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

Purpose

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)

Conditions

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

Eligibility

Eligible Ages
Between 9 Months and 7 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
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.
  • Combination Product: SKY-GJB2
    SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery 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.
  • Combination Product: SKY-GJB2
    SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.

Recruiting Locations

Massachusetts Eye and Ear Infirmary (MEEI)
Boston, Massachusetts 02114
Contact:
Michael Cheung, MSc, CCRP, Clinical Research Project Manager
617-573-6060
mcheung0@meei.harvard.edu

Lehigh Valley Hospital-Cedar Crest
Allentown, Pennsylvania 181103
Contact:
Ravi N. Samy, MD, FACS
610-402-9543
Ravi.Samy@Jefferson.edu

More Details

Status
Recruiting
Sponsor
Skylark Bio Inc.

Study Contact