Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy.

•Domagrozumab at 5, 20, and 40 mg/kg was generally safe and well tolerated.•Primary endpoint of mean change from baseline in 4SC time at week 49 was not met.•Efficacy measures did not support a significant treatment effect with domagrozumab.•Nonsignificant increase in muscle volume was observed with domagrozumab vs placebo.