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RecruitingPhase 1/Phase 2

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

Skylark Bio Inc. · NCT07627971

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 10 mo–7 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1, Phase 2
Target enrollment
10
US sites recruiting
4
Study start
May 28, 2026
Record last updated
August 27, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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)

Being studied: SKY-GJB2

Conditions: GJB2-mediated Hearing Loss; GJB2 Gene Mutation; DFNB1A

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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.

Recruiting US locations

  • National Institutes of Health-Clinical Center

    Bethesda, MD 20892

    Site contact: Maame Akumatey (Study Coordinator) · 227-233-0452 · maame.akumatey@nih.gov

  • Massachusetts Eye and Ear Infirmary (MEEI)

    Boston, MA 02114

    Site contact: Michael Cheung, MSc, CCRP, Clinical Research Project Manager · 617-573-6060 · centerforclinicalresearchoperations@meei.harvard.edu

  • Hearts for Hearing-Oklahoma City

    Oklahoma City, OK 73120

    Site contact: René Gifford, PhD · 4055484300 · research@heartsforhearing.org

  • Lehigh Valley Hospital-Cedar Crest

    Allentown, PA 181103

    Site contact: Ravi N. Samy, MD, FACS · 610-402-9543 · Ravi.Samy@Jefferson.edu

All phase 1 studies in Maryland →
Open-label Study of SKY-GJB2 in Pediatric Subjects With… (NCT07627971) | Bored Studies