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Aavanc80-3’Hotof

This article discusses the clinical trials of AAVAnc80-hOTOF, a promising gene therapy for individuals with sensorineural hearing loss caused by mutations in the otoferlin gene (OTOF). The trials aim to evaluate the safety, tolerability, and effectiveness of this innovative treatment approach for patients with a specific type of genetic hearing loss.

Table of Contents

What is AAVAnc80-hOTOF?

AAVAnc80-hOTOF is an exciting new gene therapy being developed to treat a specific type of hearing loss called otoferlin gene-mediated hearing loss[1]. This therapy is designed to help people who have hearing problems because of mutations in their otoferlin gene (OTOF).

How Does It Work?

AAVAnc80-hOTOF uses a special type of virus called AAV (adeno-associated virus) to deliver a healthy copy of the otoferlin gene to the inner ear[1]. The therapy consists of two parts:

  • AAVANC80-5’HOTOF: This is one part of the gene therapy.
  • AAVANC80-3’HOTOF: This is the other part of the gene therapy.

Together, these two parts carry the complete instructions for making the otoferlin protein, which is essential for normal hearing[1].

Who Can Benefit from This Treatment?

This therapy is being developed for people who have profound bilateral sensorineural hearing loss due to mutations in both copies of their otoferlin gene. The current clinical trial is focusing on:

  • Children aged 7 to 17 years old for the initial group
  • Children aged 2 to 17 years old for later groups[1]

To be eligible, patients must have confirmed mutations in their OTOF gene and show signs of profound hearing loss, such as no response to loud sounds in hearing tests[1].

Clinical Trials

AAVAnc80-hOTOF is currently being studied in clinical trials to determine its safety and effectiveness. The main trial is a Phase 1/2 study that aims to:

  • Assess the safety and tolerability of different doses of AAVAnc80-hOTOF
  • Measure how well the therapy improves hearing[1]

How is AAVAnc80-hOTOF Administered?

AAVAnc80-hOTOF is given as a one-time treatment directly into the inner ear. This is called an intracochlear administration. The procedure involves:

  • A surgical procedure under general anesthesia
  • Use of a special delivery device to inject the therapy into the cochlea (the part of the inner ear responsible for hearing)[1]

Safety and Efficacy

The main goals of the current clinical trial are to evaluate:

  • The safety of AAVAnc80-hOTOF, including any side effects
  • How well the therapy improves hearing, measured by various hearing tests[1]

Researchers will be closely monitoring participants for any adverse events and checking their hearing regularly to see if there are improvements.

Long-Term Follow-Up

Because AAVAnc80-hOTOF is a new type of treatment, it’s important to monitor its long-term effects. A separate long-term follow-up study is planned to:

  • Evaluate the long-term safety of AAVAnc80-hOTOF, including any late-occurring side effects
  • Assess how well the hearing improvements are maintained over time[2]

This follow-up study will involve regular check-ups and hearing tests for participants who received AAVAnc80-hOTOF in the initial clinical trial.

AAVAnc80-hOTOF represents an exciting potential breakthrough for people with otoferlin-mediated hearing loss. While it’s still in the testing phase, this gene therapy offers hope for a future where this type of genetic hearing loss could be treated effectively.

Aspect Details
Treatment AAVAnc80-hOTOF gene therapy
Target Condition Otoferlin gene-mediated sensorineural hearing loss
Trial Phases Phase 1/2 and Long-term Follow-up
Age Group 2-17 years old
Administration Intracochlear injection
Primary Objectives Safety, tolerability, and bioactivity assessment
Secondary Objectives Efficacy evaluation, including changes in hearing thresholds
Key Eligibility Criteria Confirmed biallelic OTOF mutations, profound bilateral hearing loss, preserved otoacoustic emissions
Follow-up Duration Extended long-term follow-up study

FAQ

  • What is AAVAnc80-hOTOF? AAVAnc80-hOTOF is a gene therapy that uses a modified virus to deliver a healthy copy of the otoferlin gene to the inner ear. It's designed to treat hearing loss caused by mutations in the otoferlin gene.
  • Who is eligible for these clinical trials? The trials are primarily for children and adolescents aged 2 to 17 years with confirmed biallelic mutations in the OTOF gene and profound bilateral sensorineural hearing loss. Specific eligibility criteria may vary between different phases of the trials.
  • How is the gene therapy administered? AAVAnc80-hOTOF is administered through a surgical procedure directly into the cochlea, which is part of the inner ear. This is called intracochlear administration.
  • What are the main objectives of these clinical trials? The main objectives are to assess the safety and tolerability of AAVAnc80-hOTOF, evaluate its effectiveness in improving hearing, and monitor long-term effects and potential late-occurring side effects.
  • How long will participants be followed after receiving the treatment? Participants will be followed for an extended period after receiving the treatment. There is a separate long-term follow-up study to evaluate the safety and effectiveness of AAVAnc80-hOTOF over time.

Ongoing Clinical Trials on Aavanc80-3’Hotof

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

    Recruiting

    1 1 1
    Investigated drugs:
    Spain

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

    Not yet recruiting

    1 1
    Investigated drugs:
    Spain

Glossary

  • Sensorineural hearing loss: A type of hearing loss caused by damage to the inner ear or the nerve pathways from the inner ear to the brain. In this case, it's specifically caused by mutations in the otoferlin gene.
  • Otoferlin gene (OTOF): A gene that provides instructions for making a protein called otoferlin, which is crucial for the proper functioning of hair cells in the inner ear and essential for normal hearing.
  • Gene therapy: A technique that uses genes to treat or prevent disease. In this case, it involves delivering a healthy copy of the otoferlin gene to the inner ear to potentially restore hearing function.
  • AAVAnc80-hOTOF: The name of the gene therapy product being tested. It uses a modified virus (AAVAnc80) to deliver the human otoferlin gene (hOTOF) to the inner ear.
  • Intracochlear administration: The method of delivering the gene therapy directly into the cochlea, which is part of the inner ear, through a surgical procedure.
  • Biallelic mutations: Genetic mutations that occur in both copies of a gene, one inherited from each parent. In this case, it refers to mutations in both copies of the otoferlin gene.
  • Auditory Brainstem Response (ABR): A test that measures the brain's response to sound and is used to assess hearing function, particularly in infants and young children.
  • Otoacoustic Emissions (OAEs): Sounds generated by the inner ear, specifically by the outer hair cells of the cochlea. The presence of OAEs indicates that the cochlea is functioning properly.

References

  1. http://clinicaltrials.eu/trial/study-on-gene-therapy-aavanc80-hotof-for-patients-with-sensorineural-hearing-loss-from-otoferlin-gene-mutations/
  2. http://clinicaltrials.eu/trial/study-on-long-term-safety-of-aavanc80-hotof-gene-therapy-for-patients-with-sensorineural-hearing-loss-due-to-otoferlin-gene-mutations/