Gene Therapy for Hearing Loss
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial coordinators for more details.
Is the gene therapy for hearing loss using AAV vectors safe in humans?
What makes the treatment AAVAnc80-hOTOF unique for hearing loss?
What data supports the effectiveness of the treatment AAVAnc80-hOTOF for hearing loss?
Research shows that similar gene therapy approaches using AAV vectors have been effective in restoring hearing in animal models with hearing loss due to OTOF gene mutations. The AAVAnc80 vector has been shown to efficiently target hair cells in the inner ear, which is crucial for hearing restoration.12346
Who Is on the Research Team?
Kathy Reape, MD
Principal Investigator
Akouos, Inc.
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single unilateral administration of AAVAnc80-hOTOF using an investigational medical device
Follow-up
Participants are monitored for safety, tolerability, and device performance, including auditory brainstem response threshold changes
What Are the Treatments Tested in This Trial?
Interventions
- AAVAnc80-hOTOF
- Akouos Delivery Device
Find a Clinic Near You
Who Is Running the Clinical Trial?
Akouos, Inc.
Lead Sponsor
Eli Lilly and Company
Industry Sponsor
Dr. Daniel Skovronsky
Eli Lilly and Company
Chief Medical Officer since 2018
MD from Harvard Medical School
David A. Ricks
Eli Lilly and Company
Chief Executive Officer since 2017
BSc from Purdue University, MBA from Indiana University