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RECRUITING Phase 1 / Phase 2 ClinicalTrials.gov

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

Sponsor: Skylark Bio Inc. • ClinicalTrials.gov ID: NCT07627971

Registry updated: 2026-08-27
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Description

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)

Interventions

  • SKY-GJB2 (Combination product)
    SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.

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.

Conditions

GJB2-mediated Hearing Loss GJB2 Gene Mutation DFNB1A

Locations

  • National Institutes of Health-Clinical Center — Bethesda, Maryland, United States
  • Massachusetts Eye and Ear Infirmary (MEEI) — Boston, Massachusetts, United States
  • Hearts for Hearing-Oklahoma City — Oklahoma City, Oklahoma, United States
  • Lehigh Valley Hospital-Cedar Crest — Allentown, Pennsylvania, United States
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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record