Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.
J Comp Eff Res
; 9(14): 973-984, 2020 10.
Article
em En
| MEDLINE
| ID: mdl-32851872
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.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Oxidiazóis
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Ensaios Clínicos Controlados Aleatórios como Assunto
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Códon sem Sentido
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Distrofia Muscular de Duchenne
Tipo de estudo:
Clinical_trials
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Systematic_reviews
Limite:
Humans
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article