Capricor Therapeutics has initiated the HOPE-2 clinical trial of CAP-1002 to treat patients with advanced stages of Duchenne muscular dystrophy, at UC Davis Medical Center in the US.
The randomised, placebo-controlled Phase II trial is designed to evaluate the safety and efficacy of CAP-1002 in up to 84 boys and young men.
It is expected to be conducted in around 12 to 15 investigative sites across the US.
Under the trial, patients will be randomised to receive either placebo or CAP-1002, which will be delivered intravenously every three months for a total of four administrations.
They will be followed for a one-year period following randomisation. An open-label extension is also expected to be carried out if trial results show an appropriate risk / benefit profile of CAP-1002.
HOPE-2 clinical trial national principal investigator Craig McDonald said: “The HOPE-2 trial will study whether CAP-1002 can maintain or improve cardiac and skeletal muscle function.
“Because many of the participants are non-ambulatory, the study will focus primarily on the impact on arm mobility.”
Duchenne muscular dystrophy is a genetic disorder that causes muscle degeneration and leads to death, usually before the age of 30 as a result of heart failure.
Capricor Therapeutics president and CEO Linda Marbán said: “While gene and other therapies have the potential to restore dystrophin expression and sustain muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects.
“CAP-1002 may work synergistically with the emerging disease-modifying therapies to control those additional pathological aspects of Duchenne muscular dystrophy because CAP-1002’s primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.”
CAP-1002 features allogeneic cardiosphere-derived cells (CDCs), a unique population of cells that contains cardiac progenitor cells.