Gene therapy for hearing loss caused by otoferlin gene mutations

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial aims to evaluate the safety and tolerability of a gene therapy treatment for profound bilateral sensorineural hearing loss caused by mutations in the otoferlin gene. Participants will receive a single unilateral administration of one of two dose levels of the AAVAnc80-hOTOF gene therapy via the Akouos delivery device. The study will assess the effectiveness of this approach in restoring hearing function in affected individuals. The trial is divided into two phases, focusing on different dose levels to determine the optimal treatment strategy.

Why it matters

Who can join: the full rules

Criteria for Inclusion:

1. Participants may be of any age, based on Cohort Criteria
2. At least two mutations in the otoferlin gene
3. Clinical presentation of Profound (Part A) or Severe to Profound (Parts B, C, and D) bilateral sensorineural hearing loss as assessed by ABR
4. Preserved distortion product otoacoustic emissions (DPOAEs)
5. Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial

Criteria for Exclusion:

1. Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy
2. Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
3. Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial (with the exception of AK-OTOF-101 for Part D participants)

Where it's running

• University of California, San Francisco Benioff Children's Hospital
  San Francisco, California, United States Recruiting
• University of Iowa
  Iowa City, Iowa, United States Recruiting
• Vanderbilt Bill Wilkerson Center
  Nashville, Tennessee, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Active not recruiting
• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Active not recruiting
• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Active not recruiting

Study contacts

• Study coordinator: Lilly Clinical Trials
• Email: AkouosClinicalTrials@Lilly.com
• Phone: +1 857-410-1816

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Comparing speech understanding methods for cochlear implant users

  This study tests whether a new way of setting up cochlear implants helps adults understand speech better in noisy places compared to the traditional method.

  Recruiting MED-EL Elektromedizinische Geräte GmbH · Dijon · NCT06737185

• Comparing speech perception with different cochlear implant settings

  This study tests whether different settings on cochlear implants can help adults hear and understand speech better in noisy situations.

  Recruiting MED-EL Elektromedizinische Geräte GmbH · Limoges · NCT06723262

• Comparing two fitting strategies for cochlear implants in patients with hearing loss

  This study is testing two different ways to set up cochlear implants for people with hearing loss to see which one helps them understand speech better in noisy places.

  Recruiting MED-EL Elektromedizinische Geräte GmbH · Saint-Etienne · NCT05955469

• Comparing two fitting strategies for cochlear implants in patients with hearing loss

  This study is testing two different ways to set up cochlear implants and hearing aids together to see which helps adults with hearing loss hear better in noisy situations.

  Recruiting MED-EL Elektromedizinische Geräte GmbH · Nice · NCT05898659

• Study of hearing loss related to the Otoferlin gene

  This study looks at how hearing loss caused by changes in the Otoferlin gene affects people over time, focusing on those with specific types of hearing problems.

  Recruiting at 9 sites Akouos, Inc. · Iowa City, Iowa and 9 other locations · NCT05572073

• Tracking hearing loss in children with specific genetic mutations

  This study is tracking children with hearing loss caused by specific genetic mutations to see how their condition changes over time and how it affects their quality of life.

  Recruiting Sensorion · Paris · NCT05402813

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.