2019
The Qualification of an Enrichment Biomarker for Clinical Trials Targeting Early Stages of Parkinson’s Disease
Stephenson D, Hill D, Cedarbaum JM, Tome M, Vamvakas S, Romero K, Conrado DJ, Dexter DT, Seibyl J, Jennings D, Nicholas T, Matthews D, Xie Z, Imam S, Maguire P, Russell D, Gordon MF, Stebbins GT, Somer E, Gallagher J, Roach A, Basseches P, Grosset D, Marek K, Consortium O. The Qualification of an Enrichment Biomarker for Clinical Trials Targeting Early Stages of Parkinson’s Disease. Journal Of Parkinson's Disease 2019, 9: 553-563. PMID: 31306141, PMCID: PMC6700608, DOI: 10.3233/jpd-191648.Peer-Reviewed Original ResearchConceptsEuropean Medicines AgencyParkinson's diseaseDopamine transporterEnrichment biomarkerClinical trialsPD subjectsParkinson's Progression Markers Initiative (PPMI) studyMotor Parkinson's diseaseParkinson Research ExaminationStriatal DAT bindingUPDRS part IISingle photon emission tomographyAppropriate patient selectionEarly Parkinson's diseaseIdiopathic PD patientsClinical trial populationsFuture clinical trialsClinical trial dataPhoton emission tomographyEarly motor symptomsTest therapeuticsIndependent predictorsTherapeutic trialsDopaminergic deficitMotor symptomsMolecular Neuroimaging of the Dopamine Transporter as a Patient Enrichment Biomarker for Clinical Trials for Early Parkinson's Disease
Romero K, Conrado D, Burton J, Nicholas T, Sinha V, Macha S, Ahamadi M, Cedarbaum J, Seibyl J, Marek K, Basseches P, Hill D, Somer E, Gallagher J, Dexter DT, Roach A, Stephenson D, Consortium F, Initiative T. Molecular Neuroimaging of the Dopamine Transporter as a Patient Enrichment Biomarker for Clinical Trials for Early Parkinson's Disease. Clinical And Translational Science 2019, 12: 240-246. PMID: 30706986, PMCID: PMC6510371, DOI: 10.1111/cts.12619.Peer-Reviewed Original ResearchMeSH KeywordsBiomarkersClinical Trials as TopicDisease ProgressionDopamine Plasma Membrane Transport ProteinsHumansModels, BiologicalMotor ActivityNeuroimagingParkinson DiseaseConceptsClinical trialsImaging biomarkersDopamine transporterEuropean Medicines Agency's CommitteeParkinson's disease clinical trialsEarly Parkinson's diseaseCritical Path InstitutePatient selectionEnrichment biomarkerParkinson's diseaseMolecular neuroimagingPharmaceutical partnersDiseaseBiomarkersTrialsQualification opinionHuman useInternational ConsortiumTransportersMedical productsNeuroimagingA Proposed Roadmap for Parkinson’s Disease Proof of Concept Clinical Trials Investigating Compounds Targeting Alpha-Synuclein
Merchant KM, Cedarbaum JM, Brundin P, Dave KD, Eberling J, Espay AJ, Hutten SJ, Javidnia M, Luthman J, Maetzler W, Menalled L, Reimer AN, Stoessl AJ, Weiner DM, . A Proposed Roadmap for Parkinson’s Disease Proof of Concept Clinical Trials Investigating Compounds Targeting Alpha-Synuclein. Journal Of Parkinson's Disease 2019, Preprint: 1-31. PMID: 30400107, PMCID: PMC6398545, DOI: 10.3233/jpd-181471.Peer-Reviewed Original ResearchConceptsParkinson's diseaseProgression of PDDisease-modifying therapiesConcept clinical trialMichael J. Fox FoundationDisease-modifying therapeuticsLewy pathologyClinical outcomesClinical trialsTargeting therapyAnimal modelsClinical proofAlpha-synucleinBiomarker toolkitΑ-synClinical researchInvestigational moleculesTherapyPD researchTranslational frameworkParkinson's ResearchDiseaseBiomarkersConcept studyMeaningful strides
2018
FDG PET Parkinson’s disease-related pattern as a biomarker for clinical trials in early stage disease
Matthews DC, Lerman H, Lukic A, Andrews RD, Mirelman A, Wernick MN, Giladi N, Strother SC, Evans KC, Cedarbaum JM, Even-Sapir E. FDG PET Parkinson’s disease-related pattern as a biomarker for clinical trials in early stage disease. NeuroImage Clinical 2018, 20: 572-579. PMID: 30186761, PMCID: PMC6120603, DOI: 10.1016/j.nicl.2018.08.006.Peer-Reviewed Original ResearchConceptsHealthy controlsDisease-related patternsParkinson's diseaseYahr stagePD patientsClinical trialsParkinson's disease-related patternFluorodeoxyglucose positron emission tomographyGender-matched healthy controlsEarly-stage Parkinson's diseaseMild PD patientsEarly-stage diseaseFDG-PET scansMotor symptom scoresPositron emission tomographyStage diseaseMotor symptomsSymptom scoresFDG-PETDisease stagePD progressionLentiform nucleusParacentral gyrusSymptom evaluationPD subjectsElephants, Parkinson's Disease, and Proof‐of‐Concept Clinical Trials
Cedarbaum JM. Elephants, Parkinson's Disease, and Proof‐of‐Concept Clinical Trials. Movement Disorders 2018, 33: 697-700. PMID: 29722454, DOI: 10.1002/mds.27398.Peer-Reviewed Original ResearchTargeted Therapies for Parkinson's Disease: From Genetics to the Clinic
Sardi SP, Cedarbaum JM, Brundin P. Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic. Movement Disorders 2018, 33: 684-696. PMID: 29704272, PMCID: PMC6282975, DOI: 10.1002/mds.27414.Peer-Reviewed Original ResearchMeSH KeywordsAlpha-SynucleinAnimalsClinical Trials as TopicGlucosylceramidaseHumansParkinson DiseaseProtein Serine-Threonine KinasesConceptsParkinson's diseaseGreat unmet medical needDisease-modifying treatmentsNew therapeutic approachesUnmet medical needClinical stageClinical trialsRelentless progressionTherapeutic approachesPotential therapyClinical developmentTherapeutic paradigmMedical needDiseaseGenetic variantsPD geneticsNew arsenalTreatmentGenetic discoveriesKey outstanding questionsTherapySymptomsPathologyProgressionTrials
2017
Biometric monitoring devices for assessing end points in clinical trials: developing an ecosystem
Arnerić SP, Cedarbaum JM, Khozin S, Papapetropoulos S, Hill DL, Ropacki M, Rhodes J, Dacks PA, Hudson LD, Gordon MF, Kern VD, Romero K, Vradenburg G, Au R, Karlin DR, Facheris MF, Fitzer-Attas CJ, Vitolo OV, Wang J, Miller BM, Kaye JA. Biometric monitoring devices for assessing end points in clinical trials: developing an ecosystem. Nature Reviews Drug Discovery 2017, 16: 736-736. PMID: 28935908, DOI: 10.1038/nrd.2017.153.Peer-Reviewed Original Research
2015
Item response theory analysis of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised in the Pooled Resource Open-Access ALS Clinical Trials Database
Bacci ED, Staniewska D, Coyne KS, Boyer S, White LA, Zach N, Cedarbaum JM, Consortium T. Item response theory analysis of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised in the Pooled Resource Open-Access ALS Clinical Trials Database. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2015, 17: 157-167. PMID: 26473473, DOI: 10.3109/21678421.2015.1095930.Peer-Reviewed Original ResearchConceptsAmyotrophic Lateral Sclerosis Functional Rating Scale-RevisedPooled Resource Open-Access ALS Clinical Trials DatabaseClinical trials databasesTrials databasesAmyotrophic Lateral Sclerosis Functional RatingModern test theory approachesLevel of disabilityALSFRS-R itemsMean ageDisability levelFunctional ratingGross motorDisability severitySevere disabilityPatientsConfirmatory factor analysisItem response theory analysisTreatment effectsDisabilityDomain itemsItem response categoriesResponse categoriesALSFRSInstrument's specificitySpecificityImpact 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 ResearchMeSH KeywordsAlzheimer DiseaseBiomarkersClinical Trials as TopicDatabases, BibliographicDisease ProgressionHumansLongitudinal StudiesNeuroimagingConceptsAlzheimer'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
2011
AMPA workshop on challenges faced by investigators conducting Alzheimer's disease clinical trials
Vellas B, Pesce A, Robert PH, Aisen PS, Ancoli‐Israel S, Andrieu S, Cedarbaum J, Dubois B, Siemers E, Spire J, Weiner MW, May TS. AMPA workshop on challenges faced by investigators conducting Alzheimer's disease clinical trials. Alzheimer's & Dementia 2011, 7: e109-e117. PMID: 21784343, DOI: 10.1016/j.jalz.2010.05.2020.Peer-Reviewed Original Research
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 ResearchMeSH KeywordsAlgorithmsAlzheimer DiseaseBiomarkersClinical Trials as TopicCognition DisordersDisease ProgressionHumansPatient SelectionSeverity of Illness IndexTreatment OutcomeConceptsMild 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
2004
Survival
Cedarbaum J. Survival. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2004, 5: 79-83. PMID: 15512880, DOI: 10.1080/17434470410020021.Peer-Reviewed Original ResearchAmyotrophic Lateral SclerosisClinical Trials as TopicEndpoint DeterminationHumansMeta-Analysis as TopicTime Factors
1996
Re: Pharmacokinetics and pharmacodynamics of rHCNTF in rodents
Cedarbaum J, Distcfano P, Lakings D, Lindsay R. Re: Pharmacokinetics and pharmacodynamics of rHCNTF in rodents. Annals Of Neurology 1996, 39: 552-553. PMID: 8619536, DOI: 10.1002/ana.410390420.Peer-Reviewed Original Research
1989
A double-blind crossover comparison of Sinemet CR4 and standard Sinemet 25/100 in patients with Parkinson's disease and fluctuating motor performance.
Cedarbaum JM, Hoey M, McDowell FH. A double-blind crossover comparison of Sinemet CR4 and standard Sinemet 25/100 in patients with Parkinson's disease and fluctuating motor performance. Journal Of Neurology Neurosurgery & Psychiatry 1989, 52: 207. PMID: 2649640, PMCID: PMC1032507, DOI: 10.1136/jnnp.52.2.207.Peer-Reviewed Original ResearchConceptsStandard Sinemet 25/100Sinemet 25/100Sinemet CR4Controlled-release levodopa/carbidopaDouble-blind crossover comparisonDaily medication dosesDouble-blind partMean interdose intervalLevodopa/carbidopaAntiparkinson effectMedication dosesInterdose intervalSevere dyskinesiaCrossover comparisonCrossover trialParkinsonian patientsTherapeutic responseMedication administrationMotor functionAntiparkinsonian agentsParkinson's diseaseOpen treatmentMotor responsePatientsMotor performance