Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
Recruiting
Phase 1
Phase 2
Interventional Study
GJB2-mediated Hearing Loss
GJB2 Gene Mutation
DFNB1A
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 9 Months – 7
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 10 (estimated)
- Sponsor
- Skylark Bio Inc. · Industry
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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 toler…
Trial Locations
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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 a…
Contacts
No contact information available.