PTC Therapeutics has completed a Phase 3 clinical trial of ataluren (formerly referred to as PTC124®) in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). The trial is referred to as the Ataluren Confirmatory Trial in DMD, or ACT DMD.
The primary objective of this confirmatory, global, randomized, double-blind, placebo-controlled trial was to assess the efficacy and safety of ataluren 10, 10, 20 mg/kg tid (given three times daily - morning, midday, evening) in patients with nmDMD. Efficacy was determined primarily by measuring changes in walking distance based on a 6-minute walk test (6MWT). The study enrolled 228 patients at approximately 50 investigational sites globally. Each participant was randomly assigned to receive either ataluren (10-, 10-, 20-mg/kg tid) or placebo (a substance that looks and tastes like ataluren but does not actually contain the drug), for 48 weeks.
Additional details about this ataluren clinical trial can be found at www.clinicaltrials.gov or by contacting PTC Therapeutics Patient and Professional Advocacy at (866) 282-5873 or (908) 912-9256 or PatientInfo@ptcbio.com. ACT DMD data was announced in October 2015 and can be found in the following press release.