Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss

NCTID	NCT07627971 (View at clinicaltrials.gov)
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. 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) (Show More)
Development Status 🔄	Active
Indication	GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
Disease Ontology Term 🔄	DOID:0110475
Drug Product Name 🔄	SKY-GJB2
Sponsor	Skylark Bio Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	10 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant 🔄	GJB2
Therapeutic Modality 🔄	Gene transfer
Therapy Route 🔄	in vivo
Mechanism of Action 🔄	Functional gene replacement
Route of Administration 🔄	Intracochlear
Drug Product Type 🔄	Viral vector
Gene Delivery System Type 🔄	Viral transduction
Vector Type 🔄	Adeno-associated virus
Viral Vector Subtype	undisclosed
Dose 1 🔄	Undisclosed

Eligible Age	9 Months - 7 Years
Standard Ages	Child
Sexes Eligible for Study	ALL
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. View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites 🔄	3
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	First patient was dosed in Phase 1/2 clinical trial, preliminary data is expected by year-end 2026