2022
Trial of Cinpanemab in Early Parkinson’s Disease
Lang A, Siderowf A, Macklin E, Poewe W, Brooks D, Fernandez H, Rascol O, Giladi N, Stocchi F, Tanner C, Postuma R, Simon D, Tolosa E, Mollenhauer B, Cedarbaum J, Fraser K, Xiao J, Evans K, Graham D, Sapir I, Inra J, Hutchison R, Yang M, Fox T, Budd Haeberlein S, Dam T. Trial of Cinpanemab in Early Parkinson’s Disease. New England Journal Of Medicine 2022, 387: 408-420. PMID: 35921450, DOI: 10.1056/nejmoa2203395.Peer-Reviewed Original ResearchConceptsEarly Parkinson's diseasePrimary end pointSecondary end pointsWeek 52Parkinson's diseaseEnd pointControl groupUnified Parkinson's Disease Rating Scale (UPDRS) total scoreDisease pathogenesisHuman-derived monoclonal antibodiesΑ-synucleinCommon adverse eventsPhase 2 trialDisease-modifying treatmentsMDS-UPDRS scoresLack of efficacyDAT SPECT imagingAdjusted mean differenceMovement Disorder SocietyParkinson's disease pathogenesisScale total scoreAdverse eventsIntravenous infusionDAT-SPECTDisease progression
2020
Low cerebrospinal fluid volume and the risk for post-lumbar puncture headaches
Droby A, Omer N, Gurevich T, Kestenbaum M, Mina Y, Cedarbaum JM, Aizenstein O, Giladi N, Mirelman A, Thaler A. Low cerebrospinal fluid volume and the risk for post-lumbar puncture headaches. Journal Of The Neurological Sciences 2020, 417: 117059. PMID: 32739500, DOI: 10.1016/j.jns.2020.117059.Peer-Reviewed Original ResearchConceptsPost-LP headachePost-lumbar puncture headacheLow CSF volumeT1-weighted imagesCerebrospinal fluidCSF volumePuncture headacheDisease-modifying clinical trialsLow cerebrospinal fluid (CSF) volumePatient-related factorsGradient-echo T1-weighted imagesCerebrospinal fluid volumeLP headacheAdverse eventsMedical workupClinical trialsBrain MRINeurological conditionsHeadacheMRI scansT MR scannerWhite matterCSF collectionGray matterPilot study
2019
Randomized phase I clinical trial of anti–α‐synuclein antibody BIIB054
Brys M, Fanning L, Hung S, Ellenbogen A, Penner N, Yang M, Welch M, Koenig E, David E, Fox T, Makh S, Aldred J, Goodman I, Pepinsky B, Liu Y, Graham D, Weihofen A, Cedarbaum JM. Randomized phase I clinical trial of anti–α‐synuclein antibody BIIB054. Movement Disorders 2019, 34: 1154-1163. PMID: 31211448, PMCID: PMC6771554, DOI: 10.1002/mds.27738.Peer-Reviewed Original ResearchConceptsParkinson's disease participantsΑ-synucleinHealthy volunteersParkinson's diseaseHuman-derived monoclonal antibodiesSingle-dose cohortsMost adverse eventsFurther clinical developmentImmunotherapy targetingStudy drugAdverse eventsFavorable safetySingle doseNeuronal dysfunctionSerum ratioDisease progressionCerebrospinal fluidClinical developmentPharmacokinetic parametersPharmacokinetic profileSerum exposureLaboratory assessmentMonoclonal antibodiesDiseaseDose rangeFeasibility and safety of lumbar puncture in the Parkinson's disease research participants: Parkinson's Progression Marker Initiative (PPMI)
Prakash N, Caspell-Garcia C, Coffey C, Siderowf A, Tanner C, Kieburtz K, Mollenhauer B, Galasko D, Merchant K, Foroud T, Chahine L, Weintraub D, Casaceli C, Dorsey R, Wilson R, Herzog M, Daegele N, Arnedo V, Frasier M, Sherer T, Marek K, Frank S, Jennings D, Simuni T, Marek K, Siderowf A, Seibyl J, Coffey C, Tanner C, Tosun-Turgut D, Simuni T, Shaw L, Trojanowski J, Singleton A, Kieburtz K, Toga A, Mollenhauer B, Galasko D, Poewe W, Foroud T, Poston K, Sherer T, Chowdhury S, Frasier M, Kopil C, Arnedo V, Marek K, Daegele N, Casaceli C, Dorsey R, Wilson R, Mahes S, Seibyl J, Salerno C, Coffey C, Caspell-Garcia C, Toga A, Crawford K, Foroud T, Casalin P, Malferrari G, Weisz M, Orr-Urtreger A, Trojanowski J, Shaw L, Singleton A, Foroud T, Foroud T, Montine T, Foroud T, Russell D, Tanner C, Simuni T, Dahodwala N, Mollenhauer B, Galasko D, Poewe W, Giladi N, Factor S, Hogarth P, Standaert D, Hauser R, Jankovic J, Saint-Hilaire M, Richard I, Shprecher D, Fernandez H, Brockmann K, Rosenthal L, Barone P, Espay A, Rowe D, Marder K, Santiago A, Bressman S, Hu S, Isaacson S, Corvol J, Martinez J, Tolosa E, Tai Y, Politis M, Smejdir D, Rees L, Williams K, Kausar F, Williams K, Richardson W, Willeke D, Peacock S, Heim B, Mirelman A, Sommerfeld B, Freed A, Wakeman K, Blair C, Guthrie S, Harrell L, Hunter C, Thomas C, James R, Zimmerman G, Brown V, Mule J, Hilt E, Ribb K, Ainscough S, Wethington M, Ranola M, Santana H, Moreno J, Raymond D, Speketer K, Carvajal L, Carvalho S, Croitoru I, Garrido A, Payne L, Viswanth V, Severt L, Facheris M, Soares H, Mintun M, Cedarbaum J, Taylor P, Biglan K, Vandenbroucke E, Sheikh Z, Bingol B, Fischer T, Sardi P, Forrat R, Reith A, Egebjerg J, Hillert G, Saba B, Min C, Umek R, Mather J, De Santi S, Post A, Boess F, Taylor K, Grachev I, Avbersek A, Muglia P, Merchant K, Tauscher J. Feasibility and safety of lumbar puncture in the Parkinson's disease research participants: Parkinson's Progression Marker Initiative (PPMI). Parkinsonism & Related Disorders 2019, 62: 201-209. PMID: 30738748, PMCID: PMC8978879, DOI: 10.1016/j.parkreldis.2018.12.025.Peer-Reviewed Original ResearchConceptsParkinson's Progression Markers InitiativeProgression Markers InitiativeAdverse eventsLumbar punctureParkinson's diseasePD participantsBaseline lumbar puncturePhone one weekSerial lumbar puncturesCommon adverse eventsRelated adverse eventsCerebrospinal fluid analysisEarly Parkinson's diseaseLow back painLongitudinal observation studyPPMI participantsUnderwent collectionBack painOverall incidenceDopaminergic deficiencyProgression biomarkersFemale genderSafety dataHealthy volunteersHigh incidence
2012
Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis
Shefner J, Cedarbaum JM, Cudkowicz ME, Maragakis N, Lee J, Jones D, Watson ML, Mahoney K, Chen M, Saikali K, Mao J, Russell AJ, Hansen RL, Malik F, Wolff AA, Team F. Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2012, 13: 430-438. PMID: 22591195, DOI: 10.3109/17482968.2012.684214.Peer-Reviewed Original ResearchConceptsAmyotrophic lateral sclerosisSingle dosesGlobal ImpressionLateral sclerosisFast skeletal muscle troponin activatorFrequent adverse eventsDose-related fashionLimb muscle strengthMaximum voluntary ventilationDose-dependent benefitMeasures of enduranceAdverse eventsPulmonary functionVoluntary ventilationGeneral fatigueTroponin activatorMuscle strengthPharmacodynamic markersHandgrip endurancePatientsRandom orderMaximal strengthDosesTolerabilityFurther studies
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
A Phase I Trial of an IV-Administered Vascular Endothelial Growth Factor Trap for Treatment in Patients with Choroidal Neovascularization due to Age-Related Macular Degeneration
Nguyen QD, Shah SM, Hafiz G, Quinlan E, Sung J, Chu K, Cedarbaum JM, Campochiaro PA, Group C. A Phase I Trial of an IV-Administered Vascular Endothelial Growth Factor Trap for Treatment in Patients with Choroidal Neovascularization due to Age-Related Macular Degeneration. Ophthalmology 2006, 113: 1522.e1-1522.e14. PMID: 16876249, DOI: 10.1016/j.ophtha.2006.05.055.Peer-Reviewed Original ResearchMeSH KeywordsAgedAged, 80 and overChoroidal NeovascularizationDose-Response Relationship, DrugFemaleFluorescein AngiographyHumansInfusions, IntravenousMacular DegenerationMaleMaximum Tolerated DoseMiddle AgedReceptors, Vascular Endothelial Growth FactorRecombinant Fusion ProteinsRetinaTomography, Optical CoherenceVisual AcuityConceptsVascular endothelial growth factor trapAge-related macular degenerationVEGF TrapRetinal thicknessVisual acuityMacular degenerationEarly Treatment Diabetic Retinopathy Study protocolNeovascular age-related macular degenerationDiabetic Retinopathy Study protocolPlacebo-controlled clinical trialDoses 2 weeksDose-limiting toxicityPhase I trialMean percent changeVEGF receptor 1Optical coherence tomographyAdverse eventsI trialDose groupChoroidal neovascularizationStudy protocolClinical trialsStudy populationMultiple administrationsTherapeutic window
2003
Neurotrophin-3 Improves Functional Constipation
Parkman HP, Rao SS, Reynolds JC, Schiller LR, Wald A, Miner PB, Lembo AJ, Gordon JM, Drossman DA, Waltzman L, Stambler N, Cedarbaum JM. Neurotrophin-3 Improves Functional Constipation. The American Journal Of Gastroenterology 2003, 98: ajg2003312. PMID: 12818279, DOI: 10.1111/j.1572-0241.2003.t01-1-07477.x.Peer-Reviewed Original ResearchConceptsComplete bowel movementsNeurotrophin-3Bowel movementsColon transitFunctional constipationChronic constipationStool frequencyPlacebo-controlled phase II studyTransient injection site reactionsConstipation-related symptomsFrequent adverse eventsPhase II studyThird of patientsInjection site reactionsEnd of treatmentDose-related effectsConstipated subjectsBowel functionPrimary endpointAdverse eventsII studyImproved symptomsWeekly dosingNeurotrophic factorConstipation
2001
Disease status and use of ventilatory support by ALS patients
Cedarbaum J, Stambler N. Disease status and use of ventilatory support by ALS patients. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2001, 2: 19-22. PMID: 11465928, DOI: 10.1080/146608201300079373.Peer-Reviewed Original ResearchConceptsMechanical ventilationPlacebo patientsUse of MVRespiratory adverse eventsUse of BiPAPHealth resource utilizationPhase III trialsBiPAP useR-metHuBDNFProspective databaseSymptom durationVentilatory supportAdverse eventsIII trialsALS patientsMean baselinePrescribing practicesVital capacityBehavior of physiciansBiPAPALSFRS scorePatientsMortality rateDisease statusTrials