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

ABSTRACT:

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

SUBMITTER: Victor RG 

PROVIDER: S-EPMC5664308 | biostudies-literature | 2017 Oct

REPOSITORIES: biostudies-literature