Gene therapy for otoferlin-related hearing loss
Part of Brain & nervous system, Ear, nose & throat clinical trials.
This trial tests a gene therapy that aims to restore hearing in people with a specific genetic form of hearing loss. The therapy is given as a one-time injection into the inner ear and may help you hear without needing a cochlear implant.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have hearing loss caused by changes (mutations) in the otoferlin gene, found with a genetic test.
- The hearing loss is severe or profound (little or no hearing) in both ears, as shown by a hearing test called ABR.
- Special inner-ear cells (called outer hair cells) are still working, shown by a test called DPOAE.
- You have not already had a cochlear implant in the ear that will receive the gene therapy.
- You don't have ear infections or ear problems that would make the surgery unsafe.
- You haven't taken part in another gene therapy trial before, and you agree to a long-term follow-up after this study.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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Other trials that look related to this one.
This study follows people with hearing loss caused by changes (mutations) in the otoferlin gene to better understand how hearing and hearing-related test results change over time. It may help researchers prepare for future treatments by building a clear picture of the natural course of the condition.
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.
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 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.
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.
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