Clinical trial NCT06370351: A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: OTOF Gene Mutation
DFNB9
Congenital Deafness
Hearing Disorders
Ear Diseases
Otorhinolaryngologic Diseases
Deafness
Hearing Loss, Sensorineural
Keywords: OTOF Gene Mutation
Hearing impairment
Deafness
otoferlin
AUDIOGENE
Hearing Loss
DFNB9
Hearing disorder
Cochlear implant
SENS-501
Congenital Hearing loss
Gene therapy
Lead sponsor: Sensorion
Sponsor class: INDUSTRY
First posted: 2024-04-17
Enrollment: 12
Sex: ALL
Minimum age (years): 6
Maximum age (years): 31
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Safety and tolerability of SENS-501
timeFrame: 5 years
measure: Efficacy of SENS-501 assessed by ABR
timeFrame: 5 years
Secondary outcomes:
measure: Safety and tolerability of SENS-501
timeFrame: 5 years
measure: Efficacy of SENS-501 assessed by ABR
timeFrame: 5 years
measure: Efficacy of SENS-501 assessed by PTA
timeFrame: 5 years
measure: Clinical performance of the administration system
timeFrame: 1 day
measure: Safety of the administration system
timeFrame: 5 years
measure: Usability of the administration system
timeFrame: 1 day
Other outcomes:
Locations:
city: Westmead
country: Australia
facility: Childrens Hospital Westmead
city: Paris
country: France
facility: Hopital Necker Enfants Malades
Study officials:
affiliation: Hopital Necker Enfants Malades
name: Natalie LOUNDON, Pr
role: STUDY_CHAIR
References: