Additional Relevant Details In the first portion of the MoveDMD trial (Part A), 17 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations received edasalonexent. The boys were steroid naive or had not used steroids for at least six months prior to the trial. This portion of the trial was conducted at three sites in the U.S., and assessed the safety, tolerability and pharmacokinetics of edasalonexent in patients at three dosing levels (33 mg/kg/day, 67 mg/kg/day and 100 mg/kg/day) during seven days of dosing. Phase 2 of the MoveDMD trial (Part B) is a randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of edasalonexent in DMD over a 12-week period at 5 clinical trial sites in the U.S. at two dosing levels, 67 mg/kg/day and 100 mg/kg/day. The boys that participated in the first part of the MoveDMD trial that remain eligible are asked to participate in Phase 2 and additional participants are expected to be enrolled for a total of approximately 30 boys. The open-label extension (Part C) includes dosing with edasalonexent for 36 weeks beyond the 12-week placebo-controlled portion of the trial and will evaluate longer term safety and efficacy with the same clinical end points. We are currently identifying additional patients who are interested in participating in the Phase 2 trial. Entry criteria are similar to those in the first portion of the trial. The Parent Project Muscular Dystrophy and the Muscular Dystrophy Association are providing funding to support participant travel for the MoveDMD trial.
More information about the MoveDMD trial can be found on the clinical trials page of the Catabasis website and on ClinicalTrials.gov under trial identifier NCT02439216.