GJB2-mediated Hearing Loss study: SKY-GJB2
Official title Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
ClinicalTrials.gov ID: NCT07627971
What this study is testing
What is SKY-GJB2?
an investigational gene therapy being studied as a potential treatment for GJB2-mediated hearing loss in children is designed to deliver a functional copy of the GJB2 gene to inner‑ear cells to restore normal protein function
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether SKY-GJB2 is safe and how well it works for hearing loss.
- Who it's for
- You may be able to join if you are a child with hearing loss caused by a GJB2 gene change.
- What's involved
- You will receive SKY-GJB2 using the SKY-CAT device that puts the therapy into your ear. You will have check‑ins to see if it is safe and if hearing improves.
- Time commitment
- The study lasts several months and includes visits at the start and later check‑ins.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 9 to 7
You may be able to join if
- Male or female aged 9 months to 7 years at the time of gene therapy administration
- people 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...
- 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.
You likely can't join if
- 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.
The study team makes the final eligibility decision.
Where it is enrolling
- Bethesda, Maryland, United States
- Boston, Massachusetts, United States
- Oklahoma City, Oklahoma, United States
- Allentown, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 9 months to 7 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Bethesda, Maryland, United States; Boston, Massachusetts, United States; Oklahoma City, Oklahoma, United States; Allentown, Pennsylvania, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.