Clinical Study

Duchenne Muscular Dystrophy affects 20,000 boys and men in the United States and 300,000 world wide. Currently, no cure exists.

Clinical Trial Design

10 patient, 3 arm dose escalation study based on patient weight

Primary endpoints: safety
  • First 6 months active follow-up / 18-months passive follow-up
Secondary endpoints: efficacy related, mean change from baseline:
  • NorthStar Ambulatory Assessment (NSAA), range of motion (ROM), performance of upper limb (PUL), 6MWD at month 1, 3, 6, 12, 18 and 24
  • Muscle strength by myometry and 5-grade Lovett scale at month 1, 3, 6, 12, 18 and 24. Cardiac muscle evaluation by ECHO
  • Laboratory parameters: hematological, biochemical including: CK, CKMB, liver function panel, and other standard tests