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LeaDrye,PhDJohnsHopkinsUniversityTypesofTrialDesignCopyright©2013JohnsHopkinsUniversityandLeaDrye.AllRightsReserved.LeaDrye,PhDJohnsHopkinsUni

PhaseI:FirststageintestinganewinterventioninhumansUsually10-30peopleIdentifytolerabledose,provideinformationondrugmetabolism,excretion,andtoxicityOftennotcontrolled

PhaseII:Usually30-100peoplePreliminaryinformationonefficacy,additionalinformationonsafetyandsideeffects

PhaseIII:Usually100+peopleAssessefficacyandsafetyControlled,usuallyrandomized2Phasesoftrials PhaseI:PhasesoftrialsLectureOutline

DiscussvarioustrialdesigntypesParallelCrossoverGroupallocationFactorialLargesimpleEquivalencyNon-inferiorityAdaptive3LectureOutline DiscussvariComparisonStructure:Parallel,Crossover,andGroupAllocationDesignsSectionAThematerialinthisvideoissubjecttothecopyrightoftheownersofthematerialandisbeingprovidedforeducationalpurposesunderrulesoffairuseforregisteredstudentsinthiscourseonly.Noadditionalcopiesofthecopyrightedworkmaybemadeordistributed.ComparisonStructure:ParallelParallelDesign5

Simultaneoustreatmentandcontrolgroups

Eachpersonisrandomlyassignedtoonetreatmentgroup

Randomizationremovestreatmentselectionbiasandpromotescomparabilityoftreatmentgroups

StatisticalcomparisonsmadebetweentreatmentgroupsParallelDesign5 Simultaneou|P叫ωe G巾Randomized「占v1，白6iJHSPH|P叫ωe G巾Randomized「占v1，白6iJHSPParallelDesignExample:NETTSource:NETTResearchGroup(1999).Chest1999;116:1750-61;NETTResearchGroup(1999).JThoracCardiovascSurg,118:518-528;Fishman,A.,&Martinez,F.,etal.(2003).NEnglJMed348:2059-73.7

NationalEmphysemaTreatmentTrial(NETT)- PhaseIIItrial,unmaskedPopulationPeoplewithsevereemphysemaSamplesize1,200AllocationtotreatmentRandomizedTreatments—Lungvolumereductionsurgeryplusmedicaltherapy—Medicaltherapy(standardtherapycontrol)ParallelDesignExample:NETTSParallelDesignExample:NETTSource:NETTResearchGroup(1999).Chest1999;116:1750-61;NETTResearchGroup(1999).JThoracCardiovascSurg,118:518-528;Fishman,A.,&Martinez,F.,etal.(2003).NEnglJMed348:2059-73.8HypothesistestingSuperiorityOutcomes—Primary:mortality,exercisecapacity—Secondary:qualityoflife,symptoms,lungfunctionandmechanics,functionalcapacityFollow-upUpto7.5yearsNumberofrecruitingcentersMulti-center(17)ParallelDesignExample:NETTSCrossoverDesign9

RandomizationoforderinwhichtreatmentsarereceivedABorBARandomizationpromotesbalancebetweentreatmentgroupsintimingofexposure

TestingofbothtreatmentsineachpatientEachpatientservesashis/herowncontrolVariabilityreducedbecauselessvariabilitywithinpatientthanbetweenpatients

FewerpatientsneededCrossoverDesign9 RandomizatCrossoverDesignGraphWashout10Group1,TxAGroup1,TxBGroup2,TxBGroup2,TxACrossoverDesignGraphWashout1CrossoverDesign:Disadvantages11

Treatmentcan’thavepermanenteffectsorcures

Potentialcarry-overeffectsoffirst-periodtreatmenttosecondperiodWashoutneedstobelongenoughUnequalcarry-overeffectsTreatmentduringwashout

Testforperiodbytreatmentinteractionsnotpowerful

Dropoutsmoresignificant

AnalysismaybemoredifficultCrossoverDesign:DisadvantageCrossoverDesign:Uses12

ConstantintensityofunderlyingdiseaseChronicdiseases—asthma,hypertension,arthritis

Short-termtreatmenteffectsReliefofsignsorsymptomsofdisease

Metabolic,bioavailability,ortolerabilitystudiesCrossoverDesign:Uses12 ConCrossoverDesign:Examples13

Evening-dosevs.morning-dosedtravoprostinopen-angleglaucomafor24-hourintraocularpressurecontrol

Montelukastvs.salmeterolasadjuvanttoinhaledfluticasoneforexercise-inducedasthmainchildren

Topicaloilvs.placeboforneuropathicpainCrossoverDesign:Examples13GroupAllocationDesign14

Alsoknownas“clusterrandomization”

Randomizationunitisagroupofindividuals(community,school,clinic)

IndividualrandomizationandinterventionisnotfeasibleorisunacceptableTrackingContamination

Ifthereisacorrelationintheresponseswithinagroup,designlosessomeefficiency(moreindividualsrequired)GroupAllocationDesign14 Allα[Ij川臼15Randomized〈〈lα[Ij川臼15Randomized〈〈GroupAllocationExample:SommerVitAtrial16Lancet.1986May24;1(8491):1169-73

PopulationPreschoolchildreninnorthernSumatrain1982-83

TreatmentsVitaminAsupplementationduringstudyVitaminAsupplementationafterstudy

ClustersVillages(450)selectedusingsurveysamplingmethodEachrandomlyallocatedtoonetreatmentGroupAllocationExample:SommLeaDrye,PhDJohnsHopkinsUniversityTypesofTrialDesignCopyright©2013JohnsHopkinsUniversityandLeaDrye.AllRightsReserved.LeaDrye,PhDJohnsHopkinsUni

PhaseI:FirststageintestinganewinterventioninhumansUsually10-30peopleIdentifytolerabledose,provideinformationondrugmetabolism,excretion,andtoxicityOftennotcontrolled

PhaseII:Usually30-100peoplePreliminaryinformationonefficacy,additionalinformationonsafetyandsideeffects

PhaseIII:Usually100+peopleAssessefficacyandsafetyControlled,usuallyrandomized2Phasesoftrials PhaseI:PhasesoftrialsLectureOutline

DiscussvarioustrialdesigntypesParallelCrossoverGroupallocationFactorialLargesimpleEquivalencyNon-inferiorityAdaptive3LectureOutline DiscussvariComparisonStructure:Parallel,Crossover,andGroupAllocationDesignsSectionAThematerialinthisvideoissubjecttothecopyrightoftheownersofthematerialandisbeingprovidedforeducationalpurposesunderrulesoffairuseforregisteredstudentsinthiscourseonly.Noadditionalcopiesofthecopyrightedworkmaybemadeordistributed.ComparisonStructure:ParallelParallelDesign21

Simultaneoustreatmentandcontrolgroups

Eachpersonisrandomlyassignedtoonetreatmentgroup

Randomizationremovestreatmentselectionbiasandpromotescomparabilityoftreatmentgroups

StatisticalcomparisonsmadebetweentreatmentgroupsParallelDesign5 Simultaneou|P叫ωe G巾Randomized「占v1，白22iJHSPH|P叫ωe G巾Randomized「占v1，白6iJHSPParallelDesignExample:NETTSource:NETTResearchGroup(1999).Chest1999;116:1750-61;NETTResearchGroup(1999).JThoracCardiovascSurg,118:518-528;Fishman,A.,&Martinez,F.,etal.(2003).NEnglJMed348:2059-73.23

NationalEmphysemaTreatmentTrial(NETT)- PhaseIIItrial,unmaskedPopulationPeoplewithsevereemphysemaSamplesize1,200AllocationtotreatmentRandomizedTreatments—Lungvolumereductionsurgeryplusmedicaltherapy—Medicaltherapy(standardtherapycontrol)ParallelDesignExample:NETTSParallelDesignExample:NETTSource:NETTResearchGroup(1999).Chest1999;116:1750-61;NETTResearchGroup(1999).JThoracCardiovascSurg,118:518-528;Fishman,A.,&Martinez,F.,etal.(2003).NEnglJMed348:2059-73.24HypothesistestingSuperiorityOutcomes—Primary:mortality,exercisecapacity—Secondary:qualityoflife,symptoms,lungfunctionandmechanics,functionalcapacityFollow-upUpto7.5yearsNumberofrecruitingcentersMulti-center(17)ParallelDesignExample:NETTSCrossoverDesign25

RandomizationoforderinwhichtreatmentsarereceivedABorBARandomizationpromotesbalancebetweentreatmentgroupsintimingofexposure

TestingofbothtreatmentsineachpatientEachpatientservesashis/herowncontrolVariabilityreducedbecauselessvariabilitywithinpatientthanbetweenpatients

FewerpatientsneededCrossoverDesign9 RandomizatCrossoverDesignGraphWashout26Group1,TxAGroup1,TxBGroup2,TxBGroup2,TxACrossoverDesignGraphWashout1CrossoverDesign:Disadvantages27

Treatmentcan’thavepermanenteffectsorcures

Potentialcarry-overeffectsoffirst-periodtreatmenttosecondperiodWashoutneedstobelongenoughUnequalcarry-overeffectsTreatmentduringwashout

Testforperiodbytreatmentinteractionsnotpowerful

Dropoutsmoresignificant

AnalysismaybemoredifficultCrossoverDesign:DisadvantageCrossoverDesign:Uses28

ConstantintensityofunderlyingdiseaseChronicdiseases—asthma,hypertension,arthritis

Short-termtreatmenteffectsReliefofsignsorsymptomsofdisease

Metabolic,bioavailability,ortolerabilitystudiesCrossoverDesign:Uses12 ConCrossoverDesign:Examples29

Evening-dosevs.morning-dosedtravoprostinopen-angleglaucomafor24-hourintraocularpressurecontrol

Montelukastvs.sa

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