RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

Regeneron is running a study called DB-OTO, a new type of treatment using gene therapy for children and babies with hearing loss. This hearing loss is due to a specific problem with a gene called otoferlin. The main goals of the study are to understand if DB-OTO is safe to use and if it causes any discomfort. Researchers also want to find out how well this therapy works to improve hearing. This is an early-stage study (Phase 1/2), meaning it's one of the first times this treatment is being tested in humans. The hope is to find a new way to help children whose hearing loss is caused by this particular genetic condition.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Regeneron Pharmaceuticals

Enrolment target

Start

27 Jun 2023

Estimated completion

28 Feb 2032

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

What is this study about?

Imagine hearing loss caused by a tiny blueprint error in your body – a change in a specific gene called otoferlin. This study is looking into a new treatment called DB-OTO, which is a type of 'gene therapy'. Think of gene therapy as trying to fix or replace that blueprint error. The researchers are hoping this treatment could help children and babies who have profound hearing loss because of this specific gene problem. This is an important step because it could offer a new way to help these children hear.

The main idea behind this study is to see if DB-OTO is safe and if it helps improve hearing. It's an early-stage study, meaning it's among the first times this new treatment is being tested in people. The medical team will carefully watch how participants react to the treatment and measure any changes in their hearing. They want to make sure it's not only safe but also actually makes a difference.

This research is being conducted by Regeneron, a company that develops new medicines. While it's an exciting area of science, it's important to remember that gene therapies are complex and still being understood. The study aims to gather crucial information that could, in the future, help many children with this specific type of hearing loss.

Key takeaways

This study is for children under 18 with specific genetic hearing loss.
It's testing a new gene therapy called DB-OTO.
The main goals are to check safety and how well it improves hearing.
Participation involves medical checks, the treatment, and follow-up visits.
There are potential benefits, but also potential risks, as it's a new treatment.

Who may be eligible?

To be considered for this study, the child must be under 18 years old. A parent or legal guardian needs to agree to the study rules, including genetic testing for the child to confirm the specific otoferlin gene change causing their hearing loss. The child also needs to be up-to-date with their routine childhood vaccinations.

The child must have severe hearing loss in both ears (profound sensorineural hearing loss, meaning it's due to a problem with the inner ear) due to changes in the otoferlin gene. However, there must still be certain healthy cells in the inner ear, which doctors will check using special tests. Also, the child needs to be well enough overall, with no significant health problems that would make it unsafe to take part.

For female patients old enough to have children and male patients who can father children, effective birth control methods must be used for a certain time during and after the study. This is to prevent pregnancy during the study period.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my child under 18 years old?
Does my child have profound hearing loss in both ears?
Has a doctor confirmed my child's hearing loss is due to the otoferlin gene?
Is my child healthy enough to take part in medical procedures?
Am I, as a parent/guardian, willing to agree to the study rules and tests?

Answer every question to see your result.

What does participation involve?

If you decide your child might take part, there will be several visits to the hospital or clinic. These visits will involve various check-ups, hearing tests, and possibly blood tests. The DB-OTO treatment will likely be given once, and then your child will be monitored closely for a period of time to see how they respond. This might include regular follow-up appointments and more tests to track their hearing and overall health.

The study has a "short-term follow-up period" of 48 weeks, which is about 11 months. During this time, your child will have regular appointments and checks. The exact number of visits and tests will be explained by the study team, but they are designed to carefully monitor safety and how well the treatment is working. Doctors will also need to review your child's medical history and current health before they can join.

Potential risks and benefits

Participating in this study might offer your child the chance to try a brand-new treatment that could potentially improve their hearing, which is a significant potential benefit. However, because DB-OTO is a new treatment, there could be unknown side effects or risks, and it might not work for everyone. The medical team will explain all known potential risks. Remember, taking part in any study is entirely voluntary, and you have the right to withdraw your child at any time, for any reason, without it affecting their usual medical care.

Locations (16)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of California Los Angeles Medical Center
Verified postcode
Los Angeles, United States· Recruiting
Rady Children's Hospital
Verified postcode
San Diego, United States· Recruiting
Nemours Children s Clinic
Verified postcode
Jacksonville, United States· Recruiting
Nemours Childrens Hospital
Verified postcode
Orlando, United States· Recruiting
Boston Children's Hospital - Main
Verified postcode
Boston, United States· Recruiting
Columbia University Irving Medical Center
Verified postcode
New York, United States· Recruiting
Cincinnati Children's Hospital Medical Center
Verified postcode
Cincinnati, United States· Recruiting
Seattle Children's Hospital
Verified postcode
Seattle, United States· Recruiting
Medical College of Wisconsin
Verified postcode
Milwaukee, United States· Recruiting
University Hospital Tubingen
Verified postcode
Tübingen, Germany· Recruiting
Shinshu University Hospital
Unverified
Matsumoto-shi, Japan· Recruiting
Clinica Universidad de Navarra- Pamplona
Verified postcode
Pamplona, Spain· Recruiting

Common questions

What is gene therapy?

Gene therapy is a treatment that aims to fix or replace a faulty gene in your body that's causing a health problem, like hearing loss in this case.

What does 'otoferlin gene' mean?

The otoferlin gene is like an instruction manual in your body that helps your ears send sound signals to your brain. If there's a change in this gene, it can lead to hearing loss.

Will my child's hearing definitely improve?

This is a study to find out if the treatment works. We can't guarantee hearing improvement, but the aim is to see if it makes a positive difference.

How long does the study last?

The main monitoring period after treatment is about 48 weeks (around 11 months), with ongoing follow-up as needed.

Can children with any type of hearing loss join?

No, this study is only for children whose hearing loss is specifically caused by changes in the отоferlin gene.

How to find out more

Clinical Trials Administrator

clinicaltrials@regeneron.com 844-734-6643 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.