Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT...
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| Veröffentlicht in: | Journal of comparative effectiveness research 2020-10, Vol.9 (14), p.973-984 |
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| creator | Campbell, Craig Barohn, Richard J Bertini, Enrico Chabrol, Brigitte Comi, Giacomo Pietro Darras, Basil T Finkel, Richard S Flanigan, Kevin M Goemans, Nathalie Iannaccone, Susan T Jones, Kristi J Kirschner, Janbernd Mah, Jean K Mathews, Katherine D McDonald, Craig M Mercuri, Eugenio Nevo, Yoram Péréon, Yann Renfroe, J Ben Ryan, Monique M Sampson, Jacinda B Schara, Ulrike Sejersen, Thomas Selby, Kathryn Tulinius, Már Vílchez, Juan J Voit, Thomas Wei, Lee-Jen Wong, Brenda L Elfring, Gary Souza, Marcio McIntosh, Joseph Trifillis, Panayiota Peltz, Stuart W Muntoni, Francesco |
| description | Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).
Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] ≥300– |
| doi_str_mv | 10.2217/cer-2020-0095 |
| format | Article |
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Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] ≥300–<400 or <400 m). Meta-analyses examined 6MWD change from baseline to week 48.
Statistically significant differences in 6MWD change with ataluren versus placebo were observed across all three meta-analyses. Least-squares mean difference (95% CI): ITT (n = 342), +17.2 (0.2–34.1) m, p = 0.0473; ≥300–<400 m (n = 143), +43.9 (18.2–69.6) m, p = 0.0008; <400 m (n = 216), +27.7 (6.4–49.0) m, p = 0.0109.
These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. Treatment benefit was most evident in patients with a baseline 6MWD ≥300–<400 m (the ambulatory transition phase), thereby informing future trial design.</description><identifier>ISSN: 2042-6305</identifier><identifier>EISSN: 2042-6313</identifier><identifier>DOI: 10.2217/cer-2020-0095</identifier><identifier>PMID: 32851872</identifier><language>eng</language><publisher>England: Future Medicine Ltd</publisher><subject>6-minute walk distance ; 6-minute walk test ; Annan medicin och hälsovetenskap ; ataluren ; Clinical trials ; Codon, Nonsense - genetics ; controlled trials ; Demographics ; Duchenne muscular dystrophy ; efficacy ; end-points ; Health Care Sciences & Services ; Humans ; meta-analyses ; Meta-analysis ; multicenter ; Muscular dystrophy ; Muscular Dystrophy, Duchenne - drug therapy ; Muscular Dystrophy, Duchenne - genetics ; Mutation ; nonsense mutation Duchenne muscular dystrophy ; Other Medical and Health Sciences ; Oxadiazoles - therapeutic use ; Patients ; randomized ; randomized controlled trials ; Randomized Controlled Trials as Topic ; Statistical analysis</subject><ispartof>Journal of comparative effectiveness research, 2020-10, Vol.9 (14), p.973-984</ispartof><rights>2020 Campbell C et al.</rights><rights>2020. This work is published under http://creativecommons.org/licenses/by-nc-nd/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c486t-b48c89ff77d215b8b2a8eb012cf0cdb7f19c0579e1b469219dca3d508bf3a7a63</citedby><cites>FETCH-LOGICAL-c486t-b48c89ff77d215b8b2a8eb012cf0cdb7f19c0579e1b469219dca3d508bf3a7a63</cites><orcidid>0000-0001-5216-7564</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>230,314,550,776,780,881,27901,27902</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/32851872$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink><backlink>$$Uhttps://gup.ub.gu.se/publication/297433$$DView record from Swedish Publication Index$$Hfree_for_read</backlink><backlink>$$Uhttp://kipublications.ki.se/Default.aspx?queryparsed=id:144520616$$DView record from Swedish Publication Index$$Hfree_for_read</backlink></links><search><creatorcontrib>Campbell, Craig</creatorcontrib><creatorcontrib>Barohn, Richard J</creatorcontrib><creatorcontrib>Bertini, Enrico</creatorcontrib><creatorcontrib>Chabrol, Brigitte</creatorcontrib><creatorcontrib>Comi, Giacomo Pietro</creatorcontrib><creatorcontrib>Darras, Basil T</creatorcontrib><creatorcontrib>Finkel, Richard S</creatorcontrib><creatorcontrib>Flanigan, Kevin M</creatorcontrib><creatorcontrib>Goemans, Nathalie</creatorcontrib><creatorcontrib>Iannaccone, Susan T</creatorcontrib><creatorcontrib>Jones, Kristi J</creatorcontrib><creatorcontrib>Kirschner, Janbernd</creatorcontrib><creatorcontrib>Mah, Jean K</creatorcontrib><creatorcontrib>Mathews, Katherine D</creatorcontrib><creatorcontrib>McDonald, Craig M</creatorcontrib><creatorcontrib>Mercuri, Eugenio</creatorcontrib><creatorcontrib>Nevo, Yoram</creatorcontrib><creatorcontrib>Péréon, Yann</creatorcontrib><creatorcontrib>Renfroe, J Ben</creatorcontrib><creatorcontrib>Ryan, Monique M</creatorcontrib><creatorcontrib>Sampson, Jacinda B</creatorcontrib><creatorcontrib>Schara, Ulrike</creatorcontrib><creatorcontrib>Sejersen, Thomas</creatorcontrib><creatorcontrib>Selby, Kathryn</creatorcontrib><creatorcontrib>Tulinius, Már</creatorcontrib><creatorcontrib>Vílchez, Juan J</creatorcontrib><creatorcontrib>Voit, Thomas</creatorcontrib><creatorcontrib>Wei, Lee-Jen</creatorcontrib><creatorcontrib>Wong, Brenda L</creatorcontrib><creatorcontrib>Elfring, Gary</creatorcontrib><creatorcontrib>Souza, Marcio</creatorcontrib><creatorcontrib>McIntosh, Joseph</creatorcontrib><creatorcontrib>Trifillis, Panayiota</creatorcontrib><creatorcontrib>Peltz, Stuart W</creatorcontrib><creatorcontrib>Muntoni, Francesco</creatorcontrib><creatorcontrib>PTC124-GD-007-DMD Study Group</creatorcontrib><creatorcontrib>Clinical Evaluator Training Groups</creatorcontrib><creatorcontrib>ACT DMD Study Group</creatorcontrib><creatorcontrib>the Clinical Evaluator Training Groups</creatorcontrib><creatorcontrib>on behalf of the PTC124-GD-007-DMD Study Group</creatorcontrib><title>Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy</title><title>Journal of comparative effectiveness research</title><addtitle>J Comp Eff Res</addtitle><description>Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).
Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] ≥300–<400 or <400 m). Meta-analyses examined 6MWD change from baseline to week 48.
Statistically significant differences in 6MWD change with ataluren versus placebo were observed across all three meta-analyses. Least-squares mean difference (95% CI): ITT (n = 342), +17.2 (0.2–34.1) m, p = 0.0473; ≥300–<400 m (n = 143), +43.9 (18.2–69.6) m, p = 0.0008; <400 m (n = 216), +27.7 (6.4–49.0) m, p = 0.0109.
These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. Treatment benefit was most evident in patients with a baseline 6MWD ≥300–<400 m (the ambulatory transition phase), thereby informing future trial design.</description><subject>6-minute walk distance</subject><subject>6-minute walk test</subject><subject>Annan medicin och hälsovetenskap</subject><subject>ataluren</subject><subject>Clinical trials</subject><subject>Codon, Nonsense - genetics</subject><subject>controlled trials</subject><subject>Demographics</subject><subject>Duchenne muscular dystrophy</subject><subject>efficacy</subject><subject>end-points</subject><subject>Health Care Sciences & Services</subject><subject>Humans</subject><subject>meta-analyses</subject><subject>Meta-analysis</subject><subject>multicenter</subject><subject>Muscular dystrophy</subject><subject>Muscular Dystrophy, Duchenne - drug therapy</subject><subject>Muscular Dystrophy, Duchenne - genetics</subject><subject>Mutation</subject><subject>nonsense mutation Duchenne muscular dystrophy</subject><subject>Other Medical and Health Sciences</subject><subject>Oxadiazoles - therapeutic use</subject><subject>Patients</subject><subject>randomized</subject><subject>randomized controlled trials</subject><subject>Randomized Controlled Trials as Topic</subject><subject>Statistical analysis</subject><issn>2042-6305</issn><issn>2042-6313</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BENPR</sourceid><sourceid>D8T</sourceid><recordid>eNp9kc1rFTEUxQex2FK7dCsDbtyM5msmyVKqVaHipq5DkrnzmjqTjPlAnn99M7znEwQNgdwcfudyuadpXmD0hhDM31qIHUEEdQjJ_klzQRAj3UAxfXqqUX_eXKX0gOoZBJM9edacUyJ6LDi5aHZfIOtOez3vE6Q2TK3Oei4RfBu1H8PifsHY2uBzDPNcyxydnlPrfOuDT1Bvu5Ssswu-fV_sPXi_KcmWWcd23KdqXO_3z5uzqfrg6vheNt9uPtxdf-puv378fP3utrNMDLkzTFghp4nzkeDeCEO0AIMwsROyo-ETlhb1XAI2bJAEy9FqOvZImIlqrgd62XSHvuknrMWoNbpFx70K2qmj9L1WoNggBib_y-_Kqqq0KxtPJGeUVv71gV9j-FEgZbW4ZGGetYdQkiKMcsEIFbyir_5CH0KJddOV6hHHPRdc_BnAxpBShOk0AkZqC1nVkNUWstpCrvzLY9diFhhP9O9IKyAPwFRyzTFZB96COvyqw1nn4R_NHwFj77ig</recordid><startdate>20201001</startdate><enddate>20201001</enddate><creator>Campbell, Craig</creator><creator>Barohn, Richard J</creator><creator>Bertini, Enrico</creator><creator>Chabrol, Brigitte</creator><creator>Comi, Giacomo Pietro</creator><creator>Darras, Basil T</creator><creator>Finkel, Richard S</creator><creator>Flanigan, Kevin M</creator><creator>Goemans, Nathalie</creator><creator>Iannaccone, Susan T</creator><creator>Jones, Kristi J</creator><creator>Kirschner, Janbernd</creator><creator>Mah, Jean K</creator><creator>Mathews, Katherine D</creator><creator>McDonald, Craig M</creator><creator>Mercuri, Eugenio</creator><creator>Nevo, Yoram</creator><creator>Péréon, Yann</creator><creator>Renfroe, J Ben</creator><creator>Ryan, Monique M</creator><creator>Sampson, Jacinda B</creator><creator>Schara, Ulrike</creator><creator>Sejersen, Thomas</creator><creator>Selby, Kathryn</creator><creator>Tulinius, Már</creator><creator>Vílchez, Juan J</creator><creator>Voit, Thomas</creator><creator>Wei, Lee-Jen</creator><creator>Wong, Brenda L</creator><creator>Elfring, Gary</creator><creator>Souza, Marcio</creator><creator>McIntosh, Joseph</creator><creator>Trifillis, Panayiota</creator><creator>Peltz, Stuart W</creator><creator>Muntoni, Francesco</creator><general>Future Medicine Ltd</general><scope>FUMOA</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7X7</scope><scope>7XB</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>BENPR</scope><scope>CCPQU</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>K9.</scope><scope>M0S</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>7X8</scope><scope>ADTPV</scope><scope>AOWAS</scope><scope>F1U</scope><scope>D8T</scope><scope>ZZAVC</scope><orcidid>https://orcid.org/0000-0001-5216-7564</orcidid></search><sort><creationdate>20201001</creationdate><title>Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy</title><author>Campbell, Craig ; Barohn, Richard J ; Bertini, Enrico ; Chabrol, Brigitte ; Comi, Giacomo Pietro ; Darras, Basil T ; Finkel, Richard S ; Flanigan, Kevin M ; Goemans, Nathalie ; Iannaccone, Susan T ; Jones, Kristi J ; Kirschner, Janbernd ; Mah, Jean K ; Mathews, Katherine D ; McDonald, Craig M ; Mercuri, Eugenio ; Nevo, Yoram ; Péréon, Yann ; Renfroe, J Ben ; Ryan, Monique M ; Sampson, Jacinda B ; Schara, Ulrike ; Sejersen, Thomas ; Selby, Kathryn ; Tulinius, Már ; Vílchez, Juan J ; Voit, Thomas ; Wei, Lee-Jen ; Wong, Brenda L ; Elfring, Gary ; Souza, Marcio ; McIntosh, Joseph ; Trifillis, Panayiota ; Peltz, Stuart W ; Muntoni, Francesco</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c486t-b48c89ff77d215b8b2a8eb012cf0cdb7f19c0579e1b469219dca3d508bf3a7a63</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2020</creationdate><topic>6-minute walk distance</topic><topic>6-minute walk test</topic><topic>Annan medicin och hälsovetenskap</topic><topic>ataluren</topic><topic>Clinical trials</topic><topic>Codon, Nonsense - 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Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] ≥300–<400 or <400 m). Meta-analyses examined 6MWD change from baseline to week 48.
Statistically significant differences in 6MWD change with ataluren versus placebo were observed across all three meta-analyses. Least-squares mean difference (95% CI): ITT (n = 342), +17.2 (0.2–34.1) m, p = 0.0473; ≥300–<400 m (n = 143), +43.9 (18.2–69.6) m, p = 0.0008; <400 m (n = 216), +27.7 (6.4–49.0) m, p = 0.0109.
These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. Treatment benefit was most evident in patients with a baseline 6MWD ≥300–<400 m (the ambulatory transition phase), thereby informing future trial design.</abstract><cop>England</cop><pub>Future Medicine Ltd</pub><pmid>32851872</pmid><doi>10.2217/cer-2020-0095</doi><tpages>12</tpages><orcidid>https://orcid.org/0000-0001-5216-7564</orcidid><oa>free_for_read</oa></addata></record> |
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| identifier | ISSN: 2042-6305 |
| ispartof | Journal of comparative effectiveness research, 2020-10, Vol.9 (14), p.973-984 |
| issn | 2042-6305 2042-6313 |
| language | eng |
| recordid | cdi_swepub_primary_oai_swepub_ki_se_468649 |
| source | MEDLINE; PubMed Central; Alma/SFX Local Collection; SWEPUB Freely available online |
| subjects | 6-minute walk distance 6-minute walk test Annan medicin och hälsovetenskap ataluren Clinical trials Codon, Nonsense - genetics controlled trials Demographics Duchenne muscular dystrophy efficacy end-points Health Care Sciences & Services Humans meta-analyses Meta-analysis multicenter Muscular dystrophy Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - genetics Mutation nonsense mutation Duchenne muscular dystrophy Other Medical and Health Sciences Oxadiazoles - therapeutic use Patients randomized randomized controlled trials Randomized Controlled Trials as Topic Statistical analysis |
| title | Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-02-06T01%3A01%3A03IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_swepu&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Meta-analyses%20of%20ataluren%20randomized%20controlled%20trials%20in%20nonsense%20mutation%20Duchenne%20muscular%20dystrophy&rft.jtitle=Journal%20of%20comparative%20effectiveness%20research&rft.au=Campbell,%20Craig&rft.aucorp=PTC124-GD-007-DMD%20Study%20Group&rft.date=2020-10-01&rft.volume=9&rft.issue=14&rft.spage=973&rft.epage=984&rft.pages=973-984&rft.issn=2042-6305&rft.eissn=2042-6313&rft_id=info:doi/10.2217/cer-2020-0095&rft_dat=%3Cproquest_swepu%3E2437842387%3C/proquest_swepu%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=2507157878&rft_id=info:pmid/32851872&rfr_iscdi=true |