An Evaluation of the Long Term Safety and Efficacy of AAVAnc80-hOTOF Gene Therapy in Individuals with Sensorineural Hearing Loss due to Otoferlin Gene (OTOF) Mutations

Status

Recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-512584-31-00

Acronym

AK-OTOF-LTFU

Enrollment

Registered

2024-07-24

Start date

2024-11-11

Completion date

Unknown

Last updated

2025-08-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Otoferlin gene-mediated hearing loss

Brief summary

Safety: Incidence and frequency of AEs, Safety: Concomitant Medications, Safety: Clinical labs: chemistries, hematology (CBC with differential), urinalysis, Safety: Physical examination and vital signs, Safety: Otoscopic examination, Safety: Vestibular screening, Safety: Tympanometry

Detailed description

Efficacy: Diagnostic ABR testing threshold, Efficacy: Age-appropriate behavioral audiometry, Efficacy: Standard word or speech recognition testing, Efficacy: Standard auditory questionnaires

Interventions

DRUGAAVAnc80-hOTOF

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Safety: Incidence and frequency of AEs, Safety: Concomitant Medications, Safety: Clinical labs: chemistries, hematology (CBC with differential), urinalysis, Safety: Physical examination and vital signs, Safety: Otoscopic examination, Safety: Vestibular screening, Safety: Tympanometry	—

Secondary

Measure	Time frame
Efficacy: Diagnostic ABR testing threshold, Efficacy: Age-appropriate behavioral audiometry, Efficacy: Standard word or speech recognition testing, Efficacy: Standard auditory questionnaires	—

Countries

Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026