Study on SENS-501 for Children with Severe Hearing Loss Due to Otoferlin Gene Mutations

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What is this study about?

This clinical trial is focused on children with severe to profound hearing loss caused by mutations in the Otoferlin (OTOF) gene. The study is testing a new treatment called SENS-501, which is a type of gene therapy. Gene therapy involves introducing new genetic material into a person’s cells to treat or prevent disease. In this case, SENS-501 aims to restore hearing by delivering a specific gene sequence directly into the ear to help produce a protein that is missing or not working properly in these children.

The purpose of the study is to evaluate the safety and effectiveness of SENS-501. The treatment is given as a single injection into the cochlea, which is a part of the inner ear. The study is divided into two parts. The first part focuses on assessing the safety and how well children tolerate the treatment. The second part looks at how effective the treatment is in improving hearing. Children participating in the study will receive either the SENS-501 treatment or a placebo, and their hearing will be monitored over time to see if there are any improvements.

Throughout the study, researchers will keep track of any side effects or adverse events that occur. They will also measure changes in hearing ability using tests like the Auditory Brainstem Response (ABR) and Pure Tone Audiometry (PTA). The study will also evaluate the performance and usability of the devices used to deliver the treatment, such as the SPHYNX Pump and Intracochlear Catheter. The trial is expected to continue until the end of 2030, with recruitment starting at the end of 2023.

1 initial assessment

Upon joining the clinical trial, the child will undergo an initial assessment to confirm eligibility. This includes verifying the age requirement of 6 to 31 months and confirming severe to profound hearing loss due to Otoferlin gene mutations.

Additional tests will be conducted to ensure the presence of normal cochlea and internal auditory canals, as well as intact vestibular function.

2 preparation for treatment

Once eligibility is confirmed, preparations for the treatment will begin. This involves scheduling the procedure for the **unilateral intracochlear injection** of **SENS-501**.

The medical team will provide detailed instructions on how to prepare for the procedure, including any necessary pre-procedure guidelines.

3 treatment administration

The child will receive a **unilateral intracochlear injection** of **SENS-501**. This means the treatment will be administered into one ear.

The procedure is designed to assess the safety and tolerability of the treatment in the first part of the trial.

4 post-treatment monitoring

After the treatment, the child will be monitored for any adverse events or side effects. This monitoring is crucial to ensure the child’s safety and to gather data on the treatment’s effects.

Regular follow-up visits will be scheduled to assess the child’s hearing threshold and overall response to the treatment.

5 evaluation of treatment efficacy

In the second part of the trial, the focus will shift to evaluating the efficacy of the treatment. This involves measuring any improvements in the child’s hearing threshold.

The medical team will use specific tests to assess changes in hearing ability and gather feedback on the treatment’s impact.

6 completion of trial participation

Upon completion of the trial, a final assessment will be conducted to evaluate the overall outcomes and any long-term effects of the treatment.

The child will be provided with a summary of the trial results and any recommendations for future care or follow-up.

Who Can Join the Study?

Children, both boys and girls, who are between 6 to 31 months old at the time of joining the study.
Have severe to profound hearing loss, which is a very significant level of hearing loss, confirmed by a test called ABR (Auditory Brainstem Response).
Have a biallelic mutation in the Otoferlin gene, meaning there are changes in both copies of this specific gene that can affect hearing.
Have OAEs (Otoacoustic Emissions), which are sounds given off by the inner ear when the cochlea is working normally.
Have a normal cochlea and internal auditory canals, which are parts of the ear that are important for hearing.
Have intact vestibular function, meaning their balance system, which is part of the inner ear, is working properly.

Who Cannot Join the Study?

Patients with any other ear conditions that could affect hearing.
Patients who have had previous ear surgeries that might interfere with the study.
Patients with a history of allergies to medications used in the study.
Patients who are currently participating in another clinical trial.
Patients with any serious health conditions that could affect their participation.
Pregnant or breastfeeding women.
Patients who are unable to follow the study procedures or instructions.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Hopital Necker Enfants Malades	Paris	France

Other Sites

Site Name	City	Country	Status
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Ospedale Pediatrico Bambino Gesu’	Rome	Italy

Trial status

Country	Status	Recruitment Start
France	Not recruiting	31.12.2023
Germany	Not recruiting	31.12.2023
Italy	Not recruiting	31.12.2023

Trial locations

Investigated drugs:

SENS-501 is a treatment being tested for children who have severe to profound hearing loss due to specific gene mutations. This therapy involves injecting the medication directly into the cochlea, which is a part of the inner ear. The goal of this treatment is to see if it can help improve hearing by addressing the genetic cause of the hearing loss. The trial is designed to first check if the treatment is safe and can be tolerated by the patients. After that, the study will look at how effective the treatment is in improving hearing abilities.

Otoferlin Deficiency-Related Hearing Loss – This condition is characterized by bilateral severe to profound hearing loss due to a deficiency in the otoferlin protein. Otoferlin is crucial for the proper functioning of hair cells in the cochlea, which are responsible for converting sound waves into electrical signals for the brain. Without sufficient otoferlin, these hair cells cannot effectively transmit sound information, leading to significant hearing impairment. The progression of this hearing loss typically begins at birth or early childhood and remains stable over time. It primarily affects the ability to hear high-frequency sounds, making it difficult to understand speech and environmental noises. This condition is considered a rare disease.

Trial ID:

2023-504466-28-00

Protocol code:

SENS-501-101

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study on SENS-501 for Children with Severe Hearing Loss Due to Otoferlin Gene Mutations

What is this study about?

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