2024
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
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
2020
Study Design and Rationale of EXPLORER-HCM
Ho CY, Olivotto I, Jacoby D, Lester SJ, Roe M, Wang A, Waldman CB, Zhang D, Sehnert AJ, Heitner SB. Study Design and Rationale of EXPLORER-HCM. Circulation Heart Failure 2020, 13: e006853. PMID: 32498620, DOI: 10.1161/circheartfailure.120.006853.Peer-Reviewed Original ResearchConceptsNew York Heart Association classLV outflow tract obstructionObstructive hypertrophic cardiomyopathyOutflow tract obstructionPeak oxygen consumptionEXPLORER-HCMTract obstructionAssociation classEnd pointExercise capacityNew York Heart Association functional class IIDynamic LV outflow tract obstructionKansas City Cardiomyopathy QuestionnaireLV outflow tract gradientSymptomatic obstructive hypertrophic cardiomyopathyUnexplained left ventricular hypertrophyOxygen consumptionExploratory end pointsFunctional class IIOutflow tract gradientPlacebo-controlled trialSecondary end pointsPhase 2 studyPhase 3 trialCurrent medical therapy