GJB2-mediated Hearing LossPhase 1/2SONIXNCT07627971
Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
A Phase 1/2 study testing SKY-GJB2 in people with GJB2-mediated Hearing Loss and GJB2 Gene Mutation, run by Skylark Bio Inc. at 4 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 9 months to 7 years
- Conditions the study is for
- GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
- What is being tested?
- SKY-GJB2
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 4 sitesUnited States
- Study dates
- 28 May 2026to Jun 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 months to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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 join
- 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.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Lehigh Valley Hospital-Cedar Crest
Allentown, Pennsylvania, 181103
Ravi N. Samy, MD, FACS, 610-402-9543, Ravi.Samy@Jefferson.edu
Danielle Koehler, RN (Study Coordinator), Danielle.Koehler@Jefferson.edu
- Recruiting
National Institutes of Health-Clinical Center
Bethesda, Maryland, 20892
Maame Akumatey (Study Coordinator), 227-233-0452, maame.akumatey@nih.gov
Ronna Hertzano, MD, PhD, 301-496-3805, ronna.hertzano@nih.gov
- Recruiting
Massachusetts Eye and Ear Infirmary (MEEI)
Boston, Massachusetts, 02114
Michael Cheung, MSc, CCRP, Clinical Research Project Manager, 617-573-6060, centerforclinicalresearchoperations@meei.harvard.edu
- Recruiting
Hearts for Hearing-Oklahoma City
Oklahoma City, Oklahoma, 73120
René Gifford, PhD, 4055484300, research@heartsforhearing.org
About this study
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
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
- GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
- Being tested
- SKY-GJB2
- Keywords
- GJB2, Hearing Loss, Bilateral, Hearing Loss, Hearing Loss, Sensorineural, Deafness, Autosomal Recessive 1A (DFNB1A), Connexin 26
- Medical subject headings
- Deafness, Autosomal Recessive 1A, Hearing Loss, Bilateral, Hearing Loss, Hearing Loss, Sensorineural, Deafness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SKY-GJB2Combination product
SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.
| Study group | Role | What they receive |
|---|---|---|
| Older | Experimental | 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. Receives: Combination Product: SKY-GJB2 |
| Younger | Experimental | 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. Receives: Combination Product: SKY-GJB2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety/Frequency of Adverse Events (AEs)
AEs as it relates to the the gene therapy (SKY-GJB2) and/or to the administration procedure (including the delivery device system (SKY-CAT))
Measured: Through trial completion, approximately one year
Secondary outcomes (2)
Auditory Brainstem Response (ABR) Threshold
Changes from baseline in ABR intensity threshold (measured in decibels (dB HL))
Measured: Through trial completion, approximately one year
Performance of the SKY-CAT delivery device
Looking at safety and effectiveness of the device in delivering the gene therapy.
Measured: One month post-op
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2026
- Study start28 May 2026
- Primary completionDec 2027 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GJB2-mediated Hearing LossStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Skylark Bio Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07627971
- Sponsor's protocol ID
- SKY-GJB2-001
- Acronym
- SONIX
- Status last verified
- Aug 2026
- First submitted
- 28 May 2026
- Last updated
- 27 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07627971
- Acronym / sponsor ID
- SONIX / SKY-GJB2-001
- Conditions
- GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
- Keywords
- GJB2, Hearing Loss, Bilateral, Hearing Loss, Hearing Loss, Sensorineural, Deafness, Autosomal Recessive 1A (DFNB1A), Connexin 26
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Skylark Bio Inc.Industry (pharma, biotech, devices)
- Study start
- 28 May 2026 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 4 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Older | Experimental | Combination Product: SKY-GJB2Four 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. |
| Younger | Experimental | Combination Product: SKY-GJB2Six 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. |
Who may qualify
- Ages
- 9 months to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SKY-GJB2SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device. | Combination product | Older, Younger |
Primary outcome
Safety/Frequency of Adverse Events (AEs)
Time frame: Through trial completion, approximately one year
AEs as it relates to the the gene therapy (SKY-GJB2) and/or to the administration procedure (including the delivery device system (SKY-CAT))
2 secondary outcomes
Auditory Brainstem Response (ABR) Threshold
Time frame: Through trial completion, approximately one year
Performance of the SKY-CAT delivery device
Time frame: One month post-op
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2026.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
National Institutes of Health-Clinical Center
Bethesda, United States
Massachusetts Eye and Ear Infirmary (MEEI)
Boston, United States
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SKY-GJB2 in people with GJB2-mediated Hearing Loss and GJB2 Gene Mutation, run by Skylark Bio Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety/Frequency of Adverse Events (AEs) (time frame: Through trial completion, approximately one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2026.