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

Sensorion

Status and phase: Enrolling; Phase 2; Phase 1

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

Treatments: Combination Product: SENS-501 administration

Study type: Interventional

Funder types: Industry

Identifiers

NCT06370351

2023-504466-28-00

SENS-501-101

Details and patient eligibility

About

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.

Full 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.

Enrollment: 12 estimated patients

Sex: All

Ages: 6 to 31 months old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

---

12 participants in 3 patient groups

Dose escalation - Low dose group

Experimental group

Description:

Intracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system

Treatment:

Combination Product: SENS-501 administration

Dose escalation - High dose group

Experimental group

Description:

Intracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system

Treatment:

Combination Product: SENS-501 administration

Dose expansion group

Experimental group

Description:

Intracochlear administration of SENS-501 in one ear, with a dedicated administration system, at the dose recommended following the Dose escalation phase

Treatment:

Combination Product: SENS-501 administration