A Phase 1/2 interventional study of SKY-GJB2 in GJB2-mediated Hearing Loss, GJB2 Gene Mutation and DFNB1A, sponsored by Skylark Bio Inc.. Recruiting at 4 sites in United States. Open to participants aged 9 Months to 7 Years. Per ClinicalTrials.gov, last updated 2026-08-27.
Sponsored by Skylark Bio Inc. · Phase 1/2, Interventional, and Treatment
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)
55 studies on the registry are indexed under Hearing Loss, Bilateral; 17 are open to participants now.
This study's planned enrollment of 10 is below the median of 30 across 42 interventional studies indexed under Hearing Loss, Bilateral.
Browse Hearing Loss, Bilateral studies →This is the only study on the registry with Skylark Bio Inc. as lead sponsor.
Counted across the registry records on this site, refreshed daily.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Four subjects aged 2 to 7 years old (inclusive) to receive an intracochlear infusion of SKY-GJB2 in the study ear using a sterile, one-time use investigational medical device.
Combination Product: SKY-GJB2
Six subjects aged 9 months to less than 2 years old to receive an intracochlear infusion of SKY-GJB2 in the study ear using a sterile, one-time use investigational medical device.
Combination Product: SKY-GJB2
SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.
Safety/Frequency of Adverse Events (AEs)
AEs as it relates to the the gene therapy (SKY-GJB2) and/or to the administration procedure (including the delivery device system (SKY-CAT))
Time frame: Through trial completion, approximately one year
Auditory Brainstem Response (ABR) Threshold
Changes from baseline in ABR intensity threshold (measured in decibels (dB HL))
Time frame: Through trial completion, approximately one year
Performance of the SKY-CAT delivery device
Looking at safety and effectiveness of the device in delivering the gene therapy.
Time frame: One month post-op
Plan to share: Undecided
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
No contact was published for this record. The registry link below has the sponsor’s details.
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