SKY-GJB2: SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.
Study 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)
Eligibility
Sex
ALL
Min age
9 Months
Max age
7 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Safety/Frequency of Adverse Events (AEs) — 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))
Trial sites (4)
Facility
City
Region
Status
National Institutes of Health-Clinical Center
Bethesda
Maryland
Recruiting
Massachusetts Eye and Ear Infirmary (MEEI)
Boston
Massachusetts
Recruiting
Hearts for Hearing-Oklahoma City
Oklahoma City
Oklahoma
Recruiting
Lehigh Valley Hospital-Cedar Crest
Allentown
Pennsylvania
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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