Long-term follow-up of gene therapy for otoferlin hearing loss
Part of Brain & nervous system, Ear, nose & throat clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have received the gene therapy injection in the AK-OTOF-101 clinical trial.
- You or your legal guardian must agree to follow study rules and sign a consent form.
- You must not have any other health problems that would keep you from finishing the study follow-ups.
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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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 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 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 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 study builds a patient registry and tracks the natural history of hearing loss in people whose genetic test shows changes in the otoferlin (OTOF) gene. It may help researchers understand how hearing symptoms progress over time and improve future treatments.
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