NCT07627971 · Skylark Bio Inc.
Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
(SONIX)
What this study is about
Skylark Bio is conducting a study of an experimental 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.
View original scientific description
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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.
Where
- Boston, Massachusetts
- Allentown, Pennsylvania
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 8, 2026 · Source of record for eligibility and locations