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One-Time injection aims to restore hearing in children born deaf

NCT ID NCT06370351

Cure Sponsor: Sensorion Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 08, 2026 · Last updated Jul 09, 2026 · Updated 1 time

Summary

This trial tests a gene therapy called SENS-501 for children aged 6 to 31 months who are severely or profoundly deaf due to mutations in the otoferlin gene. The therapy is injected directly into one inner ear to deliver a working copy of the gene. The goal is to restore natural hearing, measured by brain response tests and standard hearing tests. The study also closely monitors safety and side effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
SENS-501 (gene therapy)
What this could lead to
If successful, this could provide a one-time treatment to restore natural hearing in children born deaf due to otoferlin mutations, potentially eliminating the need for hearing aids or cochlear implants.
What could go wrong
This is an early-phase trial with only 12 children, so safety and effectiveness are not yet proven. The injection procedure carries risks, and the therapy may not work for all participants or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2024

Expected to finish

Jul 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 31 months

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Children (male or female) ≥ 6 to ≤ 31 months at the time of inclusion * Severe to profound hearing loss assessed by auditory brainstem response (ABR) * Biallelic mutation in the Otoferlin gene * Presence of Otoacoustic emissions (OAEs) * Documented normal cochlea and internal auditory canals * Patients with intact vestibular function Exclusion Criteria: * History of chronic, acute, or major disease, or unspecified reasons, that in the opinion of the Investigator, makes the participant unsuitable for participation in the study or constitutes an unacceptable risk. * Have been dosed in a previous gene therapy clinical trial * Patients with a prior or current cochlear implant * Any contraindication to the surgery determined by the surgeon or anesthesia determined by the anesthesiologist, or designee, or history of therapy known as ototoxic (e.g., cisplatin, high dose and long treatment with aminoglycosides, etc.) for an extended period (more than 2 weeks). * Participation in any other interventional clinical trial * Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful completion of the study or might interfere with the evaluation of study treatment * Anticipated noncompliance with the protocol requirements

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Conditions

The condition(s) this trial relates to.

autosomal recessive nonsyndromic hearing loss 9 Deafness Deafness, Autosomal Recessive 9 disorder of ear Ear Diseases hearing disorder hearing loss disorder Hearing Loss, Sensorineural nonsyndromic genetic hearing loss otorhinolaryngologic disease sensorineural hearing loss disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Congenital deafness Deafness DFNB9 Ear diseases Hearing disorders Hearing loss, sensorineural Otof gene mutation Otorhinolaryngologic diseases

Contacts and locations

Locations

  • Childrens Hospital Westmead

    Westmead, NSW 2145, Australia

  • Hopital Necker Enfants Malades

    Paris, 75015, France

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