Actively Recruiting
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
Led by Regeneron Pharmaceuticals · Updated on 2026-05-05
30
Participants Needed
16
Research Sites
452 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat children who have hearing loss due to changes in the otoferlin gene. The purpose of this study is to: * Learn about the safety of DB-OTO * Determine how well DB-OTO is tolerated (does not cause ongoing discomfort) * Evaluate the efficacy of DB-OTO (how well DB-OTO works)
CONDITIONS
Official Title
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
Who Can Participate
Eligibility Criteria
You may qualify if you...
- At least one parent or legal guardian is willing to provide written informed consent and the patient to provide assent when applicable
- Willingness to comply with the study protocol, including genetic testing and vaccinations as required
- Patient is under 18 years old and able to complete necessary assessments for enrollment and dosing
- Presence of biallelic, likely pathogenic or pathogenic otoferlin (OTOF) gene variants
- No clinically significant abnormal laboratory test results at screening
- Profound sensorineural hearing loss (>90 dB HL) diagnosed by behavioral and physiological tests
- Outer hair cell presence confirmed by otoacoustic emissions or cochlear microphonic depending on age
- No evidence that hearing loss depends on body temperature
- Female patients of childbearing potential and fertile males must agree to use effective contraception during short-term follow-up and for specified periods after treatment
You will not qualify if you...
- Prior treatment with gene therapy
- Surgical anatomy that prevents or affects the planned ear injection
- Other permanent or untreatable hearing loss conditions
- History or presence of malignancies
- History or presence of meningitis
- History or presence of cochlear implants in the ear(s) to be treated
- History of risk factors for auditory neuropathy not caused by otoferlin mutations, including prematurity, low birth weight, hyperbilirubinemia, NICU admission, or low Apgar scores
- Other protocol-defined exclusion criteria
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 16 locations
1
University of California Los Angeles Medical Center
Los Angeles, California, United States, 90024
Actively Recruiting
2
Rady Children's Hospital
San Diego, California, United States, 92123
Actively Recruiting
3
Nemours Children s Clinic
Jacksonville, Florida, United States, 32207
Actively Recruiting
4
Nemours Childrens Hospital
Orlando, Florida, United States, 32827
Actively Recruiting
5
Boston Children's Hospital - Main
Boston, Massachusetts, United States, 02115
Actively Recruiting
6
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
7
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
8
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
9
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
10
University Hospital Tubingen
Tübingen, Germany, 72076
Actively Recruiting
11
Shinshu University Hospital
Matsumoto-shi, Nagano, Japan, 390-8621
Actively Recruiting
12
Clinica Universidad de Navarra- Pamplona
Pamplona, Navarre, Spain, 31008
Actively Recruiting
13
Hospital Universitario Materno Infantil en las Palmas de Gran Canaria
Las Palmas de Gran Canaria, Spain, 35016
Withdrawn
14
Ramon y Cajal University Hospital
Madrid, Spain, 28034
Actively Recruiting
15
Addenbrooke's Hospital, Cambridge University Hospitals NHS FT
Cambridge, United Kingdom, CB2 0QQ
Actively Recruiting
16
Great Ormond Street Hospital For Children NHS Foundation Trust
London, United Kingdom, WC1N 3JH
Actively Recruiting
Research Team
C
Clinical Trials Administrator
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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