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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
Who can join
9 Months – 7 Years · All sexes
Full eligibility 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.
About the study
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)
What is being tested
- SKY-GJB2 (combination product)
Sponsor: Skylark Bio Inc. · Participants: 10 · Started: May 28, 2026
Contact the study team
Official record on ClinicalTrials.gov — NCT07627971
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.