2019
Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials
Weinreb N, Cox T, Mistry P, Charrow J, Lukina E, Foster M, Peterschmitt M. Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials. Blood 2019, 134: 4859. DOI: 10.1182/blood-2019-127337.Peer-Reviewed Original ResearchGaucher disease type 1Enzyme replacement therapySanofi GenzymeTreatment-naïve patientsMedian percent reductionMIP-1βDisease type 1Travel reimbursementOral eliglustatAdverse eventsMost patientsStable patientsClinical trialsBiomarker levelsSpeakers bureauEliglustat treatmentType 1Advisory CommitteeLong-term therapeutic goalPoor CYP2D6 metabolizersStorage of glycosphingolipidsFirst-line treatmentSubset of patientsLong-term time pointsAttrition of patients
2018
Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials
Mistry P, Charrow J, Cox T, Lukina E, Marinakis T, Foster M, Gaemers S, Peterschmitt J. Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials. Blood 2018, 132: 2396. DOI: 10.1182/blood-2018-99-117289.Peer-Reviewed Original ResearchGaucher disease type 1Enzyme replacement therapyBone crisesSanofi GenzymeTreatment-naïve patientsMIP-1β levelsSpine Z-scoreSpine T-scoreENCORE trialDisease type 1Z-scoreBone diseaseT-scoreOral eliglustatBone painAdverse eventsClinical trialsNormal rangeYears of ERTAcid β-glucosidase activityOral substrate reduction therapyType 1Phase 2Principal investigatorTravel reimbursement
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