SENS-501 administration: Administration of SENS-501 with a dedicated administration system
Study 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.
Eligibility
Sex
ALL
Min age
6 Months
Max age
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
Primary outcome measure(s)
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)
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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