The goal of the trial is to find out if the study drug, an adeno-associated viral (AAV) vector gene therapy, is safe and improves hearing in individuals 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 trial, you or your child must meet the following requirements:
- Have sensorineural hearing loss in both ears
- Otoferlin gene mutations confirmed by genetic testing (genetic testing will be provided, as needed)
- Do not have cochlear implants in both ears
Other requirements will apply
See if you or your child may qualifyTrial 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 trial.
- Study drug administration: If eligible, you or your child will have surgery to receive one dose of the study drug in one ear using the study device. You or your child will stay overnight at the trial site after surgery so the site staff can monitor your or your child’s health.
- Follow-up visits: You or your child will attend approximately nine follow-up visits at the trial site over the course of one year. Once you or your child have/has completed the last visit of this trial, you or your child will be expected to participate in a Long Term Follow-up Study (with annual visits) for approximately four additional years.
Participation in a clinical trial 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.
