Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial.

What was studied

This phase 3 trial, HOPE-3, tested deramiocel in advanced Duchenne muscular dystrophy (DMD). Deramiocel is a heart-derived cell therapy. The trial was randomized, double-blind and placebo-controlled. Participants were aged 10 years or older and had DMD. The drug was given by vein every 3 months. Of 139 participants screened, 106 were randomized. Of these, 54 got deramiocel and 52 got placebo. The main outcome was change in the PUL2.0 upper limb score. It was measured at 12 months.

What they found

The main outcome improved with deramiocel against placebo. At 12 months, the mean change favored deramiocel by 4·55. The range was 0·47-8·63. The p value was 0·029. Safety was similar to placebo. Skeletal and cardiac function were also checked at 12 months.

What it means for patients

The authors say deramiocel slows disease progression safely in advanced DMD. They say it preserves skeletal muscle function. They say its quarterly outpatient use is simple.

Why it is not certain yet

The abstract gives only one main result. The range of 0·47-8·63 was wide. The abstract reports no heart results. The trial followed patients for 12 months. Capricor Therapeutics funded it.

Source

Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial.

We restate facts from the source record in our own words. The original wording belongs to its publisher.