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

Hearing Loss, Sensorineural Clinical Trial

— AUDIOGENE  

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06370351
• Other study ID #: SENS-501-101
• Secondary ID: 2023-504466-28-0
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• Start date: April 30, 2024
• Est. completion date: April 2031

Study information

• Verified date: April 2024
• Source: Sensorion
• Contact: Lionel HOVSEPIAN, MD
• Phone: +33786311376
• Email: lionel.hovsepian[@]sensorion-pharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 12
• Est. completion date: April 2031
• Est. primary completion date: April 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 31 Months

Eligibility

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

Related Conditions & MeSH terms

• Congenital Deafness
• Deafness
• Ear Diseases
• Hearing Disorders
• Hearing Loss
• Hearing Loss, Sensorineural
• Otorhinolaryngologic Diseases

Intervention

Combination Product:
• SENS-501 administration
Administration of SENS-501 with a dedicated administration system

Locations

Country	Name	City	State
France	Hopital Necker Enfants Malades	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Sensorion

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability of SENS-501	Number of study-related adverse events (AEs) and serious adverse events (SAEs).	5 years
Primary	Efficacy of SENS-501 assessed by ABR	Improvement of hearing threshold measured by auditory brainstem response (ABR)	5 years
Secondary	Safety and tolerability of SENS-501	Number of study-related adverse events (AEs) and serious adverse events (SAEs).	5 years
Secondary	Efficacy of SENS-501 assessed by ABR	Improvement of hearing threshold measured by auditory brainstem response (ABR)	5 years
Secondary	Efficacy of SENS-501 assessed by PTA	Improvement of hearing threshold measured by Pure-tone average (PTA)	5 years
Secondary	Clinical performance of the administration system	Administration success ; Users' feedback questionnaire on the administration and use of the devices	1 day
Secondary	Safety of the administration system	Adverse device effects and Device deficiencies, Procedure complications	5 years
Secondary	Usability of the administration system	Usability questionnaire	1 day