Gene therapy for hearing loss caused by otoferlin gene changes
Part of Brain & nervous system, Ear, nose & throat clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have at least two known changes (mutations) in the otoferlin gene
- You have very severe hearing loss in both ears (confirmed with a test called ABR)
- Your doctor can still measure certain inner-ear responses on DPOAE testing
- You are able and willing to follow all study visits and do long-term follow-up after the trial
- You can have the required inner-ear procedure safely (no conditions that make surgery/anesthesia gene therapy unsafe)
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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