Clinical Research Study Viewer

Study to Evaluate Eteplirsen (Sarepta) in Older Children with Duchenne Muscular Dystrophy (DMD)

Study Details

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

An Open-Label, Multi-Center 48-Week Phase III Study with a Concurrent Untreated Control Arm to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Duchenne Muscular Dystrophy (DMD)

Study Duration

One year. 39 international sites are participating in this trial.

Inclusion Criteria

  • Age 7 to 16 years old
  • Treated Group
    • Confirmed Duchenne Muscular Dystrophy (DMD)
    • Out-of-frame deletion
    • Amenable to exon 51 skipping
  • Untreated Group
    • Confirmed Duchenne Muscular Dystrophy (DMD)
    • Out-of-frame deletion
    • Only amenable to other exon skipping (not exon 51)
  • Two intact upper arm muscle groups
  • Stable corticosteroid use, for at least 24 weeks prior to study
    • ACE inhibitors, ARBs, beta blockers and potassium are permitted
  • Ambulatory > 300m on 6MWT (2-day average, within 15%)
  • Cardiac and pulmonary function unlikely to deteriorate over the study period
    • Forced vital capacity (FVC) % of predicted > 50%
    • No nocturnal ventilation
  • Use of reliable method of contraception (if sexually active)

Exclusion Criteria

  • Drug treatment that could affect muscle function, within previous 12 weeks
    • Growth hormone
    • Anabolic steroids
  • Previous experimental treatment
    • SMT C1100/BMN 195
    • Drisapersen (PRO051), within previous 6 months
    • Shock Training System (STS), during study
  • Systemic aminoglycoside antibiotic use, within previous 12 weeks
  • Clinically significant illness (cardiac, hepatic, renal, etc. diseases)
  • Major surgery, within 3 months prior to trial

Coordinator Contact Information

Roush, Kandice 
(614) 722-2558

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