Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
Recruiting now · Phase 1/Phase 2
Conditions studied: GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
In brief
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)
Key facts
- Study ID
- NCT07627971
- Run by
- Skylark Bio Inc.
- People needed
- 10
- Starts
- 2026-05-28
- Expected to finish
- 2028-06-01
- Last updated by the study team
- 2026-07-15
Who can join
Age: 1 and older, up to 7. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- Massachusetts Eye and Ear Infirmary (MEEI) — Boston, Massachusetts, United States (enrolling)
- Hearts for Hearing-Oklahoma City — Oklahoma City, Oklahoma, United States (enrolling)
- Lehigh Valley Hospital-Cedar Crest — Allentown, Pennsylvania, United States (enrolling)
Full record on ClinicalTrials.gov
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