NCT07627971 Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
| NCT ID | NCT07627971 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Skylark Bio Inc. |
| Condition | GJB2-mediated Hearing Loss |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2026-05-28 |
| Primary Completion | 2027-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 10 participants in total. It began in 2026-05-28 with a primary completion date of 2027-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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)
Eligibility Criteria
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.
Frequently Asked Questions
Who can join the NCT07627971 clinical trial?
This trial is open to participants of all sexes, aged 9 Months or older, up to 7 Years, studying GJB2-mediated Hearing Loss. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07627971 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07627971 currently recruiting?
Yes, NCT07627971 is actively recruiting participants. Visit ClinicalTrials.gov or contact Skylark Bio Inc. to inquire about joining.
Where is the NCT07627971 trial being conducted?
This trial is being conducted at Bethesda, United States, Boston, United States, Oklahoma City, United States, Allentown, United States.
Who is sponsoring the NCT07627971 clinical trial?
NCT07627971 is sponsored by Skylark Bio Inc.. The trial plans to enroll 10 participants.