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

PTC124-GD-007-DMD Study Group, ACT DMD Study Group, Clinical Evaluator Training Groups, Craig Campbell, Richard J. Barohn, Enrico Bertini, Brigitte Chabrol, Giacomo Pietro Comi, Basil T. Darras, Richard S. Finkel, Kevin M. Flanigan, Nathalie Goemans, Susan T. Iannaccone, Kristi J. Jones, Janbernd Kirschner, Jean K. Mah, Katherine D. Mathews, Craig M. McDonald, Eugenio Mercuri, Yoram NevoYann Péréon, J. Ben Renfroe, Monique M. Ryan, Jacinda B. Sampson, Ulrike Schara, Thomas Sejersen, Kathryn Selby, Már Tulinius, Juan J. Vílchez, Thomas Voit, Lee Jen Wei, Brenda L. Wong, Gary Elfring, Marcio Souza, Joseph McIntosh, Panayiota Trifillis, Stuart W. Peltz, Francesco Muntoni

Research output: Contribution to journalArticlepeer-review


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 ( 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.

Original languageEnglish
Pages (from-to)973-984
Number of pages12
JournalJournal of Comparative Effectiveness Research
Issue number14
Publication statusPublished - Oct 1 2020


  • 6-minute walk distance
  • ataluren
  • Duchenne muscular dystrophy
  • efficacy
  • meta-analyses
  • nonsense mutation Duchenne muscular dystrophy
  • randomized controlled trials

ASJC Scopus subject areas

  • Health Policy

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