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Study on Long-Term Safety of AAVAnc80-hOTOF Gene Therapy for Patients with Sensorineural Hearing Loss Due to Otoferlin Gene Mutations

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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 caused by mutations in the Otoferlin gene (OTOF). The treatment being investigated is a gene therapy called AAVAnc80-hOTOF. This therapy uses a special delivery system, known as an adeno-associated viral (AAV) vector, to introduce a normal version of the OTOF gene into the body. The aim is to evaluate the long-term safety of this treatment, including any late-occurring side effects.

Participants in this study have previously received an injection of AAVAnc80-hOTOF directly into the cochlea, which is part of the inner ear. The study will monitor these individuals over an extended period to ensure the treatment remains safe and to observe any potential side effects. The study will also assess the effectiveness of the treatment in improving hearing through various tests, such as measuring hearing thresholds and speech recognition.

The study will involve regular check-ups, including physical exams and hearing tests, to track the participants' health and hearing abilities. The goal is to gather comprehensive data on the safety and potential benefits of AAVAnc80-hOTOF for individuals with OTOF-related hearing loss.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after successful completion of the informed consent process. This ensures understanding and agreement to comply with study requirements.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to confirm eligibility. This includes reviewing the history of receiving the AAVAnc80-hOTOF gene therapy in a previous trial.

    A comprehensive medical evaluation is performed, including a physical examination and review of medical history.

  3. Step 3

    Administration of gene therapy

    The gene therapy product, AAVAnc80-hOTOF, is administered through an intracochlear injection. This means the solution is injected directly into the cochlea, a part of the inner ear.

  4. Step 4

    Monitoring and follow-up

    Regular follow-up visits are scheduled to monitor safety and efficacy. These visits include various assessments such as physical examinations, laboratory tests, and hearing evaluations.

    Safety assessments focus on identifying any adverse events, changes in vital signs, and results from laboratory tests like blood and urine analysis.

    Hearing assessments include tests like auditory brainstem response (ABR) testing, behavioral audiometry, and speech recognition tests.

  5. Step 5

    Long-term evaluation

    The study aims to evaluate the long-term safety and efficacy of the gene therapy. This involves ongoing monitoring for any late-occurring adverse events and changes in hearing ability.

    The study is expected to continue until April 29, 2033, providing a comprehensive evaluation over an extended period.

Who can join the trial?

4 criteria

  • Individuals with **OTOF-mediated hearing loss** who have already received a specific treatment called **intracochlear administration of AAVAnc80-hOTOF** in a previous clinical trial.
  • The person or their legally authorized representative must agree to follow all the study requirements. This is shown by completing a process called **informed consent**, which means they understand and agree to participate in the study.
  • Participants can be of any gender, meaning both males and females are eligible.
  • The study includes people from different age groups, including children, teenagers, and adults.

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 a problem with the otoferlin gene.
  • Participants must be within certain age ranges, which are not specified here, but generally include children and adults.
  • Both **male** and **female** participants are eligible, but certain conditions may exclude individuals.
  • People who are considered part of a **vulnerable population** may be excluded. This term often refers to groups who might need special protection, like children or those with certain disabilities.
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Investigated drugs

AAVAnc80-hOTOF is a gene therapy being studied for its potential to treat individuals with sensorineural hearing loss caused by mutations in the Otoferlin gene (OTOF). This therapy uses a specially designed virus to deliver a healthy copy of the Otoferlin gene to the cells in the ear. The goal is to help these cells function properly, which may improve hearing. The trial is focused on assessing the long-term safety of this therapy, including any adverse events that might occur over time.

What is already known about the treatment

AAVAnc80-hOTOF – This medication is administered through gene therapy, specifically targeting the inner ear to address otoferlin gene-mediated hearing loss. It is currently under clinical investigation, with ongoing studies evaluating its long-term safety and effectiveness. The primary therapeutic indication is sensorineural hearing loss caused by mutations in the otoferlin gene. At the molecular level, the therapy works by delivering a functional copy of the otoferlin gene using an adeno-associated virus vector, which helps 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 can lead to a disruption in the transmission of sound signals to the brain, resulting in hearing loss. This type of hearing loss is typically present from birth and can vary in severity. It is often identified through hearing tests that measure the ability to hear different frequencies and volumes. 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
Age18+ yearsPhasePhase IVTrial ID2024-512584-31-00Protocol codeAK-OTOF-LTFUEstimated enrolment14 patientsSponsorAkouos Inc.

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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.

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