Study of EH002 Gene Therapy to Treat Congenital Deafness Caused by Otoferlin Gene Mutations DFNB9

What this Trial Is About

Researchers are evaluating the safety, tolerability, and early effectiveness of EH002 gene therapy for treating congenital deafness caused by mutations in the OTOF gene, known as DFNB9. This study focuses on participants with severe or profound hearing loss confirmed by genetic testing. The research aims to understand how EH002 may help restore hearing by addressing the genetic cause of this type of deafness. Participants may receive one or two doses of EH002 delivered directly into one or both ears through a surgical injection into the cochlea. The study includes dose escalation groups with varying amounts of EH002 administered. Eligibility requires passing imaging tests like CT or MRI to confirm suitable ear anatomy for surgery, with follow-up visits scheduled to monitor participants closely. During the 26-week study period, researchers will track any serious side effects or dose-related toxicities and assess changes in hearing levels compared to baseline. Participants will undergo genetic testing, audiological assessments, imaging scans, and safety monitoring throughout. The study will also evaluate hearing improvements and any adverse events to better understand the therapys impact.

A Study of EH002 Gene Therapy for Otoferlin Gene Mutation-mediated Hearing Loss