Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: SKY-GJB2.
- Who it may be relevant to
- Registry conditions: GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A. Basic parameters: 9 months — 7 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
Overview
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)
Interventions
- Combination product SKY-GJB2
SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.
Primary outcome measures
- Safety/Frequency of Adverse Events (AEs) [Time frame: Through trial completion, approximately one year]
Secondary outcome measures (2)
- Auditory Brainstem Response (ABR) Threshold [Time frame: Through trial completion, approximately one year]
- Performance of the SKY-CAT delivery device [Time frame: One month post-op]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- Massachusetts Eye and Ear Infirmary (MEEI) — Boston
- Hearts for Hearing-Oklahoma City — Oklahoma City
- Lehigh Valley Hospital-Cedar Crest — Allentown
Identifiers
NCT: NCT07627971 · SKY-GJB2-001