Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
NCT ID: NCT05821959
Last Updated: 2026-01-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
22 participants
INTERVENTIONAL
2023-09-15
2028-10-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
NCT06696456
Otoferlin Gene-mediated Hearing Loss Natural History Study
NCT05572073
An Investigator Initiated Study for OTOV101N+OTOV101C Injection
NCT05901480
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
NCT05788536
A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
NCT06370351
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
* Up to 4.1E11 total vg/cochlea
* Up to 8.1E11 total vg/cochlea
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Cohort 1a and Cohort 1b
Cohort 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
AAVAnc80-hOTOF via Akouos Delivery Device
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
Cohort 2
Cohort 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
AAVAnc80-hOTOF via Akouos Delivery Device
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
Bilateral Expansion
Bilateral 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).
AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
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.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
AAVAnc80-hOTOF via Akouos Delivery Device
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
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
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. At least two mutations in the otoferlin gene
3. Clinical presentation of Profound 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
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Eli Lilly and Company
INDUSTRY
Akouos, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Kathy Reape, MD
Role: STUDY_DIRECTOR
Akouos, Inc.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of California, San Francisco Benioff Children's Hospital
San Francisco, California, United States
University of Iowa
Iowa City, Iowa, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt Bill Wilkerson Center
Nashville, Tennessee, United States
The Hospital for Sick Children [email protected] +1 (416) 813 6683
Toronto, Ontario, Canada
National Taiwan University Hospital
Taipei, , Taiwan
University College London
London, United Kingdom, United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Role: primary
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
AK-OTOF-101
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.