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Db-Oto-5

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.

Table of Contents

What is DB-OTO?

DB-OTO is an experimental gene therapy designed to treat a specific type of genetic hearing loss[1]. 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)[1]. This therapy is given as an injection directly into the inner ear (intracochlear injection)[1].

How Does DB-OTO Work?

DB-OTO uses a combination of two special components called DB-OTO-5 and DB-OTO-3[1]. These components are delivered using a harmless virus called AAV1 (adeno-associated virus type 1). Here’s a simple breakdown of how it works:

  1. DB-OTO-5 carries the first part of the healthy otoferlin gene.
  2. DB-OTO-3 carries the second part of the healthy otoferlin gene.
  3. These components are injected into the inner ear.
  4. 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[1].

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[1]. 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[1]. 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[1]:

  • 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[1]:

  1. Evaluate the safety and tolerability of DB-OTO in children and infants with OTOF-related hearing loss.
  2. Look for early signs that the treatment is working.

To measure effectiveness, the researchers will use tests like[1]:

  • 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.

Aspect Details
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

FAQ

  • What is DB-OTO and how does it work? DB-OTO is a gene therapy product consisting of two components (DB-OTO-5 and DB-OTO-3) that work together to deliver a functional copy of the otoferlin gene to the inner ear. It aims to treat hearing loss caused by mutations in the otoferlin gene.
  • Who is eligible for this clinical trial? The trial is open to children and infants under 18 years of age who have mutations in both copies of the OTOF gene and meet specific hearing test criteria. Participants must not have other permanent hearing loss conditions or have received cochlear implants in the ears to be treated.
  • How is DB-OTO administered? DB-OTO is administered through an intracochlear injection, which means it is injected directly into the inner ear (cochlea). The trial includes a single ascending dose cohort followed by a bilateral injection expansion cohort.
  • What are the main goals of this clinical trial? The primary goal is to evaluate the safety and tolerability of DB-OTO in children and infants with otoferlin gene mutations. Secondary goals include assessing the drug's effectiveness in improving hearing function.
  • How long does the trial last and what kind of follow-up is involved? The trial includes a short-term follow-up period of 18 months. During this time, participants will undergo various tests to monitor their hearing function and any potential side effects of the treatment.

Ongoing Clinical Trials on Db-Oto-5

  • Study on DB-OTO for Safety and Tolerability in Infants and Children with Genetic Hearing Loss

    Recruiting

    2 1 1
    Investigated drugs:
    Germany Spain

Glossary

  • Otoferlin gene (OTOF): A gene that provides instructions for making a protein called otoferlin, which is important for proper hearing function. Mutations in this gene can cause hearing loss.
  • Gene therapy: A treatment approach that involves introducing genetic material into a person's cells to treat or prevent disease. In this case, it's used to provide a functional copy of the otoferlin gene.
  • Intracochlear injection: A method of delivering medication directly into the cochlea, which is part of the inner ear responsible for hearing.
  • Auditory brainstem response (ABR): A test that measures the brain's response to sound and helps determine a person's hearing threshold.
  • Otoacoustic emissions (OAE): Sounds generated by the inner ear in response to stimuli, which can be measured to assess hearing function.
  • Cochlear microphonic: An electrical signal generated by the hair cells in the cochlea in response to sound, which can be measured to assess inner ear function.
  • Adeno-associated viral vector (AAV): A modified virus used to deliver genetic material into cells for gene therapy purposes. In this trial, AAV1 is used to carry the otoferlin gene components.

References

  1. http://clinicaltrials.eu/trial/study-on-db-oto-for-safety-and-tolerability-in-infants-and-children-with-genetic-hearing-loss/