Treatment for children with hearing loss due to Otoferlin mutations

A Phase I/II, Open-ended, Adaptative, Open Label Dose Escalation and Expansion Clinical Trial to Evaluate the Efficacy and Safety of Unilateral Intracochlear Injection of SENS-501 Using an Injection System in Children with Severe to Profound Hearing Loss Due to Otoferlin Gene Mutations

This study is testing a new treatment called SENS-501 to see if it can help young children with severe hearing loss caused by Otoferlin gene mutations improve their hearing.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 6 Months to 31 Months
• Sex: All
• Sponsor: Sensorion (industry)
• Locations: 2 sites (Westmead and 1 other locations)
• Trial ID: NCT06370351 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety, tolerability, and efficacy of SENS-501 in children aged 6 to 31 months who have severe to profound hearing loss caused by mutations in the Otoferlin gene. The study employs a multicenter, adaptive, open-label design with a non-randomized, dose-escalation approach. Participants will receive intracochlear administration of SENS-501, and the trial aims to gather data on the treatment's effects on hearing restoration.

Who should consider this trial

Good fit: Ideal candidates are children aged 6 to 31 months with severe to profound hearing loss due to biallelic mutations in the Otoferlin gene.

Not a fit: Patients with a history of cochlear implants or those who have participated in previous gene therapy trials may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve hearing capabilities in children with specific genetic hearing loss.

How similar studies have performed: While gene therapy approaches for hearing loss are emerging, this specific application targeting Otoferlin mutations is relatively novel and has not been extensively tested in prior studies.

Eligibility criteria

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

Where this trial is running

Westmead and 1 other locations

• Childrens Hospital Westmead — Westmead, Australia (RECRUITING)
• Hopital Necker Enfants Malades — Paris, France (RECRUITING)

Study contacts

• Study coordinator: Lionel HOVSEPIAN, MD

Conditions: OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders, Ear Diseases, Otorhinolaryngologic Diseases, Deafness, Hearing Loss, Sensorineural