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

Conditions

• DFNB1A
• GJB2 Gene Mutation
• GJB2-mediated Hearing Loss

Eligibility Criteria

Sex

ALL

Age

9 Months - 7 Years

Healthy Volunteers

Not accepted

Interventions

• SKY-GJB2 — COMBINATION_PRODUCT
  SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.

Study Details

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)

Key Dates

Start date

May 28, 2026

Status verified

Jun 2026

Primary completion

Dec 31, 2027

Completion

Jun 30, 2028

Study Design

Enrollment

10 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Older
  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.
• Experimental: Younger
  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.

Primary Outcome Measure

Safety/Frequency of Adverse Events (AEs) [ Time Frame: Through trial completion, approximately one year ]

Locations (2)

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