2015
Long-Term Hematologic Response to Eliglustat in Patients with Gaucher Disease Type 1: Results from a Phase 2 and Two Phase 3 Trials
Weinreb N, Cox T, Lukina E, Mistry P, Angell J, Gaemers S, Peterschmitt M. Long-Term Hematologic Response to Eliglustat in Patients with Gaucher Disease Type 1: Results from a Phase 2 and Two Phase 3 Trials. Blood 2015, 126: 884. DOI: 10.1182/blood.v126.23.884.884.Peer-Reviewed Original ResearchGaucher disease type 1Enzyme replacement therapyPhase 3 trialMean platelet countHemoglobin levelsPlatelet countSpleen volumeDisease type 1Substrate reduction therapySpeakers bureauLiver volumeOpen-label phase 2 trialYears of ERTMononuclear phagocytesPlacebo-controlled phase 3 trialBaseline meanOral substrate reduction therapyIntravenous enzyme replacement therapyType 1Acid β-glucosidaseTravel reimbursementCYP2D6 metabolizer phenotypeMain efficacy parametersX109/LTreatment-naïve patients
2014
Engage - a Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Eliglustat in Adults with Gaucher Disease Type 1: Results after 18 Months
Amato D, Dasouki M, Packman S, Pastores G, Assouline S, Balwani M, Mistry P, Ortega A, Shankar S, Solano M, Ross L, Angell J, Peterschmitt J. Engage - a Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Eliglustat in Adults with Gaucher Disease Type 1: Results after 18 Months. Blood 2014, 124: 2732. DOI: 10.1182/blood.v124.21.2732.2732.Peer-Reviewed Original ResearchGaucher disease type 1Advisory board feesBone mineral densityTotal bone mineral densityDisease type 1Patients meetingEfficacy endpointTravel reimbursementFellowship trainingSecondary endpointsType 1Extension phaseMean improvementOral substrate reduction therapyOpen-label extension phaseZ-scoreExtension periodPrimary efficacy endpointSecondary efficacy endpointsPhase 3 trialBone marrow infiltrationBone marrow burden scoreNew safety concernsMulti-center studySubstrate reduction therapy
2013
Engage: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multi-center Study To Investigate The Efficacy and Safety Of Eliglustat In Adults With Gaucher Disease Type 1: 9 Month Results
Shankar S, Lukina E, Amato D, Dasouki M, Packman S, Pastores G, Assouline S, Balwani M, Mistry P, Ross L, Marulkar S, Peterschmitt M. Engage: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multi-center Study To Investigate The Efficacy and Safety Of Eliglustat In Adults With Gaucher Disease Type 1: 9 Month Results. Blood 2013, 122: 2275. DOI: 10.1182/blood.v122.21.2275.2275.Peer-Reviewed Original ResearchGaucher disease type 1Disease type 1Spleen volumeSubstrate reduction therapyType 1Efficacy endpointUntreated adultsLiver volumeReduction therapyBone marrowDisease severity scoring systemOral substrate reduction therapyBone mineral density changesAdvisory CommitteePrimary efficacy endpointSecondary efficacy endpointsSymptomatic bone diseaseMainstay of therapyOpen-label trialPhase 3 trialBone marrow burden scoreMineral density changesBurden of diseaseCommon lysosomal storage disorderSeverity scoring system