OTOV101N + OTOV101C
Study sponsor: Otovia Therapeutics
An investigator-initiated program evaluating a dual-vector OTOF gene-replacement approach in people with DFNB9 hearing loss.
Plain-language summary
Study overview
- Current study status
- Active
- Who is being studied
- 1 year and older
- How treatment is given
- Intracochlear administration
- Study start date
- Jun 26, 2023
Study start date is reported by ClinicalTrials.gov and may differ from the date the first participant received treatment.
Official study record
NCT05901480 · Status shown: Active · Registry information last checked not recently verified
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 aged 1 year and older with OTOF-related hearing loss
- Age range
- 1 year and older
- Planned enrollment
- Not publicly disclosed
- 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
- Dual Anc80L65 vectors encoding human OTOF, split between exons 20 and 21 with splice-donor/acceptor-mediated reconstitution
- Promoter
- mMyo15 hair-cell-specific promoter
Delivery to the inner ear
How the therapy is administered and whether one or both ears are treated.
- How it is delivered
- Intracochlear administration
- How the inner ear is reached
- Round-window membrane injection; preclinical program work used a transmastoid facial-recess corridor
- Fenestration
- No separate fenestration reported; round-window membrane injection
- One or both ears
- Cohort-dependent
- Study start date
- Jun 26, 2023
Public evidence reviewed
Evidence and limitations
The NCT-linked human paper identifies the clinical vector capsid. Program-specific preclinical work resolves the dual-vector split, reconstitution strategy, mMyo15 promoter, and round-window route.
Details not publicly disclosed
- Current registry status
- Human operative corridor