One-time gene therapy aims to restore hearing in kids with genetic deafness
NCT ID NCT07627971
First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 times
Summary
This early-stage trial tests a new gene therapy called SKY-GJB2 for children aged 9 months to 7 years with hearing loss caused by changes in the GJB2 gene. The therapy is given as a single injection into the inner ear using a special delivery device. The study will enroll 10 children to check if the treatment is safe and whether it can improve hearing.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
SKY-GJB2 gene therapy
What this could lead to
If successful, this could point toward a one-time gene therapy treatment that restores hearing in children with GJB2-related hearing loss.
What could go wrong
This is an early phase 1/2 trial with only 10 children, so it is primarily testing safety. It may not improve hearing, and there are risks from the gene therapy and the surgical delivery procedure.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Lehigh Valley Hospital-Cedar Crest
RECRUITINGAllentown, Pennsylvania, 181103, United States
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Massachusetts Eye and Ear Infirmary (MEEI)
RECRUITINGBoston, Massachusetts, 02114, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••