In short
A new drug called DB-OTO, which contains DB-OTO-5, is being tested in clinical trials for children and infants with genetic hearing loss. This trial aims to evaluate the safety, tolerability, and effectiveness of DB-OTO in patients with a specific genetic condition affecting their hearing. The study focuses on individuals with mutations in the otoferlin gene (OTOF) and explores the potential of gene therapy to improve hearing in these patients.
At a glance
- Drug Name
- DB-OTO (containing DB-OTO-5 and DB-OTO-3)
- Trial Phase
- Phase 1/2
- Target Condition
- Congenital Hearing Loss due to Otoferlin Gene Mutations
- Participant Age Range
- Under 18 years old
- Administration Method
- Intracochlear injection
- Primary Endpoint
- Safety and tolerability assessment
- Secondary Endpoints
- Changes in hearing thresholds and speech perception
- Follow-up Period
- 18 months
What is DB-OTO?
DB-OTO is an experimental gene therapy designed to treat a specific type of genetic hearing loss. It's being developed by Decibel Therapeutics Inc. to help children and infants who have hearing problems caused by mutations in a gene called otoferlin (OTOF). This therapy is given as an injection directly into the inner ear (intracochlear injection).
How Does DB-OTO Work?
DB-OTO uses a combination of two special components called DB-OTO-5 and DB-OTO-3. These components are delivered using a harmless virus called AAV1 (adeno-associated virus type 1). Here's a simple breakdown of how it works:
- DB-OTO-5 carries the first part of the healthy otoferlin gene.
- DB-OTO-3 carries the second part of the healthy otoferlin gene.
- These components are injected into the inner ear.
- The healthy gene helps produce the otoferlin protein that's missing in patients with this type of hearing loss.
This approach is called gene therapy, which means it aims to fix the underlying genetic cause of the hearing loss.
Who Can Benefit from DB-OTO?
DB-OTO is specifically designed for children and infants who have hearing loss due to mutations in both copies of their otoferlin gene. This condition is called congenital hearing loss secondary to biallelic mutations of the otoferlin gene (OTOF). It's important to note that this therapy is not for all types of hearing loss, but only for this specific genetic cause.
Clinical Trial Details
A clinical trial called CHORD is currently underway to test DB-OTO. Here are some key points about the trial:
- It's a Phase 1/2 trial, which means it's an early-stage study to test both safety and how well the treatment works.
- The trial is open-label, meaning both the researchers and participants know which treatment is being given.
- It's being conducted at multiple medical centers.
- The study will test different doses of DB-OTO to find the best one.
- Some participants will receive the treatment in one ear first, and then potentially in both ears later.
Eligibility Criteria
Not everyone with hearing loss can participate in this trial. Here are some of the main criteria for joining:
- Participants must have mutations in both copies of their OTOF gene.
- They must be under 18 years old.
- They must have severe to profound hearing loss, as measured by specific hearing tests.
- They must not have had cochlear implants in the ear(s) that will receive DB-OTO.
- They must not have certain other medical conditions or history of treatments that could interfere with the study.
It's important to note that there are additional criteria that a doctor would need to check to determine if someone is eligible for the trial.
Safety and Effectiveness
The main goals of this clinical trial are to:
- Evaluate the safety and tolerability of DB-OTO in children and infants with OTOF-related hearing loss.
- Look for early signs that the treatment is working.
To measure effectiveness, the researchers will use tests like:
- Auditory Brainstem Response (ABR): This test measures how well the auditory nerve responds to sounds.
- Behavioral audiometry: This includes tests to measure how well participants can hear different tones and recognize speech.
It's important to remember that DB-OTO is still experimental, and more research is needed to fully understand its safety and effectiveness. If you think your child might benefit from this treatment, it's best to discuss it with a healthcare provider who specializes in genetic hearing loss.