OralAdrenergic beta-AntagonistsAdultAgedBenzylaminesCardiomyopathy, HypertrophicCardiovascular AgentsDose-Response Relationship, DrugDrug Therapy, CombinationExercise ToleranceFemaleHumansMaleMiddle AgedOxygen ConsumptionProspective StudiesStroke VolumeUracilVentricular Function, LeftYoung AdultConceptsObstructive hypertrophic cardiomyopathyLVOT gradientDyspnea scoreAdverse eventsCohort BPeak VO2Hypertrophic cardiomyopathyLeft ventricular outflow tract gradientVentricular outflow tract gradientEnd pointCommon adverse eventsOutflow tract gradientUnrelated adverse eventsOpen-label designPrimary end pointSecondary end pointsPhase 2 trialVentricular ejection fractionML/Peak oxygen consumptionEffect of mavacamtenHigher plasma concentrationsMavacamten treatmentLVOT obstructionTract gradient