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

June 25, 2026 updated by: Skylark Bio Inc.

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 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)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Older
Four subjects aged 2 to 7 years old (inclusive) to receive an intracochlear infusion of SKY-GJB2 in the study ear using a sterile, one-time use investigational medical device.
Combination product: SKY-GJB2
SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.
Experimental: Younger
Six subjects aged 9 months to less than 2 years old to receive an intracochlear infusion of SKY-GJB2 in the study ear using a sterile, one-time use investigational medical device.
Combination product: SKY-GJB2
SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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))
Through trial completion, approximately one year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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))
Through trial completion, approximately one year
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.
One month post-op

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Skylark Bio Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 28, 2026

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

June 1, 2028

Study Registration Dates

First Submitted

May 28, 2026

First Submitted That Met QC Criteria

June 2, 2026

First Posted (Actual)

June 4, 2026

Study Record Updates

Last Update Posted (Actual)

June 30, 2026

Last Update Submitted That Met QC Criteria

June 25, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SKY-GJB2-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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