Gene Therapy Trial for OTOF-mediated Hearing Loss
Research type
Research Study
Full title
A Phase 1/2 Trial of AAVAnc80-hOTOF Gene Therapy in Individuals with Sensorineural Hearing Loss due to Biallelic Otoferlin Gene (OTOF) Mutations
IRAS ID
1007977
Contact name
Katie Wachtel
Contact email
Sponsor organisation
Akouos, Inc., a wholly owned subsidiary of Eli Lilly and Company
Clinicaltrials.gov Identifier
Research summary
OTOF-mediated hearing loss is caused by mutations in the otoferlin gene which prevents normal functioning of the ear and results in hearing loss.
Currently there are no medicinal treatment options available for OTOF-mediated hearing loss.
This study will investigate a method of gene therapy, where the study drug will be used to transfer a normal copy of the otoferlin gene into the ear by a single surgical procedure. An adeno-associated virus (AAV) which does not cause illness, will be used to carry the normal gene into the ear.
This study is split into Part A and Part B. During Part A participants will receive the study drug at one of two doses. The dose they receive will depend upon what point the study is at when the participant enrolls. The results of Part A will determine what dose level is used for Part B.
The aim of this study is to evaluate if the study drug is safe and if it improves hearing in individuals with OTOF-mediated hearing loss.
Approximately 14 participants ages 2 to 17 years old will take part in Part A of this study.
Each participant will be in the study for approximately 27 months. This includes a screening period of up to 3 months and a 24-month (2 year) observation period following administration of the study drug. Participants will be expected to participate in a separate long term follow-up protocol for up to five years after administration of the study drug.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0666
Date of REC Opinion
15 Nov 2023
REC opinion
Further Information Favourable Opinion