Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss

Plain-language summary

This study evaluates a gene therapy called SKY-GJB2 in children with bilateral sensorineural hearing loss caused by two damaging (biallelic) variants in the GJB2 gene. It enrolls children aged 9 months to 7 years with a confirmed GJB2 mutation and severe hearing loss. It is a Phase 1/Phase 2 study and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Recruiting

Sponsor

Skylark Bio Inc.

Intervention

SKY-GJB2

Start date

5/28/2026

Estimated completion

6/1/2028

Countries

United States

Related genes

Locations

• Boston, United States
• Oklahoma City, United States
• Allentown, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.