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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)
Changes from baseline in ABR intensity threshold (decibels normal hearing level \[dB nHL\]
Changes from baseline in the average of behavioral pure-tone audiometry thresholds
| Arm | Type | Description |
|---|---|---|
| Part A: 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 |
| Part A: 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 |
| Part A: 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). |
| Part B | EXPERIMENTAL | Part B: Sixteen participants of any age will be enrolled to receive a single unilateral or bilateral administration of AAVAnc80-hOTOF. Participants with one CI will receive unilateral administration of AAVAnc80-hOTOF, while participants with no CIs will have the choice to receive a unilateral or bilateral administration of AAVAnc80-hOTOF, provided both ears meet eligibility criteria. |
| Part C | EXPERIMENTAL | Up to ten eligible participants of any age will be enrolled in a region-specific expansion phase to receive a single unilateral or bilateral administration of AAVAnc80-hOTOF. Participants with one CI will receive unilateral administration of AAVAnc80-hOTOF, while participants with no CIs will have the choice to receive a unilateral or bilateral administration of AAVAnc80-hOTOF, provided both ears meet eligibility criteria. |
| Part D | EXPERIMENTAL | Participants of any age may be enrolled into Part D, which is a sequential administration phase that allows participants who received unilateral administration of AAVAnc80-hOTOF in AK-OTOF-101 (Parts A, B, or C) the option to receive dose administration of AAVAnc80-hOTOF in the contralateral ear after completing the Year 1 visit. |
| 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 (Part A) or Severe to Profound (Parts B, C, and D) bilateral sensorineural hearing loss as assessed by ABR 4. Preserved distortion...
AK-OTOF is an investigational gene therapy being developed for bilateral sensorineural hearing loss. It is designed for hearing loss mediated by mutations in the otoferlin gene. The therapy is administered using the Akouos Delivery Device and is currently being evaluated in clinical trials rather than being available as an approved treatment.
AK-OTOF is being developed by Eli Lilly and Company, which trades on the New York Stock Exchange under the ticker LLY. The therapy originated from Akouos, a company focused on inner ear gene therapies, and is now part of Lilly's clinical pipeline for hearing loss.
AK-OTOF is in Phase 3 clinical development. The program is currently recruiting participants for its Phase 3 trial, and no Phase 3 results have been completed or reported yet. It remains an investigational therapy and has not been approved by the FDA.
AK-OTOF is being studied in the Phase 3 trial NCT05821959, titled Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss. The trial is recruiting up to 48 participants across the United States, Canada, Taiwan, and the United Kingdom. It enrolls patients with bilateral sensorineural hearing loss and uses a controlled design.
Yes, AK-OTOF is also known as AAVAnc80-hOTOF via Akouos Delivery Device. This alternative name describes the gene therapy construct and the delivery device used to administer it to the inner ear. Both names refer to the same investigational treatment being developed by Eli Lilly and Company.