2015
Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use
Telen M, Wun T, McCavit T, De Castro L, Krishnamurti L, Lanzkron S, Hsu L, Smith W, Rhee S, Magnani J, Thackray H. Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use. Blood 2015, 125: 2656-2664. PMID: 25733584, PMCID: PMC4408290, DOI: 10.1182/blood-2014-06-583351.Peer-Reviewed Original ResearchConceptsVaso-occlusive crisisSickle cell diseaseComposite primary end pointPrimary end pointPhase 2 studyEnd pointRandomized phase 2 studySCD vaso-occlusive crisisOpioid analgesic useSecondary end pointsActive treatment groupPhase 3 studyVaso-occlusive eventsAnalgesic usePlacebo groupProspective multicenterStudy drugAdverse eventsOpioid useSymptom reliefMedian timeSCD patientsCell diseaseTreatment groupsAnimal models
2013
GMI 1070: Reduction In Time To Resolution Of Vaso-Occlusive Crisis and Decreased Opioid Use In a Prospective, Randomized, Multi-Center Double Blind, Adaptive Phase 2 Study In Sickle Cell Disease
Telen M, Wun T, McCavit T, De Castro L, Krishnamurti L, Lanzkron S, Hsu L, Smith W, Rhee S, Magnani J, Thackray H. GMI 1070: Reduction In Time To Resolution Of Vaso-Occlusive Crisis and Decreased Opioid Use In a Prospective, Randomized, Multi-Center Double Blind, Adaptive Phase 2 Study In Sickle Cell Disease. Blood 2013, 122: 776. DOI: 10.1182/blood.v122.21.776.776.Peer-Reviewed Original ResearchVaso-occlusive crisisSickle cell diseaseOpioid useMedian timeTreatment groupsPlacebo groupCell diseaseSustained reductionVisual analog scale pain scoreTotal adverse event rateCumulative opioid useOral pain medicationPatients ages 12Opioid analgesic usePrimary efficacy endpointScale pain scoresPhase 2 studyPhase 3 studyPrimary efficacy analysisAdverse event ratesDaily opioid useStatistical significanceOral analgesiaSerious AEsVAS reduction
2011
A Randomized, Double-Blind, Adaptive Phase 2 Multi-Center Study of Prasugrel Compared to Placebo in Adults with Sickle Cell Disease
Wun T, Soulieres D, Krishnamurti L, Kutlar A, Ataga K, Zhou C, Heath L, Nwachuku C, Jakubowski J, Winters K, Riesmeyer J. A Randomized, Double-Blind, Adaptive Phase 2 Multi-Center Study of Prasugrel Compared to Placebo in Adults with Sickle Cell Disease. Blood 2011, 118: 847. DOI: 10.1182/blood.v118.21.847.847.Peer-Reviewed Original ResearchSickle cell diseasePrasugrel armHemorrhagic eventsCell diseaseMedical attentionPain rateAdult patientsPlacebo armPlatelet inhibitionAntiplatelet agentsPharmacodynamic effectsEli LillyP2Y12 ADP receptor antagonistsHemorrhagic adverse eventsInsufficient platelet inhibitionP2Y12 reactivity unitsPrasugrel-treated patientsSerious hemorrhagic eventsADP receptor antagonistsPlatelet reactivity indexAcute coronary syndromeBaseline pain intensityPhase 2 studyPhase 3 studySeverity of pain