2024
COBALT: A Confirmatory Trial of Obeticholic Acid in Primary Biliary Cholangitis With Placebo and External Controls.
Kowdley K, Hirschfield G, Coombs C, Malecha E, Bessonova L, Li J, Rathnayaka N, Mells G, Jones D, Trivedi P, Hansen B, Smith R, Wason J, Hiu S, Kareithi D, Mason A, Bowlus C, Muller K, Carbone M, Berenguer M, Milkiewicz P, Adekunle F, Villamil A. COBALT: A Confirmatory Trial of Obeticholic Acid in Primary Biliary Cholangitis With Placebo and External Controls. The American Journal Of Gastroenterology 2024 PMID: 39140490, DOI: 10.14309/ajg.0000000000003029.Peer-Reviewed Original ResearchPrimary biliary cholangitisObeticholic acidClinical outcomesFunctional unblindingPlacebo armBiliary cholangitisConfirmatory trialsUS healthcare claims databasePrimary biliary cholangitis patientsOCA therapyObeticholic acid treatmentPrimary composite endpointAs-treated analysisHealthcare claims databaseNegative clinical outcomesUncontrolled ascitesPlacebo patientsHepatic decompensationPrimary endpointLiver transplantationComposite endpointCommercial therapySafety signalsPlaceboTreatment crossover
2023
Open‐label, clinical trial extension: Two‐year safety and efficacy results of seladelpar in patients with primary biliary cholangitis
Mayo M, Vierling J, Bowlus C, Levy C, Hirschfield G, Neff G, Galambos M, Gordon S, Borg B, Harrison S, Thuluvath P, Goel A, Shiffman M, Swain M, Jones D, Trivedi P, Kremer A, Aspinall R, Sheridan D, Dörffel Y, Yang K, Choi Y, McWherter C. Open‐label, clinical trial extension: Two‐year safety and efficacy results of seladelpar in patients with primary biliary cholangitis. Alimentary Pharmacology & Therapeutics 2023, 59: 186-200. PMID: 37904314, DOI: 10.1111/apt.17755.Peer-Reviewed Original ResearchConceptsPrimary biliary cholangitisBiliary cholangitisLiver injurySerious treatment-related adverse eventsBiochemical markersLong-term extension studyTreatment-related adverse eventsAnti-pruritic effectsClinical trial extensionsSecondary efficacy analysesTwo-Year SafetyUnexpected histological findingsNon-alcoholic steatohepatitisLong-term safetyAdverse eventsElevated bilirubinComposite endpointDose adjustmentEfficacy analysisHistological findingsTrial extensionEfficacy dataEfficacy resultsSelective peroxisomePatients