Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
Key facts
- Who runs it
- Skylark Bio Inc.
- Where
- Allentown, Bethesda, Boston, and Oklahoma City, United StatesSee the 4 sites
- Trial code
- Status
- Recruiting
- Phase
- Phase 1/2
- People taking part
- 10 people, planned
- Started
- 28 May 2026
- Expected to end
- Jun 2028
- Type of study
- Interventional, 2 groups
- Treatments tested
- SKY-GJB2
- Record updated
- 27 August 2026
Who can take part
- Age
- 9 months to 7 years
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
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Who can take part
- 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
Who cannot take part
- Bilateral Cochlear Implants
- 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
Where in United States
- Lehigh Valley Hospital-Cedar CrestAllentown, United StatesContact: Ravi N. Samy, MD, FACSTaking people
- National Institutes of Health Clinical CenterBethesda, United StatesContact: Maame Akumatey (Study Coordinator)Taking people
- Massachusetts Eye and Ear Infirmary(MEEI)Boston, United StatesContact: Michael Cheung, MSc, CCRP, Clinical Research Project ManagerTaking people
- Hearts for Hearing-Oklahoma CityOklahoma City, United StatesContact: René Gifford, PhDTaking people
About the trial
What the trial wants to find out
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.
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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)
The 2 groups in the trial
- OlderTreatment being tested
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.
Treatment: SKY-GJB2
- YoungerTreatment being tested
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.
Treatment: SKY-GJB2
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- A Phase 1/2, Open-label, Single Dose Study of SKY-GJB2 Given as a Single Unilateral Intracochlear Administration in Pediatric Subjects With GJB2-mediated Hearing Loss
- Record updated
- 27 August 2026