NCT07627971

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

Recruiting
PHASE1Ages 9–7InterventionalTreatment
Skylark Bio Inc.
~10 participants
Updated 2026-07-15 on ClinicalTrials.gov
What's tested:SKY-GJB2

At a glance

Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety/Frequency of Adverse Events (AEs)
Measured over Through trial completion, approximately one year
GJB2-mediated Hearing Loss
GJB2 Gene Mutation
DFNB1A
3 sites across 3 states
Massachusetts1
Oklahoma1
Pennsylvania1
Michael Cheung, MSc, CCRP, Clinical Research Project Manager
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Eligibility criteria

Inclusion

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

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.
  • Safety/Frequency of Adverse Events (AEs)Through trial completion, approximately one year

    AEs as it relates to the the gene therapy (SKY-GJB2) and/or to the administration procedure (including the delivery device system (SKY-CAT))