Recruiting
Phase 1
Phase 2

SKY-GJB2

Sponsor:

Skylark Bio Inc.

Code:

NCT07627971

Conditions

GJB2-mediated Hearing Loss

GJB2 Gene Mutation

DFNB1A

Eligibility Criteria

Sex: All

Age: 0 - 7

Healthy Volunteers: Not accepted

Interventions

SKY-GJB2

Study Details

Brief summary:

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)

Conditions

GJB2-mediated Hearing Loss

GJB2 Gene Mutation

DFNB1A

Study ID

NCT07627971

Start date

May 28, 2026

Status verified date

Aug, 2026

Completion date

Jun, 2028

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 7

Healthy Volunteers: Not accepted

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 Design

Enrollment

10 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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.

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.

Interventions

SKY-GJB2

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

Primary outcome measure

  • Safety/Frequency of Adverse Events (AEs) [ Time Frame: Through trial completion, approximately one year ]

Central Contacts and Locations

Locations

National Institutes of Health-Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

Maame Akumatey (Study Coordinator)

227-233-0452maame.akumatey@nih.gov

Ronna Hertzano, MD, PhD

301-496-3805ronna.hertzano@nih.gov

Massachusetts Eye and Ear Infirmary (MEEI)

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Michael Cheung, MSc, CCRP, Clinical Research Project Manager

617-573-6060centerforclinicalresearchoperations@meei.harvard.edu

Hearts for Hearing-Oklahoma City

Recruiting

Oklahoma City, Oklahoma, United States, 73120

Contacts

René Gifford, PhD

4055484300research@heartsforhearing.org

Lehigh Valley Hospital-Cedar Crest

Recruiting

Allentown, Pennsylvania, United States, 181103

Contacts

Ravi N. Samy, MD, FACS

610-402-9543Ravi.Samy@Jefferson.edu

Danielle Koehler, RN (Study Coordinator)

Danielle.Koehler@Jefferson.edu

More Information

Sponsor

Skylark Bio Inc.

Last update posted

Aug 27, 2026

Last verified

Aug, 2026

Keywords

  • GJB2
  • Hearing Loss, Bilateral
  • Hearing Loss
  • Hearing Loss, Sensorineural
  • Deafness, Autosomal Recessive 1A (DFNB1A)
  • Connexin 26

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Skylark Bio Inc. on 2026-08-27.