Key result
Ataluren significantly improved 6-minute walk distance change from baseline to week 48 compared to placebo in patients with nmDMD (mean difference +17.2 m; 95% CI 0.2-34.1; p=0.0473).
Why the study?
To assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy.
Does ataluren improve 6-minute walk distance in patients with nonsense mutation Duchenne muscular dystrophy?
Meta-Analysis (n=342)
Does ataluren improve 6-minute walk distance in patients with nonsense mutation Duchenne muscular dystrophy?
Ataluren significantly slows disease progression, as measured by 6-minute walk distance, compared to placebo in patients with nonsense mutation Duchenne muscular dystrophy over 48 weeks.
Answers are grounded in this paper and cite their sources.
Supports ataluren to slow progression in nonsense mutation DMD; confirms benefit on 6MWD via meta-analysis.
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.
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Campbell et al. (2020) conducted a meta-analysis in nonsense mutation Duchenne muscular dystrophy (nmDMD) (n=342). ataluren vs. placebo was evaluated on 6-min walk distance (6MWD) change from baseline to week 48 (MD +17.2, 95% CI 0.2-34.1, p=0.0473). Ataluren significantly improved 6-minute walk distance change from baseline to week 48 compared to placebo in patients with nmDMD (mean difference +17.2 m; 95% CI 0.2-34.1; p=0.0473).
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