Sky-GJB2 gene therapy for GJB2-related hearing loss in young children
Official title: A Phase 1/2, Open-label, Single Dose Study of SKY-GJB2 Given as a Single Unilateral Intracochlear Administration in Pediatric Subjects With GJB2-mediated Hearing Loss
Run by Skylark Bio Inc. · NCT07627971 · Data updated 2026-11-12
This trial tests SKY-GJB2 gene therapy delivered by SKY-CAT in children aged 9 months to 7 years with GJB2-related bilateral sensorineural hearing loss.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 9 Months to 7 Years |
| Sex | All |
| Enrollment | 10 (estimated) |
| Sponsor | Skylark Bio Inc. Industry-sponsored |
| Locations | 4 sites (Bethesda, Maryland and 3 other locations) |
| Status | Recruiting |
| Started | May 28, 2026 |
| Main results expected | December 2027 |
| Study ends | June 2028 |
| Registry record updated | August 27, 2026 |
| Trial ID | NCT07627971 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are children aged 9 months to 7 years with biallelic GJB2 mutations and bilateral sensorineural hearing loss of ≥85 dB HL in the treated ear.
✕Probably not a fit
Patients without GJB2-related hearing loss or those without adequate ABR-confirmed hearing loss may not benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This early-phase trial in children will evaluate the safety and tolerability of SKY-GJB2, a gene therapy designed to treat hearing loss caused by GJB2 mutations, delivered via a dedicated delivery device SKY-CAT. The study combines Phase 1 and Phase 2 elements to gather initial safety data and preliminary signals of effectiveness. Eligible participants have confirmed biallelic GJB2 variants and significant bilateral hearing loss, and will be closely monitored for adverse events and changes in hearing function. The study is conducted at three centers in the United States under Skylark Bio Inc. leadership.
Why it matters
Potential benefit: If successful, SKY-GJB2 could offer a targeted treatment to improve or stabilize hearing in children with GJB2-related deafness.
How similar studies have gone: Gene-therapy approaches for monogenic deafness are being explored, with limited but encouraging early signals in related conditions.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male or female aged 9 months to 7 years at the time of gene therapy administration * Subjects must have at least two (biallelic) pathogenic or likely pathogenic variants in the GJB2 gene * Bilateral sensorineural hearing loss as assessed by ABR. Hearing loss at ≥85 dB HL for at least one of the frequencies (500-4000 Hz) in the study treatment ear. * Subject's parent(s)/guardian(s) provide informed consent before the initiation of study-related procedures. * Subject is able and willing to comply (or provide assent if old enough) will all study requirements. Exclusion Criteria: * Subject has non-GJB2 mediated hearing loss including genetic, syndromic, or non-syndromic hearing loss that is not associated with GJB2 mutations. * Subject has autosomal dominant nonsyndromic hearing loss due to GJB2 mutation. * No response on ABR testing. * Bilateral Cochlear Implants.
Where it's running
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National Institutes of Health-Clinical Center
Bethesda, Maryland, United States Recruiting -
Massachusetts Eye and Ear Infirmary (MEEI)
Boston, Massachusetts, United States Recruiting -
Hearts for Hearing-Oklahoma City
Oklahoma City, Oklahoma, United States Recruiting -
Lehigh Valley Hospital-Cedar Crest
Allentown, Pennsylvania, United States Recruiting
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Tracking hearing loss in children with specific genetic mutations
This study is tracking children with hearing loss caused by specific genetic mutations to see how their condition changes over time and how it affects their quality of life.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.