What is the mechanism of action of this treatment?

Gene therapy for Sensorineural Hearing Loss (SNHL), such as the AAVAnc80-hOTOF trial, involves using adeno-associated virus (AAV) vectors to deliver functional copies of the otoferlin gene to the inner ear. Otoferlin is crucial for the proper functioning of hair cells in the cochlea, which are responsible for converting sound waves into electrical signals for the brain. By restoring the expression of otoferlin, this therapy aims to correct the underlying genetic defect causing SNHL, potentially improving or restoring hearing. This approach is significant for SNHL patients as it targets the root cause of hearing loss rather than just alleviating symptoms, offering a promising avenue for long-term treatment.

What side effects are associated with this type of intervention?

Common treatments for Sensorineural Hearing Loss (SNHL) include hearing aids, cochlear implants, and gene therapy. Gene therapy, such as the AAVAnc80-hOTOF trial, involves using an adeno-associated virus (AAV) vector to deliver a functional gene to the affected cells. Known side effects of gene therapy can include immune reactions to the viral vector, which may cause inflammation or other immune responses. Additionally, there is a risk of off-target effects where the gene is inserted into unintended locations in the genome, potentially leading to other complications. Other general side effects may include mild to moderate pain at the injection site, headache, and temporary hearing changes.

What prior FDA approvals exist?

As of now, there are no FDA-approved gene therapies specifically for the treatment of Sensorineural Hearing Loss. The trial involving AAVAnc80-hOTOF represents a novel approach using an AAV vector to deliver the otoferlin gene, which is still under investigation. Most current treatments for Sensorineural Hearing Loss focus on hearing aids, cochlear implants, and pharmacological interventions, rather than gene therapy. The field of gene therapy for hearing loss is still emerging, with ongoing research and clinical trials aimed at developing effective treatments.

What other types of research exist?

Promising alternatives to AAVAnc80-hOTOF for sensorineural hearing loss include: 1) CRISPR-Cas9 gene editing, which offers precise genetic modifications to correct mutations causing hearing loss; 2) RNA-based therapies, such as antisense oligonucleotides and RNA interference, which can target and modulate gene expression; 3) Regenerative medicine approaches, including stem cell therapy, which aims to regenerate damaged hair cells in the cochlea; and 4) Pharmacological agents targeting molecular pathways involved in hearing loss, such as neurotrophic factors and small molecules that promote hair cell survival and function.

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Virus Therapy

Gene Therapy for Hearing Loss

Phase 1 & 2

Recruiting

Research Sponsored by Akouos, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through trial completion, approximately 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new gene therapy delivered to the ear to help people with hearing issues. The therapy aims to improve ear cell function by introducing a healthy gene. Two different doses will be tested to ensure safety and effectiveness. Gene therapy has emerged as a promising strategy for treating hearing loss by addressing genetic dysfunction.

Who is the study for?

This trial is for children and teens aged 2 to 17 with profound bilateral sensorineural hearing loss due to biallelic mutations in the otoferlin gene. They must be able to follow the study's procedures, including a long-term follow-up. Those with cochlear implants, persistent ear infections, or conditions that make surgery risky can't participate.

What is being tested?

The trial tests a gene therapy called AAVAnc80-hOTOF delivered through an Akouos device into one ear. It aims to see if it's safe and works as intended at two different dose levels.

What are the potential side effects?

Potential side effects are not detailed here but may include reactions related to surgery, anesthesia, and gene therapy administration such as inflammation or immune responses.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through trial completion, approximately 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through trial completion, approximately 1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and through trial completion, approximately 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Frequency of Adverse Events (AEs)

Secondary study objectives

Auditory Brainstem Response (ABR) Threshold

Performance of the Akouos Delivery Device

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment1 Intervention

Cohort 2: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age

Group II: Cohort 1a and Cohort 1bExperimental Treatment1 Intervention

Cohort 1a: Two participants aged 7 to 17 years old (inclusive) to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device Cohort 1b: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Gene therapy for Sensorineural Hearing Loss (SNHL), such as the AAVAnc80-hOTOF trial, involves using adeno-associated virus (AAV) vectors to deliver functional copies of the otoferlin gene to the inner ear. Otoferlin is crucial for the proper functioning of hair cells in the cochlea, which are responsible for converting sound waves into electrical signals for the brain. By restoring the expression of otoferlin, this therapy aims to correct the underlying genetic defect causing SNHL, potentially improving or restoring hearing. This approach is significant for SNHL patients as it targets the root cause of hearing loss rather than just alleviating symptoms, offering a promising avenue for long-term treatment.