A responsive outcome for Parkinson's disease neuroprotection futility studies
Identifieur interne : 000A84 ( Main/Corpus ); précédent : 000A83; suivant : 000A85A responsive outcome for Parkinson's disease neuroprotection futility studies
Auteurs : Jordan J. Elm ; Christopher G. Goetz ; Bernard Ravina ; Kathleen Shannon ; George Fredrick Wooten ; Caroline M. Tanner ; Yuko Y. Palesch ; Peng Huang ; Paulo Guimaraes ; Cornelia Kamp ; Barbara C. Tilley ; Karl KieburtzSource :
- Annals of Neurology [ 0364-5134 ] ; 2005-02.
Abstract
Futility studies are designed to test new treatments over a short period in a small number of subjects to determine if those treatments are worthy of larger and longer term studies, or if they should be abandoned. An appropriate outcome measure for a neuroprotection futility study in Parkinson's disease (sensitive to tracking disease progression in the short‐term) has not been determined. Data sets from three clinical trials were used to compare Parkinson's disease outcome measures. Total Unified Parkinson's Disease Rating Scale (UPDRS; Mentation + Activities of Daily Living + Motor) change and Motor plus Activities of Daily Living UPDRS change, measured in untreated patients, required the smallest sample sizes of all the outcome measures explored. Other outcomes (UPDRS Motor, UPDRS Activities of Daily Living, and time to need levodopa) required somewhat larger sample sizes. Futility designs in Parkinson's disease are feasible in terms of short duration and small sample size requirements, and this design is being applied in two ongoing Parkinson's disease studies to select agents for future larger and longer term neuroprotection studies. Ann Neurol 2005;57:197–203
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DOI: 10.1002/ana.20361
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An appropriate outcome measure for a neuroprotection futility study in Parkinson's disease (sensitive to tracking disease progression in the short‐term) has not been determined. Data sets from three clinical trials were used to compare Parkinson's disease outcome measures. Total Unified Parkinson's Disease Rating Scale (UPDRS; Mentation + Activities of Daily Living + Motor) change and Motor plus Activities of Daily Living UPDRS change, measured in untreated patients, required the smallest sample sizes of all the outcome measures explored. Other outcomes (UPDRS Motor, UPDRS Activities of Daily Living, and time to need levodopa) required somewhat larger sample sizes. Futility designs in Parkinson's disease are feasible in terms of short duration and small sample size requirements, and this design is being applied in two ongoing Parkinson's disease studies to select agents for future larger and longer term neuroprotection studies. 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Palesch PhD</name><affiliations><json:string>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</json:string></affiliations></json:item><json:item><name>Peng Huang PhD</name><affiliations><json:string>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</json:string></affiliations></json:item><json:item><name>Paulo Guimaraes PhD</name><affiliations><json:string>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</json:string></affiliations></json:item><json:item><name>Cornelia Kamp MBA, CCRC</name><affiliations><json:string>Department of Neurology, University of Rochester, Clinical Trials Coordination Center, Rochester, NY</json:string></affiliations></json:item><json:item><name>Barbara C. Tilley PhD</name><affiliations><json:string>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</json:string></affiliations></json:item><json:item><name>Karl Kieburtz MD, MPH</name><affiliations><json:string>Department of Neurology, University of Rochester, Clinical Trials Coordination Center, Rochester, NY</json:string></affiliations></json:item></author><articleId><json:string>ANA20361</json:string></articleId><language><json:string>eng</json:string></language><abstract>Futility studies are designed to test new treatments over a short period in a small number of subjects to determine if those treatments are worthy of larger and longer term studies, or if they should be abandoned. An appropriate outcome measure for a neuroprotection futility study in Parkinson's disease (sensitive to tracking disease progression in the short‐term) has not been determined. Data sets from three clinical trials were used to compare Parkinson's disease outcome measures. Total Unified Parkinson's Disease Rating Scale (UPDRS; Mentation + Activities of Daily Living + Motor) change and Motor plus Activities of Daily Living UPDRS change, measured in untreated patients, required the smallest sample sizes of all the outcome measures explored. Other outcomes (UPDRS Motor, UPDRS Activities of Daily Living, and time to need levodopa) required somewhat larger sample sizes. Futility designs in Parkinson's disease are feasible in terms of short duration and small sample size requirements, and this design is being applied in two ongoing Parkinson's disease studies to select agents for future larger and longer term neuroprotection studies. 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No. U01NS43128;</note></notesStmt><sourceDesc><biblStruct type="inbook"><analytic><title level="a" type="main" xml:lang="en">A responsive outcome for Parkinson's disease neuroprotection futility studies</title><author><persName><forename type="first">Jordan J.</forename><surname>Elm</surname></persName><roleName type="degree">MA</roleName><note type="correspondence"><p>Correspondence: Medical University of South Carolina, Department of Biostatistics, Bioinformatics & Epidemiology, 135 Cannon St., Suite 303, Charleston, SC 29425</p></note><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation></author><author><persName><forename type="first">Christopher G.</forename><surname>Goetz</surname></persName><roleName type="degree">MD</roleName><affiliation>Department of Neurological Sciences, Rush‐Presbyterian‐St. Luke's Medical Center, Chicago, IL</affiliation></author><author><persName><forename type="first">Bernard</forename><surname>Ravina</surname></persName><roleName type="degree">MD</roleName><affiliation>National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD</affiliation></author><author><persName><forename type="first">Kathleen</forename><surname>Shannon</surname></persName><roleName type="degree">MD</roleName><affiliation>Department of Neurological Sciences, Rush‐Presbyterian‐St. Luke's Medical Center, Chicago, IL</affiliation></author><author><persName><forename type="first">George Fredrick</forename><surname>Wooten</surname></persName><roleName type="degree">MD</roleName><affiliation>Department of Neurology, University of Virginia Health Sciences Center, Charlottesville, VA</affiliation></author><author><persName><forename type="first">Caroline M.</forename><surname>Tanner</surname></persName><roleName type="degree">MD, PhD</roleName><affiliation>The Parkinson's Institute, Sunnyvale, CA</affiliation></author><author><persName><forename type="first">Yuko Y.</forename><surname>Palesch</surname></persName><roleName type="degree">PhD</roleName><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation></author><author><persName><forename type="first">Peng</forename><surname>Huang</surname></persName><roleName type="degree">PhD</roleName><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation></author><author><persName><forename type="first">Paulo</forename><surname>Guimaraes</surname></persName><roleName type="degree">PhD</roleName><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation></author><author><persName><forename type="first">Cornelia</forename><surname>Kamp</surname></persName><roleName type="degree">MBA, CCRC</roleName><affiliation>Department of Neurology, University of Rochester, Clinical Trials Coordination Center, Rochester, NY</affiliation></author><author><persName><forename type="first">Barbara C.</forename><surname>Tilley</surname></persName><roleName type="degree">PhD</roleName><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation></author><author><persName><forename type="first">Karl</forename><surname>Kieburtz</surname></persName><roleName type="degree">MD, MPH</roleName><affiliation>Department of Neurology, University of Rochester, Clinical Trials Coordination Center, Rochester, NY</affiliation></author></analytic><monogr><title level="j">Annals of Neurology</title><title level="j" type="abbrev">Ann Neurol.</title><idno type="pISSN">0364-5134</idno><idno type="eISSN">1531-8249</idno><idno type="DOI">10.1002/(ISSN)1531-8249</idno><imprint><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher><pubPlace>Hoboken</pubPlace><date type="published" when="2005-02"></date><biblScope unit="volume">57</biblScope><biblScope unit="issue">2</biblScope><biblScope unit="page" from="197">197</biblScope><biblScope unit="page" to="203">203</biblScope></imprint></monogr><idno type="istex">6CEF53E8734AAC44FB6D21ADF120CA2E615353D0</idno><idno type="DOI">10.1002/ana.20361</idno><idno type="ArticleID">ANA20361</idno></biblStruct></sourceDesc></fileDesc><profileDesc><creation><date>2005</date></creation><langUsage><language ident="en">en</language></langUsage><abstract xml:lang="en"><p>Futility studies are designed to test new treatments over a short period in a small number of subjects to determine if those treatments are worthy of larger and longer term studies, or if they should be abandoned. 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corresponding="yes"><personName><givenNames>Jordan J.</givenNames><familyName>Elm</familyName><degrees>MA</degrees></personName><contactDetails><email>elmj@musc.edu</email></contactDetails></creator><creator xml:id="au2" creatorRole="author" affiliationRef="#af2"><personName><givenNames>Christopher G.</givenNames><familyName>Goetz</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au3" creatorRole="author" affiliationRef="#af3"><personName><givenNames>Bernard</givenNames><familyName>Ravina</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au4" creatorRole="author" affiliationRef="#af2"><personName><givenNames>Kathleen</givenNames><familyName>Shannon</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au5" creatorRole="author" affiliationRef="#af4"><personName><givenNames>George Fredrick</givenNames><familyName>Wooten</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au6" creatorRole="author" affiliationRef="#af5"><personName><givenNames>Caroline M.</givenNames><familyName>Tanner</familyName><degrees>MD, PhD</degrees></personName></creator><creator xml:id="au7" creatorRole="author" affiliationRef="#af1"><personName><givenNames>Yuko Y.</givenNames><familyName>Palesch</familyName><degrees>PhD</degrees></personName></creator><creator xml:id="au8" creatorRole="author" affiliationRef="#af1"><personName><givenNames>Peng</givenNames><familyName>Huang</familyName><degrees>PhD</degrees></personName></creator><creator xml:id="au9" creatorRole="author" affiliationRef="#af1"><personName><givenNames>Paulo</givenNames><familyName>Guimaraes</familyName><degrees>PhD</degrees></personName></creator><creator xml:id="au10" creatorRole="author" affiliationRef="#af6"><personName><givenNames>Cornelia</givenNames><familyName>Kamp</familyName><degrees>MBA, CCRC</degrees></personName></creator><creator xml:id="au11" creatorRole="author" affiliationRef="#af1"><personName><givenNames>Barbara C.</givenNames><familyName>Tilley</familyName><degrees>PhD</degrees></personName></creator><creator xml:id="au12" creatorRole="author" affiliationRef="#af6"><personName><givenNames>Karl</givenNames><familyName>Kieburtz</familyName><degrees>MD, MPH</degrees></personName></creator></creators><affiliationGroup><affiliation xml:id="af1" countryCode="US" type="organization"><unparsedAffiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</unparsedAffiliation></affiliation><affiliation xml:id="af2" countryCode="US" type="organization"><unparsedAffiliation>Department of Neurological Sciences, Rush‐Presbyterian‐St. Luke's Medical Center, Chicago, IL</unparsedAffiliation></affiliation><affiliation xml:id="af3" countryCode="US" type="organization"><unparsedAffiliation>National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD</unparsedAffiliation></affiliation><affiliation xml:id="af4" countryCode="US" type="organization"><unparsedAffiliation>Department of Neurology, University of Virginia Health Sciences Center, Charlottesville, VA</unparsedAffiliation></affiliation><affiliation xml:id="af5" countryCode="US" type="organization"><unparsedAffiliation>The Parkinson's Institute, Sunnyvale, CA</unparsedAffiliation></affiliation><affiliation xml:id="af6" countryCode="US" type="organization"><unparsedAffiliation>Department of Neurology, University of Rochester, Clinical Trials Coordination Center, Rochester, NY</unparsedAffiliation></affiliation></affiliationGroup><fundingInfo><fundingAgency>NIH (National Institute of Neurological Disorders and Stroke)</fundingAgency><fundingNumber>U01NS043127</fundingNumber><fundingNumber>U01NS43128</fundingNumber></fundingInfo><abstractGroup><abstract type="main" xml:lang="en"><title type="main">Abstract</title><p>Futility studies are designed to test new treatments over a short period in a small number of subjects to determine if those treatments are worthy of larger and longer term studies, or if they should be abandoned. An appropriate outcome measure for a neuroprotection futility study in Parkinson's disease (sensitive to tracking disease progression in the short‐term) has not been determined. Data sets from three clinical trials were used to compare Parkinson's disease outcome measures. Total Unified Parkinson's Disease Rating Scale (UPDRS; Mentation + Activities of Daily Living + Motor) change and Motor plus Activities of Daily Living UPDRS change, measured in untreated patients, required the smallest sample sizes of all the outcome measures explored. Other outcomes (UPDRS Motor, UPDRS Activities of Daily Living, and time to need levodopa) required somewhat larger sample sizes. Futility designs in Parkinson's disease are feasible in terms of short duration and small sample size requirements, and this design is being applied in two ongoing Parkinson's disease studies to select agents for future larger and longer term neuroprotection studies. Ann Neurol 2005;57:197–203</p></abstract></abstractGroup></contentMeta></header></component></istex:document></istex:metadataXml><mods version="3.6"><titleInfo lang="en"><title>A responsive outcome for Parkinson's disease neuroprotection futility studies</title></titleInfo><titleInfo type="abbreviated" lang="en"><title>PD Outcome Measures</title></titleInfo><titleInfo type="alternative" contentType="CDATA" lang="en"><title>A responsive outcome for Parkinson's disease neuroprotection futility studies</title></titleInfo><name type="personal"><namePart type="given">Jordan J.</namePart><namePart type="family">Elm</namePart><namePart type="termsOfAddress">MA</namePart><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation><description>Correspondence: Medical University of South Carolina, Department of Biostatistics, Bioinformatics & Epidemiology, 135 Cannon St., Suite 303, Charleston, SC 29425</description><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Christopher G.</namePart><namePart type="family">Goetz</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Department of Neurological Sciences, Rush‐Presbyterian‐St. Luke's Medical Center, Chicago, IL</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Bernard</namePart><namePart type="family">Ravina</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Kathleen</namePart><namePart type="family">Shannon</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Department of Neurological Sciences, Rush‐Presbyterian‐St. Luke's Medical Center, Chicago, IL</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">George Fredrick</namePart><namePart type="family">Wooten</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Department of Neurology, University of Virginia Health Sciences Center, Charlottesville, VA</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Caroline M.</namePart><namePart type="family">Tanner</namePart><namePart type="termsOfAddress">MD, PhD</namePart><affiliation>The Parkinson's Institute, Sunnyvale, CA</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Yuko Y.</namePart><namePart type="family">Palesch</namePart><namePart type="termsOfAddress">PhD</namePart><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Peng</namePart><namePart type="family">Huang</namePart><namePart type="termsOfAddress">PhD</namePart><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Paulo</namePart><namePart type="family">Guimaraes</namePart><namePart type="termsOfAddress">PhD</namePart><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Cornelia</namePart><namePart type="family">Kamp</namePart><namePart type="termsOfAddress">MBA, CCRC</namePart><affiliation>Department of Neurology, University of Rochester, Clinical Trials Coordination Center, Rochester, NY</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Barbara C.</namePart><namePart type="family">Tilley</namePart><namePart type="termsOfAddress">PhD</namePart><affiliation>Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Karl</namePart><namePart type="family">Kieburtz</namePart><namePart type="termsOfAddress">MD, MPH</namePart><affiliation>Department of Neurology, University of Rochester, Clinical Trials Coordination Center, Rochester, NY</affiliation><role><roleTerm type="text">author</roleTerm></role></name><typeOfResource>text</typeOfResource><genre type="article" displayLabel="article"></genre><originInfo><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher><place><placeTerm type="text">Hoboken</placeTerm></place><dateIssued encoding="w3cdtf">2005-02</dateIssued><dateCaptured encoding="w3cdtf">2004-07-26</dateCaptured><dateValid encoding="w3cdtf">2004-10-13</dateValid><copyrightDate encoding="w3cdtf">2005</copyrightDate></originInfo><language><languageTerm type="code" authority="rfc3066">en</languageTerm><languageTerm type="code" authority="iso639-2b">eng</languageTerm></language><physicalDescription><internetMediaType>text/html</internetMediaType><extent unit="figures">1</extent><extent unit="tables">1</extent><extent unit="references">28</extent><extent unit="words">5901</extent></physicalDescription><abstract lang="en">Futility studies are designed to test new treatments over a short period in a small number of subjects to determine if those treatments are worthy of larger and longer term studies, or if they should be abandoned. An appropriate outcome measure for a neuroprotection futility study in Parkinson's disease (sensitive to tracking disease progression in the short‐term) has not been determined. Data sets from three clinical trials were used to compare Parkinson's disease outcome measures. Total Unified Parkinson's Disease Rating Scale (UPDRS; Mentation + Activities of Daily Living + Motor) change and Motor plus Activities of Daily Living UPDRS change, measured in untreated patients, required the smallest sample sizes of all the outcome measures explored. Other outcomes (UPDRS Motor, UPDRS Activities of Daily Living, and time to need levodopa) required somewhat larger sample sizes. Futility designs in Parkinson's disease are feasible in terms of short duration and small sample size requirements, and this design is being applied in two ongoing Parkinson's disease studies to select agents for future larger and longer term neuroprotection studies. Ann Neurol 2005;57:197–203</abstract><note type="funding">NIH (National Institute of Neurological Disorders and Stroke) - No. U01NS043127; No. U01NS43128; </note><relatedItem type="host"><titleInfo><title>Annals of Neurology</title></titleInfo><titleInfo type="abbreviated"><title>Ann Neurol.</title></titleInfo><genre type="Journal">journal</genre><subject><genre>article category</genre><topic>Original Article</topic></subject><identifier type="ISSN">0364-5134</identifier><identifier type="eISSN">1531-8249</identifier><identifier type="DOI">10.1002/(ISSN)1531-8249</identifier><identifier type="PublisherID">ANA</identifier><part><date>2005</date><detail type="volume"><caption>vol.</caption><number>57</number></detail><detail type="issue"><caption>no.</caption><number>2</number></detail><extent unit="pages"><start>197</start><end>203</end><total>7</total></extent></part></relatedItem><identifier type="istex">6CEF53E8734AAC44FB6D21ADF120CA2E615353D0</identifier><identifier type="DOI">10.1002/ana.20361</identifier><identifier type="ArticleID">ANA20361</identifier><accessCondition type="use and reproduction" contentType="copyright">Copyright © 2005 American Neurological Association</accessCondition><recordInfo><recordContentSource>WILEY</recordContentSource><recordOrigin>Wiley Subscription Services, Inc., A Wiley Company</recordOrigin></recordInfo></mods></metadata><serie></serie></istex></record>Pour manipuler ce document sous Unix (Dilib)
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