HOPE THROUGH RESEARCH:
A clinical study testing an investigational therapy targeting GJB2-hearing loss
Study Overview
Watch our video to learn more
What is the purpose of the Sonix study?
The purpose of this study is to find out if an investigational drug called SKY-GJB2 is safe and effective when administered to children with GJB2-mediated hearing loss. ‘Investigational’ means that SKY-GJB2 has not yet been approved by the U.S. Food and Drug Administration for the treatment of GJB2-mediated hearing loss.
What is GJB2-mediated hearing loss?
Hearing loss can have many different causes. GJB2-mediated hearing loss is a genetic condition caused by mutations in a person’s GJB2 gene. This gene encodes a protein called Connexin 26, which is important for maintaining the balance of potassium levels in the inner ear (the cochlea). Disruptions to this balance affects the inner ear’s ability to function properly. GJB2 gene mutations are responsible for a majority of autosomal recessive non-syndromic hearing loss that are present from birth across many populations worldwide.
What is the study drug, SKY-GJB2?
SKY-GJB2 is an investigational gene therapy. Gene therapy is a way to modify (add, remove, or change) genes contained in a person's DNA to treat or cure a disease or condition. Think of your DNA as an instruction manual that contains instructions for your body to function properly. Sometimes there is a ‘typo’ in the manual—a faulty gene that leads to the disease, in this case, hearing loss. Gene therapy ‘corrects the typo’ and fixes the manual so your body can follow the right instructions and function properly.
Who can participate in the Sonix study?
To be eligible, your child must:
- Be 9 months to 7 years of age at the time of dosing
- Be diagnosed with GJB2-mediated hearing loss
Your child’s medical history will be reviewed and additional study criteria will be used to determine eligibility.
Taking Part in the Study
What is involved in the study?
The main study lists up to a year and is divided into several parts:
| Study Period | Description |
|---|---|
| 1 Screening Period (up to 4 weeks) | Tests (medical exams and laboratory tests) will be done to see if your child can be included in the study. A CT scan of your child’s head will be performed if not available within the last 4 years. |
| 2 Dosing (1 visit) | If your child is eligible, they will be given a single dose of SKY-GJB2 in one ear as an infusion through the ear canal into the cochlea (inner ear). This is a same-day outpatient surgical procedure performed under general anesthesia. |
| 3 Follow-up (12 months) | For the next 12 months your child will have 10 more scheduled visits for study testing and health checks. |
Will the study cost anything?
The study drug, study-specific tests and procedures, and clinic visits that are required for the study are all provided at no cost to qualified families. Additionally, families will be reimbursed for reasonable study-related travel expenses and compensated for each visit. Talk to the study doctor for more information.
Should I enroll my child in the Sonix study?
Your child may or may not benefit from participation in this study. Your family’s participation will help to add knowledge about SKY-GJB2 and GJB2-mediated hearing loss, which may help others in the future.
Taking part in a clinical study is completely voluntary. Participation is not required, and if enrolled, you can choose to end your child’s participation at any time and for any reason.
About Clinical Studies
What is a clinical study?
A clinical study, also known as a clinical trial, is designed to determine how safe and how well an investigational drug is able to treat a specific disease or condition. The results of clinical studies help health authorities decide if an investigational drug should be made available to patients. Volunteer involvement in clinical studies is a key part in the development and advancement of future therapies.
How are my rights protected?
Every clinical study is reviewed by an ethics committee, which helps make sure that the study is conducted safely and that the rights of study participants are protected. Clinical studies are conducted by trained medical professionals who monitor the health of participants throughout the study.