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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy

Campbell, C; Barohn, RJ; Bertini, E; Chabrol, B; Comi, GP; Darras, BT; Finkel, RS; ... Clinical Evaluator Training Groups; + view all (2020) Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy. Journal of Comparative Effectiveness Research 10.2217/cer-2020-0095. (In press). Green open access

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Abstract

Aim: Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). / Materials & methods: 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. / Results: 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. / Conclusion: 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.

Type: Article
Title: Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.2217/cer-2020-0095
Publisher version: https://doi.org/10.2217/cer-2020-0095
Language: English
Additional information: This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/
Keywords: 6-minute walk distance, Duchenne muscular dystrophy, ataluren, efficacy, meta-analyses, nonsense mutation Duchenne muscular dystrophy, randomized controlled trials
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10109632
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