Plain-English translation of NCT05821959 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing a new gene therapy called AAVAnc80-hOTOF, which is injected directly into the inner ear during a surgical procedure. The treatment is designed to replace a faulty gene (otoferlin) that causes severe hearing loss from birth. Researchers want to see if this one-time treatment can help restore or improve hearing in people with this rare genetic form of deafness.
People born with mutations in the otoferlin gene experience profound hearing loss that cannot be helped by traditional hearing aids. There are currently no approved treatments for this genetic cause of deafness, so this trial is exploring whether delivering a healthy copy of the gene directly to the inner ear could offer a new option.
You likely qualify if…
You likely don't qualify if…
If you qualify, you would undergo a one-time surgical procedure in which the treatment is injected directly into your inner ear under anesthesia. You would then return for follow-up visits to have your hearing tested and monitored for safety and effectiveness. After the main trial ends, you would be asked to enroll in a separate long-term follow-up study to continue monitoring how well the treatment works over time.
AI-generated summary from trial data · Jun 16, 2026 · Not medical advice
United States