A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

Ronald G Victor, H Lee Sweeney, Richard Finkel, Craig M McDonald, Barry Byrne, Michelle Eagle, Nathalie Goemans, Krista Vandenborne, Alberto L Dubrovsky, Haluk Topaloglu, M Carrie Miceli, Pat Furlong, John Landry, Robert Elashoff, David Cox, Tadalafil DMD Study Group, Claudio Bruno

Research output: Contribution to journalArticlepeer-review


OBJECTIVE: 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).

METHODS: Three hundred thirty-one participants with DMD 7 to 14 years of age taking glucocorticoids were randomized to tadalafil 0.3 mg·kg-1·d-1, tadalafil 0.6 mg·kg-1·d-1, 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.

RESULTS: 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 (p = 0.307 vs placebo), and 59.1 ± 9.4 m with high-dose tadalafil (p = 0.538 vs placebo). Tadalafil also had no effect on secondary outcomes. In boys >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.

CONCLUSIONS: 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.


CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that tadalafil does not slow ambulatory decline in 7- to 14-year-old boys with Duchenne muscular dystrophy.

Original languageEnglish
Pages (from-to)1811-1820
Number of pages10
Issue number17
Publication statusPublished - Oct 24 2017


  • Adolescent
  • Area Under Curve
  • Child
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Follow-Up Studies
  • Glucocorticoids
  • Heart Rate
  • Humans
  • International Cooperation
  • Male
  • Muscular Dystrophy, Duchenne
  • Quality of Life
  • Respiratory Function Tests
  • Tadalafil
  • Treatment Outcome
  • Vasodilator Agents
  • Ventricular Function, Left
  • Walking
  • Clinical Trial, Phase III
  • Journal Article
  • Multicenter Study
  • Randomized Controlled Trial


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