SENS-501 (Audiogene)
Study sponsor: Sensorion
An adaptive dose-escalation and expansion study evaluating unilateral intracochlear SENS-501 administration in very young children with OTOF-related hearing loss.
Plain-language summary
Study overview
- Current study status
- Active, Not Recruiting
- Who is being studied
- 6–31 months
- How treatment is given
- Unilateral intracochlear injection with a proprietary delivery device
- Study start date
- Jun 21, 2024
Study start date is reported by ClinicalTrials.gov and may differ from the date the first participant received treatment.
Official study record
NCT06370351 · Status shown: Active, Not Recruiting · Registry information last checked Jul 8, 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
- Children with severe-to-profound pre-lingual OTOF-related hearing loss
- Age range
- 6–31 months
- Planned enrollment
- 12
- Study regions
- Australia and France
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
- Hybrid dual AAV8 vectors encoding human OTOF transcript variant 5, with AP1-mediated reconstitution
- Promoter
- CMV promoter
Delivery to the inner ear
How the therapy is administered and whether one or both ears are treated.
- How it is delivered
- Unilateral intracochlear injection with a proprietary delivery device
- How the inner ear is reached
- Round-window membrane injection; translational work describes stapedotomy pressure relief
- Fenestration
- Yes — stapes footplate (stapedotomy)
- One or both ears
- Unilateral
- Study start date
- Jun 21, 2024
Public evidence reviewed
Evidence and limitations
A program-specific peer-reviewed paper resolves the dual-AAV8 construct, OTOF isoform, AP1 recombination design, CMV promoter, and round-window delivery details missing from the registry.