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Also known as AAVAnc80-hOTOF via Akouos Delivery Device
AK-OTOF · 1 trial · 1 indication
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system)
| Arm | Type | Description |
|---|---|---|
| Cohort 1a and Cohort 1b | EXPERIMENTAL | Cohort 1a: Two participants aged 7 to 17 years old (inclusive) to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device Cohort 1b: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age |
| Cohort 2 | EXPERIMENTAL | Cohort 2: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age |
| Bilateral Expansion | EXPERIMENTAL | Bilateral Expansion: up to eight eligible participants of any age, and no cochlear implants, may be eligible for enrollment into the Bilateral Expansion Cohort (simultaneous bilateral administration of AAVAnc80-hOTOF). |
| Name | Type | Description |
|---|---|---|
| AAVAnc80-hOTOF via Akouos Delivery Device | COMBINATION_PRODUCT | AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device. |
| AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism | COMBINATION_PRODUCT | AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time bilateral intracochlear administration using the Akouos Delivery Device together with the Precision Delivery Mechanism. |
Criteria for Inclusion: 1. Participants may be of any age, based on Cohort Criteria 2. At least two mutations in the otoferlin gene 3. Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR 4. Preserved distortion product otoacoustic emissions (DPOAEs) 5. Able and...