Gene Therapy for Congenital Hearing Loss
(CHORD Trial)
Trial Summary
What is the purpose of this trial?
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)
Will I have to stop taking my current medications?
The trial information does not specify whether participants must stop taking their current medications. It is best to discuss this with the trial coordinators or your doctor.
What data supports the effectiveness of the treatment DB-OTO for congenital hearing loss?
Research shows that gene therapy, like DB-OTO, has successfully restored hearing in animal models with genetic forms of deafness. For example, a study using a similar approach with dual AAV vectors restored hearing in mice with a specific type of genetic deafness, suggesting potential for human treatment.12345
How is the treatment DB-OTO unique for congenital hearing loss?
DB-OTO is a novel gene therapy that uses a dual AAV (adeno-associated virus) approach to deliver the otoferlin gene, which is too large for a single AAV vector, directly into the cochlea. This method aims to restore hearing by addressing the genetic cause of deafness, unlike traditional treatments like cochlear implants that only bypass the damaged parts of the ear.12367
Eligibility Criteria
This trial is for children under 18 with profound sensorineural hearing loss due to OTOF gene mutations, who meet cochlear implant criteria and have not benefited from ear amplification. They must not have had previous gene therapy or cochlear implants in the affected ear(s), nor other untreatable hearing conditions.Inclusion Criteria
Exclusion Criteria
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Participants receive unilateral intracochlear dosing to evaluate safety and tolerability
Dose Expansion
Participants receive bilateral intracochlear dosing using the dose selected based on safety and efficacy data from the Dose Escalation phase
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- DB-OTO (Gene Therapy)