Is It Possible to Conduct a Multi-Arm Multi-Stage Platform Trial in Parkinson’s Disease: Lessons Learned from Other Neurodegenerative Disorders and Cancer
dc.contributor.author | Zeissler, M-L | |
dc.contributor.author | Li, V | |
dc.contributor.author | Parmar, MKB | |
dc.contributor.author | Carroll, Camille | |
dc.date.accessioned | 2020-04-17T11:44:23Z | |
dc.date.available | 2020-04-17T11:44:23Z | |
dc.date.issued | 2020-04-03 | |
dc.identifier.issn | 1877-7171 | |
dc.identifier.issn | 1877-718X | |
dc.identifier.uri | http://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.extent | 413-428 | |
dc.format.medium | ||
dc.language | eng | |
dc.language.iso | en | |
dc.publisher | IOS Press | |
dc.subject | Parkinson's disease | |
dc.subject | adaptive clinical trial | |
dc.subject | outcome measure | |
dc.subject | clinical trial protocol | |
dc.subject | multiple sclerosis | |
dc.subject | motor neuron disease | |
dc.title | Is It Possible to Conduct a Multi-Arm Multi-Stage Platform Trial in Parkinson’s Disease: Lessons Learned from Other Neurodegenerative Disorders and Cancer | |
dc.type | journal-article | |
dc.type | Journal Article | |
dc.type | Research Support, Non-U.S. Gov't | |
dc.type | Review | |
plymouth.author-url | https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000526818700007&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=11bb513d99f797142bcfeffcc58ea008 | |
plymouth.issue | 2 | |
plymouth.volume | 10 | |
plymouth.publication-status | Published | |
plymouth.journal | Journal of Parkinson's Disease | |
dc.identifier.doi | 10.3233/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.place | Netherlands | |
dcterms.dateAccepted | 2020-02-07 | |
dc.rights.embargodate | 2020-6-6 | |
dc.identifier.eissn | 1877-718X | |
dc.rights.embargoperiod | Not known | |
rioxxterms.versionofrecord | 10.3233/jpd-191856 | |
rioxxterms.licenseref.uri | http://www.rioxx.net/licenses/all-rights-reserved | |
rioxxterms.licenseref.startdate | 2020-04-03 | |
rioxxterms.type | Journal Article/Review |