EH002
Study sponsor: Yilai Shu / Eye & ENT Hospital of Fudan University
A dose-escalation study of EH002 administered into one or both ears for congenital hearing loss caused by OTOF variants.
Plain-language summary
Study overview
- Current study status
- Actively Recruiting
- Who is being studied
- 6 months and older; additional eligibility criteria apply
- How treatment is given
- Intracochlear injection
- Study start date
- Nov 22, 2024
Study start date is reported by ClinicalTrials.gov and may differ from the date the first participant received treatment.
Official study record
NCT06722170 · Status shown: Actively Recruiting · Registry information last checked Jul 25, 2025
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 DFNB9 congenital hearing loss
- Age range
- 6 months and older; additional eligibility criteria apply
- Planned enrollment
- 24
- Study regions
- China
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
- OTOF gene therapy with program-specific AAV1 neutralizing-antibody screening; the complete vector cassette and split strategy are not publicly disclosed
- Promoter
- 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
- Intracochlear injection
- How the inner ear is reached
- The precise cochlear access route is not publicly disclosed
- Fenestration
- Not publicly disclosed
- One or both ears
- One or both ears; one or two administrations
- Study start date
- Nov 22, 2024
Public evidence reviewed
Evidence and limitations
The EH002 registry includes AAV1 neutralizing-antibody screening but does not disclose the complete cassette, promoter, or cochlear access route. Related AAV1-hOTOF work from the investigator is shown only as platform context and is not used to fill EH002-specific fields.
Details not publicly disclosed
- Complete vector cassette
- Split strategy
- Promoter identity
- Precise cochlear access route
- Fenestration location