Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). 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...
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| Published in | Journal of comparative effectiveness research Vol. 9; no. 14; pp. 973 - 984 |
|---|---|
| Main Authors | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
England
Future Medicine Ltd
01.10.2020
|
| Subjects | |
| Online Access | Get full text |
| ISSN | 2042-6305 2042-6313 2042-6313 |
| DOI | 10.2217/cer-2020-0095 |
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| Abstract | Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).
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.
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.
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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| AbstractList | 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.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. 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. 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. Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). 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. 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. 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. 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. |
| Author | Jones, Kristi J Flanigan, Kevin M Wong, Brenda L Goemans, Nathalie Mathews, Katherine D Muntoni, Francesco McDonald, Craig M Tulinius, Már Comi, Giacomo Pietro Darras, Basil T Renfroe, J Ben Campbell, Craig Iannaccone, Susan T Finkel, Richard S Ryan, Monique M Chabrol, Brigitte Elfring, Gary McIntosh, Joseph Trifillis, Panayiota Schara, Ulrike Voit, Thomas Vílchez, Juan J Sampson, Jacinda B Bertini, Enrico Mercuri, Eugenio Mah, Jean K Péréon, Yann Sejersen, Thomas Nevo, Yoram Wei, Lee-Jen Barohn, Richard J Kirschner, Janbernd Selby, Kathryn Souza, Marcio Peltz, Stuart W |
| AuthorAffiliation | 9Nationwide Children’s Hospital, Columbus, OH 43205, USA 28NIHR Great Ormond Street Hospital Biomedical Research Centre, University College London & UCL Great Ormond Street Institute of Child Health, Great Ormond Street Hospital Trust, London, WC1N 1EH, UK 1Schulich School of Medicine & Dentistry, Western University, London, ON, N6A 5C1, Canada 4Hôpital de la Timone, Unité de Médecine Infantile, Marseille, 13385, France 3Bambino Gesù Children’s Research Hospital, Rome, 00146, Italy 24Karolinska University Hospital, Karolinska Institutet, Stockholm, 171 76, Sweden 25Division of Neurology, British Columbia Children’s Hospital, Vancouver, BC, V6H 3N1, Canada 23Department of Pediatric Neurology, University Hospital Essen, University of Duisburg-Essen, Essen, 45122, Germany 17Department of Pediatric Neurology, Catholic University, Rome, 00168, Italy 12Kids Neuroscience, The Children’s Hospital at Westmead, Westmead, NSW, 2145, Australia 30University of Massachusetts Medical School, UMass, Worcester, |
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| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/32851872$$D View this record in MEDLINE/PubMed https://gup.ub.gu.se/publication/297433$$DView record from Swedish Publication Index http://kipublications.ki.se/Default.aspx?queryparsed=id:144520616$$DView record from Swedish Publication Index |
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| References | McDonald CM (e_1_3_8_19_2) 2018; 17 McDonald CM (e_1_3_8_18_2) 2013; 48 Birnkrant DJ (e_1_3_8_3_2) 2018; 17 e_1_3_8_26_2 e_1_3_8_13_2 Pane M (e_1_3_8_22_2) 2014; 6 Riebling P (e_1_3_8_24_2) 2017; 21 e_1_3_8_16_2 e_1_3_8_25_2 US Food and Drug Administration (e_1_3_8_10_2) 2015 Willcocks RJ (e_1_3_8_21_2) 2016; 79 Henricson E (e_1_3_8_17_2) 2013; 5 McDonald CM (e_1_3_8_11_2) 2013; 48 Haas M (e_1_3_8_14_2) 2015; 25 McDonald CM (e_1_3_8_20_2) 2018; 391 e_1_3_8_6_2 European Medicines Agency (e_1_3_8_9_2) 2015 Pane M (e_1_3_8_12_2) 2014; 9 Peltz SW (e_1_3_8_5_2) 2013; 64 Pichavant C (e_1_3_8_4_2) 2011; 19 McDonald CM (e_1_3_8_8_2) 2017; 390 Bushby K (e_1_3_8_7_2) 2014; 50 Moher D (e_1_3_8_15_2) 2009; 339 Goemans N (e_1_3_8_23_2) 2016; 11 |
| References_xml | – volume: 17 start-page: 389 issue: 5 year: 2018 ident: e_1_3_8_19_2 article-title: Evidence-based care in Duchenne muscular dystrophy publication-title: Lancet Neurol. doi: 10.1016/S1474-4422(18)30115-7 – volume: 79 start-page: 535 issue: 4 year: 2016 ident: e_1_3_8_21_2 article-title: Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large Duchenne muscular dystrophy cohort publication-title: Ann. Neurol. doi: 10.1002/ana.24599 – volume: 339 start-page: b2535 year: 2009 ident: e_1_3_8_15_2 article-title: Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement publication-title: BMJ doi: 10.1136/bmj.b2535 – volume: 17 start-page: 251 issue: 3 year: 2018 ident: e_1_3_8_3_2 article-title: Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis and neuromuscular, rehabilitation, endocrine and gastrointestinal and nutritional management publication-title: Lancet Neurol. doi: 10.1016/S1474-4422(18)30024-3 – volume: 390 start-page: 1489 issue: 10101 year: 2017 ident: e_1_3_8_8_2 article-title: Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, Phase III trial publication-title: Lancet doi: 10.1016/S0140-6736(17)31611-2 – volume: 19 start-page: 830 issue: 5 year: 2011 ident: e_1_3_8_4_2 article-title: Current status of pharmaceutical and genetic therapeutic approaches to treat DMD publication-title: Mol. Ther. doi: 10.1038/mt.2011.59 – volume: 21 start-page: e94 year: 2017 ident: e_1_3_8_24_2 article-title: Slope analysis of 6-minute walk distance as an alternative method to determine treatment effect in trials in Duchenne muscular dystrophy publication-title: Eur. J. Paediatr. Neurol. doi: 10.1016/j.ejpn.2017.04.1211 – ident: e_1_3_8_6_2 – volume: 5 year: 2013 ident: e_1_3_8_17_2 article-title: The 6-minute walk test and person-reported outcomes in boys with Duchenne muscular dystrophy and typically developing controls: longitudinal comparisons and clinically-meaningful changes over one year publication-title: PLoS Curr. – volume: 50 start-page: 477 issue: 4 year: 2014 ident: e_1_3_8_7_2 article-title: Ataluren treatment of patients with nonsense mutation dystrophinopathy publication-title: Muscle Nerve doi: 10.1002/mus.24332 – volume: 11 start-page: e0164684 issue: 10 year: 2016 ident: e_1_3_8_23_2 article-title: Individualized prediction of changes in 6-minute walk distance for patients with Duchenne muscular dystrophy publication-title: PLoS One doi: 10.1371/journal.pone.0164684 – volume: 9 start-page: e108205 issue: 10 year: 2014 ident: e_1_3_8_12_2 article-title: Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes publication-title: PLoS One doi: 10.1371/journal.pone.0108205 – year: 2015 ident: e_1_3_8_10_2 article-title: Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment publication-title: Guidance for Industry. – volume: 48 start-page: 357 issue: 3 year: 2013 ident: e_1_3_8_18_2 article-title: The 6-minute walk test and other clinical endpoints in Duchenne muscular dystrophy: reliability, concurrent validity and minimal clinically important differences from a multicenter study publication-title: Muscle Nerve doi: 10.1002/mus.23905 – volume: 25 start-page: 5 issue: 1 year: 2015 ident: e_1_3_8_14_2 article-title: European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene publication-title: Neuromuscul. Disord. doi: 10.1016/j.nmd.2014.11.011 – ident: e_1_3_8_25_2 doi: 10.2217/cer-2019-0086 – volume: 64 start-page: 407 year: 2013 ident: e_1_3_8_5_2 article-title: Ataluren as an agent for therapeutic nonsense suppression publication-title: Annu. Rev. Med. doi: 10.1146/annurev-med-120611-144851 – ident: e_1_3_8_13_2 – year: 2015 ident: e_1_3_8_9_2 article-title: Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy publication-title: Report No. EMA/CHMP/236981/2011, Corr. 1. – ident: e_1_3_8_16_2 – volume: 6 start-page: e108205 year: 2014 ident: e_1_3_8_22_2 article-title: The 6minute walk test and performance of upper limb in ambulant Duchenne muscular dystrophy boys publication-title: PLoS Curr. – ident: e_1_3_8_26_2 doi: 10.2217/cer-2019-0171 – volume: 48 start-page: 343 issue: 3 year: 2013 ident: e_1_3_8_11_2 article-title: The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study publication-title: Muscle Nerve doi: 10.1002/mus.23902 – volume: 391 start-page: 451 issue: 10119 year: 2018 ident: e_1_3_8_20_2 article-title: Long-term effects of glucocorticoids on function, quality of life and survival in patients with Duchenne muscular dystrophy: a prospective cohort study publication-title: Lancet doi: 10.1016/S0140-6736(17)32160-8 |
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| Snippet | Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).
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| SubjectTerms | 6-minute walk distance 6-minute walk test Annan medicin och hälsovetenskap ataluren Clinical trials Codon, Nonsense - genetics controlled trials Demographics Duchenne muscular dystrophy efficacy end-points Health Care Sciences & Services Humans meta-analyses Meta-analysis multicenter Muscular dystrophy Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - genetics Mutation nonsense mutation Duchenne muscular dystrophy Other Medical and Health Sciences Oxadiazoles - therapeutic use Patients randomized randomized controlled trials Randomized Controlled Trials as Topic Statistical analysis |
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