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<jats:sec><jats:title>Objective:</jats:title><jats:p>To conduct a randomized trial to test the primary hypothesis that once-daily tadalafil, administered orally for 48 weeks, lessens the decline in ambulatory ability in boys with Duchenne muscular dystrophy (DMD).</jats:p></jats:sec><jats:sec><jats:title>Methods:</jats:title><jats:p>Three hundred thirty-one participants with DMD 7 to 14 years of age taking glucocorticoids were randomized to tadalafil 0.3 mg·kg<jats:sup>−1</jats:sup>·d<jats:sup>−1</jats:sup>, tadalafil 0.6 mg·kg<jats:sup>−1</jats:sup>·d<jats:sup>−1</jats:sup>, or placebo. The primary efficacy measure was 6-minute walk distance (6MWD) after 48 weeks. Secondary efficacy measures included North Star Ambulatory Assessment and timed function tests. Performance of Upper Limb (PUL) was a prespecified exploratory outcome.</jats:p></jats:sec><jats:sec><jats:title>Results:</jats:title><jats:p>Tadalafil had no effect on the primary outcome: 48-week declines in 6MWD were 51.0 ± 9.3 m with placebo, 64.7 ± 9.8 m with low-dose tadalafil (<jats:italic>p</jats:italic> = 0.307 vs placebo), and 59.1 ± 9.4 m with high-dose tadalafil (<jats:italic>p</jats:italic> = 0.538 vs placebo). Tadalafil also had no effect on secondary outcomes. In boys &gt;10 years of age, total PUL score and shoulder subscore declined less with low-dose tadalafil than placebo. Adverse events were consistent with the known safety profile of tadalafil and the DMD disease state.</jats:p></jats:sec><jats:sec><jats:title>Conclusions:</jats:title><jats:p>Tadalafil did not lessen the decline in ambulatory ability in boys with DMD. Further studies should be considered to confirm the hypothesis-generating upper limb data and to determine whether ambulatory decline can be slowed by initiation of tadalafil before 7 years of age.</jats:p></jats:sec><jats:sec><jats:title>Clinicaltrials.gov identifier:</jats:title><jats:p>NCT01865084.</jats:p></jats:sec><jats:sec><jats:title>Classification of evidence:</jats:title><jats:p>This study provides Class I evidence that tadalafil does not slow ambulatory decline in 7- to 14-year-old boys with Duchenne muscular dystrophy.</jats:p></jats:sec>

Original publication

DOI

10.1212/wnl.0000000000004570

Type

Journal article

Journal

Neurology

Publisher

Ovid Technologies (Wolters Kluwer Health)

Publication Date

24/10/2017

Volume

89

Pages

1811 - 1820