Clinical Research Study Viewer

Follistatin Gene Therapy Study for Duchenne Muscular Dystrophy (DMD)

Study Details

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

Intramuscular Gene Transfer of Follistatin for Duchenne Muscular Dystrophy (DMD), Phase I/IIa Study

Study Duration

Two years. Follow-up visits at 1 week, 2 weeks, 4 weeks, 6 weeks, 2 months, 3 months, 6 months, 9 months, 12 months, 18 months and 24 months at Nationwide Children's Hospital.

Inclusion Criteria

  • Males (any ethnic group)
  • Age 7 or older
  • Confirmed diagnosis of Duchenne muscular dystrophy (DMD)
  • Impaired muscle function
    • Difficulty with stairs
    • Gower's
    • Weakness of extremities
  • Able to cooperate with muscle testing
  • Steroid use prior to (3 months) and following the procedure, as part of standard of care
  • Use of reliable method of contraception (if sexually active)

Exclusion Criteria

  • Active viral infection
  • No weakness or loss of function (despite DMD diagnosis)
  • 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
  • Clinically significant, abnormal lab values
  • Ongoing illness or treatment in which PI deems unsafe for gene transfer
  • HIV infection or autoimmune disease
  • Hep A, B or C infection
  • AAV1 antibody titer > 1:50 by ELISA testing

Coordinator Contact Information

McColly, Markus

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