Gene therapy for children with inherited deafness
Part of Ear, nose & throat, Genetic & congenital clinical trials.
This trial tests an injection called EHT102 for children who have severe or profound hearing loss due to mutations in the Otoferlin gene. The goal is to see if the treatment can restore or improve hearing.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 1 and 17 years old.
- Genetic testing must show you have two Otoferlin gene mutations causing DFNB9 deafness.
- Hearing loss must be severe or profound (65 dB or higher by ABR test).
- You must be a candidate for ear surgery, with no inner ear or nerve problems.
- You must have present DPOAE test results (showing outer hair cell function).
- You cannot have a cochlear implant or certain other ear conditions.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study is developing a system to evaluate and improve hearing for people with congenital hearing loss. It compares participants who have hearing loss (and have used hearing aids, cochlear implants, or gene therapy) with healthy controls, to better understand and treat hearing challenges.
This trial tests a one-time gene therapy delivered into the ear to improve hearing in people with certain OTOF gene changes. It studies safety and how well the treatment can work, especially in children and infants, as doctors monitor hearing tests for up to 48 weeks.
This trial tests a gene therapy called EH002 for people with severe or profound hearing loss due to changes in the OTOF gene. The treatment aims to help the ear hear better by replacing the faulty gene.
This early-stage study tests a one-time gene therapy to help patients with profound hearing loss linked to otoferlin gene mutations. It may help hearing by delivering a working version of the otoferlin gene to the inner ear, followed by long-term monitoring.
This study follows children with hearing loss caused by changes in specific genes (GJB2 or OTOF). It helps researchers understand how hearing develops over time and how children may do with or without cochlear implants.
This study follows up with people who received a gene therapy (AAVAnc80-hOTOF) in a previous clinical trial. It checks how well the treatment works over time and how safe it is.
Hear when a new Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.