Ataluren preserves muscle burst activity in boys with Duchenne muscular dystrophy
Click Here to Manage Email Alerts
Key takeaways:
- Treatment with ataluren improved mean change from baseline to week 72 in all timed function tests.
- Ataluren was assessed in groups of patients with 6-minute walk distances of at least 150 m and 300 m to 400 m.
Ataluren preserved muscle burst activity across a range of ambulatory tests compared with placebo in boys with nonsense mutation Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.
Seeking to evaluate the effects of ataluren on muscle burst activity in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD), Craig McDonald, MD, professor of pediatrics and physical medicine & rehabilitation at University of California Davis Health, and colleagues conducted an international, phase 3, randomized, double-blind, placebo-controlled 72-week trial.
Researchers included 359 boys, aged 5 years and older, with nmDMD who were prescribed corticosteroids and had a 6-minute walk distance (6MWD) of at least 150 m. Participants were randomized on a 1:1 basis to receive ataluren (n = 183) or placebo (n = 176). A subgroup in the trial included boys with 300 m to 400 m 6MWD.
Secondary endpoints for the study included change from baseline to week 72 in timed function test results, including 10 m walk/run and four stair ascent and descent.
Researchers found that treatment with ataluren significantly reduced mean change from baseline in time to complete the 10 m walk/run by 20% (0.78 s) in the main study group and by 30% (1.29 s) in the 300 m to 400 m 6MWD subgroup.
They also reported significant differences in mean change from baseline in time to complete the four-stair ascent in the main group (1.06 s) and subgroup (2.29 s), with a relative change of 18% and 30%, respectively, compared with placebo. Similar results were observed for performance time in the four-stair descent for those who received ataluren compared with placebo (main study group: 0.29 s; 300–400 m 6MWD subgroup: 0.97 s).
“These results demonstrate that ataluren preserves performance in [timed function tests], and therefore muscle burst activity, in patients with nmDMD,” McDonald and colleagues wrote.