The study intends to investigate the safety and efficacy of eteplirsen in DMD patients over 24 weeks of dosing.
In the trial, the patients will be given once weekly intravenous infusions of either 50mg/kg of eteplirsen, 30mg/kg of eteplirsen or placebo, and will be evaluated on a number of safety and efficacy endpoints.
AVI president and CEO Chris Garabedian said in this Phase 2 study they will evaluate eteplirsen at higher doses and over a longer duration of treatment, which will help them understand the potential disease-modifying effects and safety of eteplirsen as chronically-administered therapy.
"We expect data from this study around the end of the second quarter of 2012, which will guide our design for a pivotal study," Garabedian said.