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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

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.