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dc.contributor.authorZeissler, M-L
dc.contributor.authorLi, V
dc.contributor.authorParmar, MKB
dc.contributor.authorCarroll, Camille
dc.date.accessioned2020-04-17T11:44:23Z
dc.date.available2020-04-17T11:44:23Z
dc.date.issued2020-04-03
dc.identifier.issn1877-7171
dc.identifier.issn1877-718X
dc.identifier.urihttp://hdl.handle.net/10026.1/15573
dc.description.abstract

Many potential disease modifying therapies have been identified as suitable for clinical evaluation in Parkinson's disease (PD). Currently, the evaluation of compounds in phase II and phase III clinical trials in PD are set up in isolation, a process that is lengthy, costly and lacks efficiency. This review will introduce the concept of a multi-arm, multi-stage (MAMS) trial platform which allows for the assessment of several potential therapies at once, transitioning seamlessly from a phase II safety and efficacy study to a phase III trial by means of an interim analysis. At the interim checkpoint, ineffective arms are dropped and replaced by new treatment arms, thereby allowing for the continuous evaluation of interventions. MAMS trial platforms already exist for prostate, renal and oropharyngeal cancer and are currently being developed for progressive multiple sclerosis (PMS) and motor neuron disease (MND) within the UK. As a MAMS trial will evaluate many potential treatments it is of critical importance that a widely endorsed core protocol is developed which will investigate outcomes and objectives meaningful to patients. This review will discuss the challenges of drug selection, trial design, stratification and outcome measures and will share strategies implemented in the planned MAMS trials for MND and PMS that may be of interest to the PD field.

dc.format.extent413-428
dc.format.mediumPrint
dc.languageeng
dc.language.isoen
dc.publisherIOS Press
dc.subjectParkinson's disease
dc.subjectadaptive clinical trial
dc.subjectoutcome measure
dc.subjectclinical trial protocol
dc.subjectmultiple sclerosis
dc.subjectmotor neuron disease
dc.titleIs It Possible to Conduct a Multi-Arm Multi-Stage Platform Trial in Parkinson’s Disease: Lessons Learned from Other Neurodegenerative Disorders and Cancer
dc.typejournal-article
dc.typeJournal Article
dc.typeResearch Support, Non-U.S. Gov't
dc.typeReview
plymouth.author-urlhttps://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000526818700007&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=11bb513d99f797142bcfeffcc58ea008
plymouth.issue2
plymouth.volume10
plymouth.publication-statusPublished
plymouth.journalJournal of Parkinson's Disease
dc.identifier.doi10.3233/jpd-191856
plymouth.organisational-group/Plymouth
plymouth.organisational-group/Plymouth/Faculty of Health
plymouth.organisational-group/Plymouth/Faculty of Health/Peninsula Medical School
plymouth.organisational-group/Plymouth/REF 2021 Researchers by UoA
plymouth.organisational-group/Plymouth/REF 2021 Researchers by UoA/UoA03 Allied Health Professions, Dentistry, Nursing and Pharmacy
plymouth.organisational-group/Plymouth/Research Groups
plymouth.organisational-group/Plymouth/Research Groups/FoH - Applied Parkinson's Research
plymouth.organisational-group/Plymouth/Research Groups/FoH - Community and Primary Care
plymouth.organisational-group/Plymouth/Research Groups/Institute of Translational and Stratified Medicine (ITSMED)
plymouth.organisational-group/Plymouth/Research Groups/Institute of Translational and Stratified Medicine (ITSMED)/CCT&PS
plymouth.organisational-group/Plymouth/Research Groups/Plymouth Institute of Health and Care Research (PIHR)
plymouth.organisational-group/Plymouth/Users by role
plymouth.organisational-group/Plymouth/Users by role/Academics
dc.publisher.placeNetherlands
dcterms.dateAccepted2020-02-07
dc.rights.embargodate2020-6-6
dc.identifier.eissn1877-718X
dc.rights.embargoperiodNot known
rioxxterms.versionofrecord10.3233/jpd-191856
rioxxterms.licenseref.urihttp://www.rioxx.net/licenses/all-rights-reserved
rioxxterms.licenseref.startdate2020-04-03
rioxxterms.typeJournal Article/Review


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