AK-OTOF-101
Study sponsor: Akouos, Inc. / Eli Lilly
A dose-escalation and bilateral-expansion study of a dual-AAVAnc80 OTOF construct delivered with a dedicated intracochlear administration system.
Plain-language summary
Study overview
- Current study status
- Actively Recruiting
- Who is being studied
- Cohort 1a: 7–17 years Cohort 1b: first 3 participants are 2 years and older, next 3 may be any age Cohort 2: first 3 participants are 2 years and older, next 3 may be any age bilateral expansion: any age
- How treatment is given
- One-time intracochlear administration using the Akouos delivery device
- Study start date
- Sep 15, 2023
Study start date is reported by ClinicalTrials.gov and may differ from the date the first participant received treatment.
Official study record
NCT05821959 · Status shown: Actively Recruiting · Registry information last checked Mar 4, 2026
Terminology Dictionary
- Target gene
- The gene the treatment is designed to address.
- Gene replacement
- Delivery of a working gene copy intended to restore a missing or impaired function.
- Vector / payload
- The delivery vehicle and the genetic material it carries.
- Promoter
- A DNA control sequence that helps determine where and how strongly the delivered gene is active.
- Cochlear access
- The route used to reach the inner ear and administer the treatment.
- Laterality
- Whether treatment is given to one ear or both ears.
- Phase 1 / 2
- An early human study designed mainly to examine safety, dosing, and initial signs of biological or clinical effect.
- Early clinical
- An early human study for which a formal clinical phase is not assigned or publicly stated.
- Biallelic
- A disease-causing change is present in both copies of a gene.
- Dual vector
- Two delivery vehicles carry separate parts of a gene that is too large to fit into one vehicle.
- Intracochlear
- Delivered into the fluid-filled inner-ear structure involved in hearing.
Who is being studied
The population described by the public study record.
- Study population
- People with OTOF-mediated sensorineural hearing loss
- Age range
- Cohort 1a: 7–17 years Cohort 1b: first 3 participants are 2 years and older, next 3 may be any age Cohort 2: first 3 participants are 2 years and older, next 3 may be any age bilateral expansion: any age
- Planned enrollment
- 22
- Study regions
- Canada, Taiwan, United Kingdom, and United States
This summary describes the study population; it cannot determine whether an individual qualifies.
Therapy design
The genetic treatment and how its activity is controlled.
- Target gene
- OTOF
- Treatment approach
- Gene replacement
- Delivery vector and gene cargo
- Dual AAVAnc80 vectors carrying 5′ and 3′ components of approximately 6 kb human OTOF cDNA
- Promoter
- Ubiquitous promoter; specific identity is not publicly disclosed
Delivery to the inner ear
How the therapy is administered and whether one or both ears are treated.
- How it is delivered
- One-time intracochlear administration using the Akouos delivery device
- How the inner ear is reached
- Transcanal tympanotomy to the round-window membrane with a stapes-footplate vent
- Fenestration
- Yes — stapes footplate vent (fenestration)
- One or both ears
- Unilateral cohorts; bilateral expansion
- Study start date
- Sep 15, 2023
Public evidence reviewed
Evidence and limitations
The registry anchors the clinical record. Akouos technical material resolves the two-component vector, payload scale, promoter class, round-window route, pressure vent, and intended human corridor; the exact promoter identity remains undisclosed.
Details not publicly disclosed
- Specific promoter identity