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 symptoms
1997
Performance of the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) in multicenter clinical trials
Cedarbaum J, Stambler N. Performance of the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) in multicenter clinical trials. Journal Of The Neurological Sciences 1997, 152: s1-s9. PMID: 9419047, DOI: 10.1016/s0022-510x(97)00237-2.Peer-Reviewed Original ResearchConceptsAmyotrophic Lateral Sclerosis Functional Rating ScaleFunctional Rating ScaleMulticenter clinical trialFunctional statusRating ScaleClinical trialsPatients' functional statusClinical Global ImpressionMuscle strength testingArea of feedingPlacebo patientsTherapeutic trialsTest-retest reliabilityGlobal ImpressionPatient levelTreatment studiesChange ScaleTrialsPatientsMaximum scoreIndependent assessmentStrength testingAmbulation