RECRUITING

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

Part A of this trial will evaluate the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos Delivery Device to safely achieve the intended product performance.

Official Title

A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations

Quick Facts

Study Start:2023-09-15
Study Completion:2028-10
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05821959

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Akouos Clinical Trials
CONTACT
+1 857 410-1816
AkouosClinicalTrials@Lilly.com

Principal Investigator

Kathy Reape, MD
STUDY_DIRECTOR
Akouos, Inc.

Study Locations (Sites)

University of Iowa
Iowa City, Iowa, 52242
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146
United States
Vanderbilt Bill Wilkerson Center
Nashville, Tennessee, 37232
United States

Collaborators and Investigators

Sponsor: Akouos, Inc.

  • Kathy Reape, MD, STUDY_DIRECTOR, Akouos, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-09-15
Study Completion Date2028-10

Study Record Updates

Study Start Date2023-09-15
Study Completion Date2028-10

Terms related to this study

Keywords Provided by Researchers

  • Otoferlin
  • Auditory neuropathy (AN)
  • Deafness, autosomal recessive 9 (DFNB9)
  • Hearing loss
  • Hearing loss, bilateral
  • Hearing loss, sensorineural

Additional Relevant MeSH Terms

  • Sensorineural Hearing Loss, Bilateral