NEW YORK – Addenbrooke's Hospital, a National Health Service hospital in Cambridge, England, this week announced it is enrolling patients with a rare form of hearing loss into a clinical trial of Regeneron Pharmaceuticals' investigational gene therapy DB-OTO.
Investigators in Spain, the UK, and the US are enrolling up to 18 pediatric patients in the global Phase I/II study, called CHORD, to evaluate whether the gene therapy can restore hearing in children with congenital hearing loss caused by mutations in the OTOF gene. DB-OTO, administered via intracochlear injection, aims to deliver a functional copy of OTOF.
Patients will receive a starting dose of DB-OTO in one ear, and after evaluating safety, a higher dose of the gene therapy in the same ear followed by administration of the gene therapy in both ears. Researchers will follow patients for five years. If the gene therapy is not effective six months after treatment, families will be able to receive a cochlear implant for patients in the trial.
"Although experimental, the therapy could also potentially result in better quality hearing compared to cochlear implants," the current standard of care for OTOF-related hearing loss, Manohar Bance, an ear surgeon at Cambridge University Hospitals NHS Foundation Trust and chief investigator for the trial, said in a statement.
DB-OTO was codeveloped by Regeneron and Decibel Therapeutics, a biotech firm that Regeneron acquired in September.