PTC Therapeutics publishes results of ACT DMD trial study ataluren
PTC Therapeutics announced the publication of the results of the ACT DMD trial study ataluren, the only treatment for the underlying cause of nonsense mutation Duchene muscular dystrophy, in the current issue of The Lancet. ACT DMD is a multi-center, randomized, double-blind, Phase 3 clinical trial involving 228 patients in 53 sites across 18 countries. Patients with nmDMD were randomized to receive either ataluren 40mg/kg per day or placebo over 48 weeks. The primary endpoint was change from baseline in the six-minute walk test. Treatment effects are more likely to be observed in patients in the transition stage of disease. Analyses of data from pre-specified subgroups, including the pre-specified subgroup of patients with baseline six-minute walk distance of 300 - 400 meters, were also completed. Key secondary outcome measures were timed-function tests, including time to run or walk 10 meters and the time to ascend or descend four stairs. Exploratory efficacy endpoints were change in physical function as assessed by change in the North Star Ambulatory Assessment, parent-reported health related quality of life, and activities of daily living.