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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
- Campbell, C. (author)
- Barohn, R. J. (author)
- Bertini, E. (author)
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- Chabrol, B. (author)
- Comi, G. P. (author)
- Darras, B. T. (author)
- Finkel, R. S. (author)
- Flanigan, K. M. (author)
- Goemans, N. (author)
- Jones, K. J. (author)
- Kirschner, J. (author)
- Mah, J. K. (author)
- Mathews, K. D. (author)
- McDonald, C. M. (author)
- Mercuri, E. (author)
- Nevo, Y. (author)
- Pereon, Y. (author)
- Ben Renfroe, J. (author)
- Ryan, M. M. (author)
- Sampson, J. B. (author)
- Schara, U. (author)
- Sejersen, T. (author)
- Selby, K. (author)
- Vilchez, J. J. (author)
- Voit, T. (author)
- Wei, L. J. (author)
- Wong, B. L. (author)
- Elfring, G. (author)
- Souza, M. (author)
- McIntosh, J. (author)
- Trifillis, P. (author)
- Peltz, S. W. (author)
- Muntoni, F. (author)
- P. TC124-GD-007-Study Grp, P. TC124-GD-007-Study Grp (author)
- Act Dmd Study Grp, Act Dmd Study Grp (author)
- Clinical Evaluator Training, Grp (author)
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- (creator_code:org_t)
- Becaris Publishing Limited, 2020
- 2020
- English.
- In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:14
- Journal article (peer-reviewed)
Abstract
Subject headings
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Annan medicin och hälsovetenskap (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Other Medical and Health Sciences (hsv//eng)
Keyword
- 6-minute walk distance
- ataluren
- Duchenne muscular dystrophy
- efficacy
- meta-analyses
- nonsense mutation Duchenne muscular dystrophy
- randomized
- controlled trials
- 6-minute walk test
- end-points
- multicenter
- Health Care Sciences & Services
Publication and Content Type
- ref (subject category)
- art (subject category)
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- Campbell, C.
- Barohn, R. J.
- Bertini, E.
- Chabrol, B.
- Comi, G. P.
- Darras, B. T.
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- Finkel, R. S.
- Flanigan, K. M.
- Goemans, N.
- Iannaccone, S. T ...
- Jones, K. J.
- Kirschner, J.
- Mah, J. K.
- Mathews, K. D.
- McDonald, C. M.
- Mercuri, E.
- Nevo, Y.
- Pereon, Y.
- Ben Renfroe, J.
- Ryan, M. M.
- Sampson, J. B.
- Schara, U.
- Sejersen, T.
- Selby, K.
- Tulinius, Mar, 1 ...
- Vilchez, J. J.
- Voit, T.
- Wei, L. J.
- Wong, B. L.
- Elfring, G.
- Souza, M.
- McIntosh, J.
- Trifillis, P.
- Peltz, S. W.
- Muntoni, F.
- P. TC124-GD-007- ...
- Act Dmd Study Gr ...
- Clinical Evaluat ...
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- University of Gothenburg
- Karolinska Institutet
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