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