Congenital Deafness, Deafness, DFNB9, Ear Diseases, Hearing Disorders, Hearing Loss, Sensorineural, OTOF Gene Mutation, Otorhinolaryngologic Diseases
Conditions
Keywords
OTOF Gene Mutation, Hearing impairment, Deafness, otoferlin, AUDIOGENE, Hearing Loss, DFNB9, Hearing disorder, Cochlear implant, SENS-501, Congenital Hearing loss, Gene therapy
Brief summary
This study intends to assess safety, tolerability, and efficacy of SENS-501 in children between the ages of 6-31 months with pre-lingual hearing loss due to a mutation in the Otoferlin gene.
Detailed description
It is a multicenter, adaptive open-label, non-randomized, dose-escalation, and expansion study to assess safety, tolerability, and efficacy following intracochlear administration of SENS-501.
Interventions
COMBINATION_PRODUCTSENS-501 administration
Administration of SENS-501 with a dedicated administration system
Sponsors
Sensorion
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 31 Months
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability of SENS-501 | 5 years | Number of study-related adverse events (AEs) and serious adverse events (SAEs). |
| Efficacy of SENS-501 assessed by ABR | 5 years | Improvement of hearing threshold measured by auditory brainstem response (ABR) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability of SENS-501 | 5 years | Number of study-related adverse events (AEs) and serious adverse events (SAEs). |
| Efficacy of SENS-501 assessed by ABR | 5 years | Improvement of hearing threshold measured by auditory brainstem response (ABR) |
| Efficacy of SENS-501 assessed by PTA | 5 years | Improvement of hearing threshold measured by Pure-tone audiometry (PTA) |
| Clinical performance of the administration system | 1 day | Administration success ; Users' feedback questionnaire on the administration and use of the devices |
| Safety of the administration system | 5 years | Adverse device effects and Device deficiencies, Procedure complications |
| Usability of the administration system | 1 day | Usability questionnaire |
Countries
Australia, France
Contacts
STUDY_CHAIRNatalie LOUNDON, Pr
Hopital Necker Enfants Malades
Outcome results
None listed