Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
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
Skylark Bio Inc.
10 participants
May 28, 2026
INTERVENTIONAL
Conditions
Summary
Skylark Bio is conducting a study of an investigational new drug called SKY-GJB2 and its delivery device, the SKY-CAT. SKY-GJB2 is a gene therapy that is being developed to treat children who have hearing loss due to changes in the GJB2 gene (also referred to as Connexin 26). The SKY-CAT is a device that delivers the gene therapy. The purpose of this study is to: Learn about the safety and tolerability (does not cause ongoing discomfort) of SKY-GJB2 Evaluate the efficacy of SKY-GJB2 (how well is works)
Eligibility
Inclusion Criteria5
- 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 Criteria4
- 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.
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Interventions
SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.
Locations(2)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07627971