A Phase 1/2, open label study of DB-OTO in children/infants with a biallelic otoferlin gene mutation
Research type
Research Study
Full title
A Phase 1/2, Open-Label, Multicenter Trial With a Single Ascending Dose Cohort With Unilateral Intracochlear Injection Followed by a Bilateral Injection Expansion Cohort to Evaluate the Safety, Tolerability, and Efficacy of DB-OTO in Children and Infants With Biallelic hOTOF Mutations
IRAS ID
1005935
Contact name
Penny Logan
Contact email
Sponsor organisation
Decibel Therapeutics
Eudract number
2022-000079-38
Clinicaltrials.gov Identifier
Research summary
This is a phase 1/2 study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat young patients (patients will be enrolled at 2 years or under), who have hearing loss due to changes in the otoferlin gene. Patients with this diagnosis may be born with profound hearing loss and are not expected to improve without treatment. Gene therapies use modified viruses (DB-OTO is a modified “adeno-associated virus”, AAV) to deliver gene to parts of the body. In the case of DB-OTO it is delivering a working copy of otoferlin to the inner ear. The purpose of this study is to learn about the safety and effectiveness of DB-OTO and if it is tolerated in children. There are 2 parts to this study, the first (Part A) is dose escalation, where children will get 1 of 2 dose level of DB-OTO (a single intra-cochlear injection in one ear). If that is well tolerated, then the next group will get the second dose level of DB-OTO. Additional doses may be explored to select the best tolerated dose to be used in Part B where DB-OTO will be given as a single intra-cochlear injection in each ear. DB-OTO is injected under anaesthesia. Up to 18 children will be enrolled in study sites around the world. Participants will be followed for 5 years after dosing with DB-OTO to evaluate short- and long-term safety, tolerability, and efficacy. There will be a number of assessments (medical history, physical exams, vital signs, blood/urine samples, saliva, urine and stool samples for viral shedding, blood sample for antibody assays, blood distribution of DB-OTO, genetic testing, a number of different ear examinations /hearing tests and questionnaires) to be completed.
REC name
North East - York Research Ethics Committee
REC reference
22/NE/0227
Date of REC Opinion
9 Jan 2023
REC opinion
Further Information Favourable Opinion