Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
1 other identifier
interventional
22
4 countries
9
Brief Summary
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, together with the Precision Delivery Mechanism, to safely achieve the intended product performance.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Sep 2023
Longer than P75 for phase_1
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 8, 2023
CompletedFirst Posted
Study publicly available on registry
April 20, 2023
CompletedStudy Start
First participant enrolled
September 15, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 1, 2028
March 4, 2026
December 1, 2025
5 years
February 8, 2023
March 3, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Frequency of Adverse Events (AEs)
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system)
through trial completion, approximately 1 year
Secondary Outcomes (2)
Auditory Brainstem Response (ABR) Threshold
through trial completion, approximately 1 year
Performance of the Akouos Delivery Device and Precision Delivery Mechanism
through trial completion, approximately 1 year
Study Arms (3)
Cohort 1a and Cohort 1b
EXPERIMENTALCohort 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
Cohort 2
EXPERIMENTALCohort 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
Bilateral Expansion
EXPERIMENTALBilateral Expansion: up to eight eligible participants of any age, and no cochlear implants, may be eligible for enrollment into the Bilateral Expansion Cohort (simultaneous bilateral administration of AAVAnc80-hOTOF).
Interventions
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time bilateral intracochlear administration using the Akouos Delivery Device together with the Precision Delivery Mechanism.
Eligibility Criteria
You may qualify if:
- Participants may be of any age, based on Cohort Criteria
- At least two mutations in the otoferlin gene
- Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
- Preserved distortion product otoacoustic emissions (DPOAEs)
- 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
You may not qualify if:
- 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
- Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Akouos, Inc.lead
- Eli Lilly and Companycollaborator
Study Sites (9)
University of California, San Francisco Benioff Children's Hospital
San Francisco, California, 94158, United States
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
The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683
Toronto, Ontario, M5G 1X8, Canada
National Taiwan University Hospital
Taipei, 100, Taiwan
University College London
London, United Kingdom, NW1 2PG, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Peter Marks, MD
Eli Lilly and Company
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 8, 2023
First Posted
April 20, 2023
Study Start
September 15, 2023
Primary Completion (Estimated)
October 1, 2028
Study Completion (Estimated)
October 1, 2028
Last Updated
March 4, 2026
Record last verified: 2025-12