A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations

Phase 1

Not yet recruiting

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

• Registration Number: NCT06370351
• Lead Sponsor: Sensorion

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.

Study Timeline

• Status Verified: 2024-04
• Study First Submitted: 2024-04-09
• Study First Submitted QC: 2024-04-12
• Last Update Submitted: 2024-04-12
• Study First Posted: 2024-04-17
• Last Update Posted: 2024-04-17
• Study Start: 2024-04-30
• Primary Completion: 2027-04
• Study Completion: 2031-04

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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 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
Efficacy of SENS-501 assessed by ABR	5 years	Improvement of hearing threshold measured by auditory brainstem response (ABR)

Trial Locations

Locations (2)

Childrens Hospital Westmead; 🇦🇺 Westmead, Australia

Hopital Necker Enfants Malades; 🇫🇷 Paris, France