Clinical Research Study Viewer

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

This study is closed to enrollment.

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