Clinical Research Study Viewer

MicroDystrophin Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy (DMD)

Study Details

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy (DMD) Using rAAVrh74.MCK.micro-dystrophin


The vector delivered via direct intramuscular injection of rAAVrh74.MCK.micro-dystrophin transferred into the top of one foot (or altneratively a small leg muscle) with the other side receiving saline alone.

Study Duration

Two years. Follow-up visits after 1 week, 2 weeks, 4 weeks, 6 weeks, 2 months, 3 months, 6 months, 1 year and 2 years at Nationwide Children's Hospital. 

Inclusion Criteria

  • Confirmed dystrophin mutation
  • Males (of any ethnic group) eligible
  • Age 10 years or older
  • Wheelchair dependent
  • Able to cooperate with muscle testing
  • Use of reliable method of contraception (if sexually active)

Exclusion Criteria

  • Active viral infection
  • Symptoms of cardiomyopathy
    • Dyspnea on exertion
    • Shortness of breath when supine (lying down)
    • Echocardiogram with ejection fraction < 40%
    • Pedal edema
    • Rales at base of lungs
  • Ongoing illness or need for treatment which principal investigator deems unsafe for gene transfer
  • Clinically significant, abnormal lab values
  • HIV or autoimmune disease
  • Hep A, B, or C infection
  • rAAVrh74 antibody titer >1.50 by ELISA testing

Coordinator Contact Information

Beverly Galliers

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