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NCT Number: NCT07627971

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

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)

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Key information

Conditions

GJB2-mediated Hearing Loss DFNB1A Deafness Deafness, Autosomal Recessive 1A Ear Diseases GJB2 Gene Mutation Hearing Disorders Hearing Loss Hearing Loss, Bilateral Hearing Loss, Sensorineural Nervous System Diseases Neurologic Manifestations Otorhinolaryngologic Diseases Pathological Conditions, Signs and Symptoms Sensation Disorders Signs and Symptoms

Age range

9 month–7 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Massachusetts Eye and Ear Infirmary (MEEI), Boston, Massachusetts, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

SKY-GJB2

Combination Product

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

Primary outcomes

  1. Safety/Frequency of Adverse Events (AEs)

    Time frame: 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))

Secondary outcomes

  1. Auditory Brainstem Response (ABR) Threshold

    Time frame: Through trial completion, approximately one year

    Changes from baseline in ABR intensity threshold (measured in decibels (dB HL))

  2. Performance of the SKY-CAT delivery device

    Time frame: One month post-op

    Looking at safety and effectiveness of the device in delivering the gene therapy.

Sponsors and collaborators

Lead sponsor

Skylark Bio Inc.

Industry

Registry information

Official study title

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

Acronym: SONIX

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
Jun 4, 2026
Registry last updated
Jul 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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