NCT07627971

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)

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
23mo left

Started May 2026

Typical duration for phase_1

Geographic Reach
1 country

3 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress9%
May 2026Jun 2028

First Submitted

Initial submission to the registry

May 28, 2026

Completed
Same day until next milestone

Study Start

First participant enrolled

May 28, 2026

Completed
7 days until next milestone

First Posted

Study publicly available on registry

June 4, 2026

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2027

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2028

Last Updated

July 15, 2026

Status Verified

July 1, 2026

Enrollment Period

1.5 years

First QC Date

May 28, 2026

Last Update Submit

July 13, 2026

Conditions

GJB2-mediated Hearing LossGJB2 Gene MutationDFNB1A

Keywords

GJB2Hearing Loss, BilateralHearing LossHearing Loss, SensorineuralDeafness, Autosomal Recessive 1A (DFNB1A)Connexin 26

Outcome Measures

Primary Outcomes (1)

  • 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))

    Through trial completion, approximately one year

Secondary Outcomes (2)

  • Auditory Brainstem Response (ABR) Threshold

    Through trial completion, approximately one year

  • Performance of the SKY-CAT delivery device

    One month post-op

Study Arms (2)

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.

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.

Combination Product: SKY-GJB2

Interventions

SKY-GJB2COMBINATION_PRODUCT

SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.

OlderYounger

Eligibility Criteria

Age9 Months - 7 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Massachusetts Eye and Ear Infirmary (MEEI)

Boston, Massachusetts, 02114, United States

RECRUITING

Hearts for Hearing-Oklahoma City

Oklahoma City, Oklahoma, 73120, United States

RECRUITING

Lehigh Valley Hospital-Cedar Crest

Allentown, Pennsylvania, 181103, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Deafness, Autosomal Recessive 1AHearing Loss, BilateralHearing LossHearing Loss, SensorineuralDeafness

Condition Hierarchy (Ancestors)

Hearing DisordersEar DiseasesOtorhinolaryngologic DiseasesSensation DisordersNeurologic ManifestationsNervous System DiseasesSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 28, 2026

First Posted

June 4, 2026

Study Start

May 28, 2026

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

June 1, 2028

Last Updated

July 15, 2026

Record last verified: 2026-07

Locations

US(3)