Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age9-7
SponsorSkylark Bio Inc.
About this trial
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
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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 design
Design model
Sequential
Treatments tested in this trial
SKY-GJB2
Combination productSKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.
Treatment groups
10 Participants
are divided into 2 treatment groupsGroup A: OlderExperimental treatment 1 intervention
Group B: YoungerExperimental treatment 1 intervention
Trial outcomes
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))
Time frame
Through trial completion, approximately one yearSecondary outcomes
1
Auditory Brainstem Response (ABR) Threshold
Changes from baseline in ABR intensity threshold (measured in decibels (dB HL))
Time frame
Through trial completion, approximately one year2
Performance of the SKY-CAT delivery device
Looking at safety and effectiveness of the device in delivering the gene therapy.
Time frame
One month post-opSponsors and contacts
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Source ClinicalTrials.gov