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.
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)
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).
Sequential
SENS-501 administration
Combination productAdministration of SENS-501 with a dedicated administration system
Safety and tolerability of SENS-501
Number of study-related adverse events (AEs) and serious adverse events (SAEs).
Efficacy of SENS-501 assessed by ABR
Improvement of hearing threshold measured by auditory brainstem response (ABR)
Safety and tolerability of SENS-501
Number of study-related adverse events (AEs) and serious adverse events (SAEs).
Efficacy of SENS-501 assessed by ABR
Improvement of hearing threshold measured by auditory brainstem response (ABR)
Efficacy of SENS-501 assessed by PTA
Improvement of hearing threshold measured by Pure-tone audiometry (PTA)
Clinical performance of the administration system
Administration success ; Users' feedback questionnaire on the administration and use of the devices
