SKY-GJB2-001

Full Clinical Trial Name

A Phase 1/2, open-label, single dose study of SKY-GJB2 given as a single unilateral intracochlear administration in pediatric subjects with severe-to-profound GJB2-mediated hearing loss

Clinical Trial

  • Surgery
  • Pediatrics
  • Hearing Loss in Children

Phase

Phase
1

Patient Type

Pediatric
Doctor(s) running this study
Ravi N. Samy, MD
Location(s):
Call 610-402-9543
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Purpose

The primary objective is to evaluate the safety and tolerability of the compound SKY-GJB2 given as an intracochlear (inner ear) infusion. Another aim is to explore the efficacy and potential future applications of SKY-GJB2 on treating GJB2-mediated hearing loss.  

Description

SKY-GJB2 will be administered as an intracochlear (IC) infusion to one ear using a transcanal endoscopic tympanotomy approach to access the round window under general anesthesia. Study participants will have an initial screening visit approximately one month before the study procedure. After the procedure, study participants are followed for one year by the investigator and study team to evaluate safety and collect data. 

Eligibility

Inclusion Criteria

Inclusion Criteria-including but may not be limited to:

  1. Males or females aged 9 months to 7 years inclusive.
  2. Participants must have at least two (biallelic) pathogenic or likely pathogenic variants in the GJB2 gene as confirmed as part of a comprehensive hearing loss/deafness gene panel test.
  3. Participants have a clinical presentation of bilateral, sensorineural hearing loss. With severe to profound hearing loss in the study treatment ear.

 

To Participate

Please contact the Network Office of Research and Innovation at 610-402-9543.

Learn more about the full clinical trial

Full description at sonixstudy.com

 

Doctor(s) running this study

Ravi N. Samy, MD

Specialties

  • Otolaryngology, Head and Neck Surgery (ENT)

Affiliated with