The goal of the study is to find out if the study drug, an adeno-associated viral (AAV) vector gene therapy, is safe and improves hearing in children with sensorineural hearing loss (SNHL) due to otoferlin gene (OTOF) mutations.
To learn more about the otoferlin gene and its role in SNHL, click the button below.
Sensorineural Hearing LossTo join the study, your child must meet the following requirements:
- Between age 7 to 17 years old (inclusive, Group 1a); between age 2 to 17 years old (inclusive, Groups 1b and 2) with sensorineural hearing loss in both ears
- Otoferlin gene mutations confirmed by genetic testing (genetic testing will be provided, as needed)
- Does not have cochlear implants in both ears
Other study requirements will apply
See if you may qualifyStudy participants can expect the following after reviewing and signing the Informed Consent Form (ICF):
- Screening period: Participant receives assessments to confirm they qualify to join the study.
- Study drug administration: If eligible, your child will have surgery to receive one dose of the study drug in one ear using a study device. Your child will stay overnight at the study site after surgery so the site staff can monitor their health.
- Follow-up visits: You and your child will attend 11 follow-up visits at the study site over the course of 2 years. Once you have completed the last visit of this study, you and your child will be expected to participate in a Long Term Follow-up Study (with annual visits) for an additional 3 years.
Participation in a clinical study is voluntary. You may be eligible for travel coverage or reimbursement. You can ask any questions you have and may leave the study at any time, for any reason.
