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

RecruitingPhase 1, Phase 2Ages 9 Months - 7 YearsAllSkylark Bio Inc.
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In plain words

What the trial is, and whether it could suit you

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)

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 โ‰ฅ85 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.
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What it tests

  • SKY-GJB2
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Where

4 sites
National Institutes of Health-Clinical Center
Bethesda, United States
Massachusetts Eye and Ear Infirmary (MEEI)
Boston, United States
Hearts for Hearing-Oklahoma City
Oklahoma City, United States
Lehigh Valley Hospital-Cedar Crest
Allentown, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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