2024
Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN)
Garcia-Manero G, McCloskey J, Scott B, Griffiths E, Kiner-Strachan B, Brunner A, Zeidan A, Traer E, Madanat Y, Meyer J, Erba H, Baratam P, Borate U, Sano Y, Oganesian A, Zhu L, Keer H, Savona M. Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN). Blood 2024, 144: 662. DOI: 10.1182/blood-2024-203569.Peer-Reviewed Original ResearchDose-limiting toxicityPhase 1 trialDose-expansion partMDS/MPN overlap syndromesMyelodysplastic syndromeAdverse eventsDose combinationMDS/myeloproliferative neoplasmImmediate-releaseOverlap syndromeAUC exposureOpen-label phase 1 trialDelayed-releaseCytidine deaminaseProlonged grade 4 neutropeniaIncreased absolute bioavailabilityMarrow complete responsePhase 2 doseGrade 4 neutropeniaDose-escalation partErythropoiesis-stimulating agentsAcute myeloid leukemiaTreatment of patientsClinical efficacy assessmentDNA methyltransferase inhibitor
2023
A Randomized, Double‐blind, Sham‐controlled, Adaptive‐Design Pivotal Trial of Sensory Stimulation in Subjects with Alzheimer’s Disease
Boasso A, Houser C, Hajos M, Newberger J, Seshagiri C, Leach J, Konisky A, Galley A, Hempel E, Dickson S, Hendrix S, Malchano Z, Megerian J. A Randomized, Double‐blind, Sham‐controlled, Adaptive‐Design Pivotal Trial of Sensory Stimulation in Subjects with Alzheimer’s Disease. Alzheimer's & Dementia 2023, 19 DOI: 10.1002/alz.080670.Peer-Reviewed Original ResearchMini-Mental State ExamClinical Dementia Rating ScaleZarit Burden IndexPivotal trialsSensory stimulationAlzheimer's Disease Cooperative Study-ActivitiesConcurrent medication useOccipital cortical thicknessKey secondary outcomesProspective clinical trialsPrimary outcome measureSelf-administered therapyClinical laboratory assessmentsClinical efficacy assessmentBrain structural changesDaily living testDementia Rating ScaleSubgroup of subjectsMental State ExamAlzheimer's disease symptomsHOPE trialSecondary outcomesAdverse eventsMedication useADCS-ADL
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