2024
Validating new symptom emergence as a patient-centric outcome measure for PD clinical trials
Zou H, Stebbins G, Simuni T, Luo S, Cedarbaum J. Validating new symptom emergence as a patient-centric outcome measure for PD clinical trials. Parkinsonism & Related Disorders 2024, 128: 107118. PMID: 39353265, DOI: 10.1016/j.parkreldis.2024.107118.Peer-Reviewed Original ResearchPD clinical trialsClinical trialsEmergent symptomsPhase 3 clinical trialsPhase 3 studySlowing of disease progressionOutcome measuresParkinson's diseaseDe novo Parkinson's diseaseMDS-UPDRSItem-level dataFrequency of ESSymptomatic medicationsFrequent administrationMonths of observationEfficacy assessmentDisease progressionSymptom emergenceRating ScaleItem responsesIb and IIUrate elevationTrialsValidity of the Short Weekly Calendar Planning Activity in patients with Parkinson disease and nonmanifesting LRRK2 and GBA carriers
Schejter‐Margalit T, Binyamin N, Thaler A, Maidan I, Cedarbaum J, Orr‐Urtreger A, Weisz M, Goldstein O, Giladi N, Mirelman A, Kizony R. Validity of the Short Weekly Calendar Planning Activity in patients with Parkinson disease and nonmanifesting LRRK2 and GBA carriers. European Journal Of Neurology 2024, 31: e16327. PMID: 38743695, PMCID: PMC11235808, DOI: 10.1111/ene.16327.Peer-Reviewed Original ResearchKnown-groups validityWeekly Calendar Planning ActivityColor Trails TestActivities of Daily Living ScaleKnown-groups construct validityIdiopathic PDPhysical Activity ScaleDaily Living ScaleEngland Activities of Daily Living ScaleNonmanifesting carriersPD to healthy controlsIdiopathic PD groupMontreal Cognitive AssessmentEcological validitySE-ADLActivity ScaleCompare personsConstruct validityOutcome measuresConvergent validityCognitive AssessmentGBA carriersEngland ActivitiesParkinson's diseaseCognitive testsThe ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials
Genge A, Cedarbaum J, Shefner J, Chio A, Al-Chalabi A, Van Damme P, McDermott C, Glass J, Berry J, van Eijk R, Fournier C, Grosskreutz J, Andrews J, Bertone V, Bunte T, Couillard M, Cummings C, Kittle G, Polzer J, Salmon K, Straub C, van den Berg L. The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2024, 25: 382-387. PMID: 38396337, DOI: 10.1080/21678421.2024.2320880.Peer-Reviewed Original ResearchAmyotrophic Lateral Sclerosis Functional Rating ScaleALSFRS-RClinical trialsStatistical analysis planMonitor functional changesRating ScaleOutcome measuresFunctional Rating ScalePrimary outcome measureClinical trial designALS clinical trialsFunctional changesTrial designTrialsClinical trialistsScaleALS trials
2022
A qualitative evaluation of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) by the patient community: a web-based cross-sectional survey
Boyce D, Robinson M, Cedarbaum J, Shank L, McDermott C, van Eijk R. A qualitative evaluation of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) by the patient community: a web-based cross-sectional survey. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2022, 24: 272-280. PMID: 36330850, DOI: 10.1080/21678421.2022.2140592.Peer-Reviewed Original ResearchConceptsAmyotrophic Lateral Sclerosis Functional Rating ScaleFunctional Rating ScaleOutcome measuresRating ScaleWeb-based cross-sectional surveyAbility of patientsCross-sectional surveyALS studiesWeb-based surveyALSFRSPatient communityPerspectives of peopleCaregiversPatientsALSItem questionsLiteracy levelsTracking Emergence of New Motor and Non-Motor Symptoms Using the MDS-UPDRS: A Novel Outcome Measure for Early Parkinson’s Disease?
Tosin M, Simuni T, Stebbins G, Cedarbaum J. Tracking Emergence of New Motor and Non-Motor Symptoms Using the MDS-UPDRS: A Novel Outcome Measure for Early Parkinson’s Disease? Journal Of Parkinson's Disease 2022, 12: 1345-1351. PMID: 35466955, PMCID: PMC9198734, DOI: 10.3233/jpd-223170.Peer-Reviewed Original ResearchConceptsEmergent symptomsDisease progressionOutcome measuresParkinson's disease clinical trialsNon-motor symptomsEarly Parkinson's diseaseDaily Living ScalePatient-reported experiencesClinical rating scalesNovel outcome measuresSymptomatic treatmentPD progressionClinical trialsMedian numberDaily livingLiving ScaleParkinson's diseaseSummary scoresMDS-UPDRSUseful markerRating ScaleSTX groupProgressionStxSymptoms
2015
Impact of the Alzheimer's Disease Neuroimaging Initiative, 2004 to 2014
Weiner MW, Veitch DP, Aisen PS, Beckett LA, Cairns NJ, Cedarbaum J, Donohue MC, Green RC, Harvey D, Jack CR, Jagust W, Morris JC, Petersen RC, Saykin AJ, Shaw L, Thompson PM, Toga AW, Trojanowski JQ, Initiative A. Impact of the Alzheimer's Disease Neuroimaging Initiative, 2004 to 2014. Alzheimer's & Dementia 2015, 11: 865-884. PMID: 26194320, PMCID: PMC4659407, DOI: 10.1016/j.jalz.2015.04.005.Peer-Reviewed Original ResearchConceptsAlzheimer's Disease Neuroimaging InitiativeClinical trialsDisease Neuroimaging InitiativeAlzheimer's diseaseAD clinical trialsSurrogate outcome measureAD risk factorsTraumatic brain injuryPost-traumatic stress disorderAD risk allelesMultiple sclerosisRisk factorsBrain injuryAD progressionOutcome measuresEffective treatmentParkinson's diseaseImaging ligandsMilitary populationStress disorderRisk allelesDiseaseMultiple centersBiomarkersStandardized biomarkersCommonalities and Challenges in the Development of Clinical Trial Measures in Neurology
Cedarbaum JM, Stephenson D, Rudick R, Carrillo MC, Stebbins G, Kerr D, Heemskerk J, Galpern WR, Kaufmann P, Cella D, Isaac M, Walton MK. Commonalities and Challenges in the Development of Clinical Trial Measures in Neurology. Neurotherapeutics 2015, 12: 151-169. PMID: 25384682, PMCID: PMC4322066, DOI: 10.1007/s13311-014-0310-1.Peer-Reviewed Original ResearchConceptsOutcome measuresNeurological disordersDisease-specific outcome measuresClinical trial measuresAssessment of dysfunctionAutonomic system functionMotor functionNervous systemRelated disordersDisordersDisease areasAmerican SocietyDrug developmentDysfunctionTrial measuresTerms of cognitionNeurologyAssessment developmentSystem functionAnnual MeetingNeurologistsSymptomsNeurotherapeutics
2010
Seeing with new eyes: Finding a path to early intervention trials in Alzheimer's disease
Cedarbaum JM, Crans G, Grundman M. Seeing with new eyes: Finding a path to early intervention trials in Alzheimer's disease. The Journal Of Nutrition, Health & Aging 2010, 14: 306-309. PMID: 20306002, DOI: 10.1007/s12603-010-0069-y.Peer-Reviewed Original ResearchConceptsMild cognitive impairmentAlzheimer's diseaseOutcome measuresClinical Dementia Rating SumClinical staging algorithmsPrimary outcome measureAD clinical trialsEarly intervention trialsComprehensive outcome measuresIdentification of MCIClinical courseIntervention trialsAD patientsClinical trialsDementia pathologyClinical characterizationCognitive impairmentEarly identificationDegenerative disordersNormal cognitionStaging algorithmDiseaseBiomarker informationBiomarker dataTrials
2009
An exploratory study of the safety, tolerability and bioactivity of a single intravitreal injection of vascular endothelial growth factor Trap-Eye in patients with diabetic macular oedema
V D, Nguyen QD, Shah SM, Browning DJ, Haller JA, Chu K, Yang K, Cedarbaum JM, Vitti RL, Ingerman A, Campochiaro PA. An exploratory study of the safety, tolerability and bioactivity of a single intravitreal injection of vascular endothelial growth factor Trap-Eye in patients with diabetic macular oedema. British Journal Of Ophthalmology 2009, 93: 144. PMID: 19174400, DOI: 10.1136/bjo.2008.138271.Peer-Reviewed Original ResearchConceptsVEGF Trap-EyeDiabetic macular edemaSingle intravitreal injectionVascular endothelial growth factor (VEGF) Trap-EyeExcess foveal thicknessFoveal thicknessIntravitreal injectionOptical coherence tomographyMacular edemaUnrelated serious adverse eventsMedian baseline BCVASerious adverse eventsBaseline BCVAETDRS lettersAdverse eventsRetinal thicknessVisual acuityOcular toxicityOutcome measuresBCVAMedian improvementPatientsCoherence tomographyAdditional studiesObservation period
2006
Dynamic and Quantitative Analysis of Choroidal Neovascularization by Fluorescein Angiography
Shah SM, Tatlipinar S, Quinlan E, Sung JU, Tabandeh H, Nguyen QD, Fahmy AS, Zimmer-Galler I, Symons RC, Cedarbaum JM, Campochiaro PA. Dynamic and Quantitative Analysis of Choroidal Neovascularization by Fluorescein Angiography. Investigative Ophthalmology & Visual Science 2006, 47: 5460-5468. PMID: 17122137, DOI: 10.1167/iovs.06-0012.Peer-Reviewed Original ResearchConceptsFluorescein angiographyFluorescein angiogramsClinical trialsVEGF TrapVascular endothelial growth factor trapPlacebo-treated patientsPhotodynamic therapyChoroidal neovascularization lesion sizeAreas of hyperfluorescenceMacular volumeChoroidal neovascularizationRetrospective gradingOutcome measuresGroup 2Group 1Masked observersPatientsFluorescence areaLesion sizeAngiogramsClinical settingDye injectionInitial assessmentAUCTrials
2005
NT-3 promotes nerve regeneration and sensory improvement in CMT1A mouse models and in patients
Sahenk Z, Nagaraja HN, McCracken BS, King WM, Freimer ML, Cedarbaum JM, Mendell JR. NT-3 promotes nerve regeneration and sensory improvement in CMT1A mouse models and in patients. Neurology 2005, 65: 681-689. PMID: 16157899, DOI: 10.1212/01.wnl.0000171978.70849.c5.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAnimalsCharcot-Marie-Tooth DiseaseDisease Models, AnimalDouble-Blind MethodFemaleHumansMaleMiceMice, Neurologic MutantsMice, NudeMice, TransgenicMiddle AgedMyelin ProteinsNerve RegenerationNeurotrophin 3Pilot ProjectsRecovery of FunctionSciatic NeuropathySural NerveTransplantation, HeterologousTreatment OutcomeConceptsNeuropathy Impairment ScoreNT-3 groupNT-3 treatmentNeurotrophin-3Placebo groupAnimal modelsNerve regenerationNude mice axonsQuantitative muscle testingSural nerve biopsyPrimary outcome measurePeripheral myelin proteinPilot clinical studyTooth type 1ANerve biopsyMuscle testingAxonal regenerationClinical studiesImpairment scoresOutcome measuresPreclinical studiesMouse modelEndpoint measuresElectrophysiologic measurementsNude mice