Abstract
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled
clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study
participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function.
Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be
randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses
during the first 12-months of the study. All participants will be eligible to receive 4 doses
of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.
