Clinical Research Study Viewer

Study to Evaluate Eteplirsen (Sarepta) in Early-Stage Duchenne Muscular Dystrophy (DMD)

Study Details

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

An Open-Label, Multi-Center Phase III Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy (DMD)

Study Duration

One year. 20 centers in the United States are participating in this trial.

Inclusion Criteria

  • Age four to six years old
  • Confirmed Duchenne Muscular Dystrophy (DMD) with out-of-frame deletion amenable to exon 51 skipping
  • Two intact upper arm muscle groups
  • Stable corticosteroid use, for at least 12 weeks prior to study
    • Or no steroid use for at least 12 weeks prior to the study and not expected to start use within the first 12 weeks of the trial

Exclusion Criteria

  • Left ventricular ejection fraction (LVEF) < 50%
  • QTcF > 450 msec
  • Drug treatment that could affect muscle function, within previous 12 weeks
    • Growth hormone
    • Anabolic steroids
  • Other experimental treatment, within previous 12 weeks
  • Systemic aminoglycoside antibiotic use, within previous 12 weeks
  • Clinically significant illness (cardiac, hepatic, renal, etc. diseases)
  • Major surgery, within 3 months prior to trial
  • Ongoing illness or need for treatment in which principal investigator deems unfit for trial

Coordinator Contact Information

Roush, Kandice 
(614) 722-2558

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000