Edasalonexent is an oral drug that blocks the NF-kB protein, which drives muscle inflammation and fibrosis (scarring) in people with Duchenne muscular dystrophy. Blocking NF-kB could potentially reduce muscle degeneration in Duchenne.
At the moment, only trial sites in the US are enrolling patients. Sites in Canada are expected to open later this month, while sites in Europe, Australia and Israel are expected to open in the next few months.
There will be four trial sites in the UK: Evelina London Children’s Hospital, Great Ormond Street Hospital, Bristol Royal Hospital for Children and Royal Manchester Children’s Hospital. We’ll let you know when they’re ready to enrol patients.
PolarisDMD is a randomised, double-blind, placebo-controlled trial. Two boys will receive edasalonexent for every boy who receives placebo. After 12 months, all boys are expected to have the opportunity to receive edasalonexent in an open-label extension study.
To take part, boys must be aged between 4 – 7 years old and must not have taken steroids for at least 6 months. You can find the full list of eligibility criteria on clinicaltrials.gov. We’d recommend speaking to your son’s clinical team for advice on whether he might be eligible.
For more information, read Catabasis’ family newsletter. You can also register your interest by emailing the Catabasis team at DMDtrials@catabasis.com.