Gene therapy for GJB2-related hearing loss in young children
Part of Brain & nervous system, Ear, nose & throat clinical trials.
This study tests an experimental gene therapy (SKY-GJB2) designed to treat hearing loss caused by mutations in the GJB2 gene. The therapy is given directly to children ages 9 months to 7 years who have significant bilateral hearing loss, and researchers will monitor safety and whether hearing improves.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child is between 9 months and 7 years old at the time of treatment
- Has two copies of a GJB2 gene mutation (one from each parent) that is pathogenic or likely pathogenic
- Has bilateral sensorineural hearing loss (both ears affected due to inner ear nerve damage) confirmed by ABR testing, with hearing loss of at least 85 dB HL at one or more frequencies in the treatment ear
- Parent or guardian can provide informed consent and child can comply with study visits and procedures
- Does not have hearing loss from other causes (genetic syndromes, non-GJB2 mutations, or other sources)
- Does not have two cochlear implants already in place
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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