Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not yet recruitingRare disease

Study on Gene Therapy AAVAnc80-hOTOF for Patients with Sensorineural Hearing Loss from Otoferlin Gene Mutations

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as sensorineural hearing loss, which is caused by mutations in the Otoferlin gene (OTOF). The treatment being tested is a gene therapy called AAVAnc80-hOTOF. This investigational treatment uses a special type of virus, called an adeno-associated viral (AAV) vector, to deliver a healthy version of the OTOF gene to the inner ear. The therapy is administered directly into the cochlea, which is the part of the ear responsible for hearing, using a sterile delivery device.

The purpose of the study is to evaluate the safety and effects of different doses of AAVAnc80-hOTOF in individuals with this specific type of hearing loss. Participants will receive the treatment through an injection into the cochlea, and the study will monitor their response over time. The trial will assess how well the treatment is tolerated and whether it has any impact on hearing ability.

Throughout the study, participants will undergo various tests, including auditory brainstem response (ABR) testing, to measure changes in their hearing. The study aims to gather information on any side effects related to the treatment and the procedure used to administer it. This research is expected to continue until 2028, providing valuable insights into the potential of gene therapy for treating hearing loss caused by OTOF mutations.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria, including age and genetic testing for OTOF gene mutations.

    Participants must have profound bilateral sensorineural hearing loss and preserved otoacoustic emissions in the study ear.

  2. Step 2

    Administration of gene therapy

    The investigational product, AAVAnc80-hOTOF, is administered as a solution for injection directly into the cochlea (inner ear).

    The objective is to assess safety, tolerability, and bioactivity of the treatment.

  3. Step 3

    Monitoring and follow-up

    Participants are monitored for any adverse effects related to the treatment or the procedure.

    Regular assessments include changes in auditory brainstem response (ABR) testing thresholds to evaluate hearing improvements.

  4. Step 4

    Long-term follow-up

    Participants are expected to engage in a long-term follow-up protocol after the completion of the trial to monitor ongoing effects and safety.

Who can join the trial?

7 criteria

  • Age between seven and 17 years old for one group; age between two and 17 years old for other groups at the time of treatment.
  • Have changes in both copies of the **OTOF gene**, confirmed by a specific genetic test. The **OTOF gene** is related to hearing.
  • Show signs of **Profound bilateral sensorineural hearing loss**, which means very severe hearing loss in both ears, as determined by a specific hearing test called **ABR**. This test checks how the brain responds to sound.
  • Have normal **OAEs** in the study ear. **OAEs** are sounds that the ear produces in response to a sound, and they help check if the ear is working properly.
  • If able to have children, agree to use effective birth control methods for one year after treatment. This applies to both males and females who can have children.
  • The person responsible for the participant, or the participant themselves, must agree to follow all trial requirements and complete the consent process.
  • The person responsible for the participant, or the participant themselves, must agree to take part in a separate follow-up study after this trial is completed.

Who cannot join the trial?

4 criteria

  • Individuals who do not have **Otoferlin gene-mediated hearing loss** cannot participate. This is a specific type of hearing loss caused by changes in the otoferlin gene.
  • Participants must be within the specified age range, which is typically defined by the study. If you are outside this age range, you cannot join.
  • Both **male and female** participants are allowed, but if the study has specific gender requirements, those not meeting them cannot participate.
  • If you belong to a **vulnerable population**, such as children or individuals unable to give consent, you may not be eligible unless specific provisions are made.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not yet recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

AAVAnc80-hOTOF is a gene therapy being studied for its potential to help individuals with sensorineural hearing loss caused by mutations in the otoferlin gene. This therapy uses a specially designed virus to deliver a healthy copy of the otoferlin gene to the cells in the ear, with the aim of restoring hearing function. The trial is focused on evaluating how safe and tolerable this therapy is, as well as how effectively it works at different dose levels.

What is already known about the treatment

AAVAnc80-hOTOF – This medication is administered through gene therapy, specifically targeting the inner ear to address hearing loss. It is currently in Phase 1/2 clinical trials, focusing on its safety and effectiveness. The main therapeutic indication is for individuals with sensorineural hearing loss caused by mutations in the otoferlin gene. At the molecular level, it works by delivering a functional copy of the otoferlin gene to the affected cells, aiming to restore normal hearing function. It falls under the pharmacological classification of gene therapy products.

Investigated diseases

Otoferlin Gene-Mediated Hearing Loss – This is a genetic condition that affects hearing due to mutations in the otoferlin gene. The otoferlin gene is crucial for the proper functioning of hair cells in the inner ear, which are responsible for converting sound waves into nerve signals. When this gene is mutated, it disrupts the normal process of hearing, leading to hearing loss. The condition is typically present from birth and can vary in severity. It primarily affects the ability to hear high-frequency sounds, which can impact speech and language development. The progression of hearing loss can be stable or may worsen over time, depending on the specific genetic mutation.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase I/IITrial ID2023-505128-76-00Protocol codeAK-OTOF-101Estimated enrolment14 patientsSponsorAkouos Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).