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

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ClinicalTrials.gov Identifier: NCT06370351

Recruitment Status : Not yet recruiting

First Posted : April 17, 2024

Last Update Posted : April 17, 2024

See Contacts and Locations

View this study on the modernized ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Sensorion

Tracking Information

First Submitted Date ^ICMJE

April 9, 2024

First Posted Date ^ICMJE

April 17, 2024

Last Update Posted Date

April 17, 2024

Estimated Study Start Date ^ICMJE

April 30, 2024

Estimated Primary Completion Date

April 2027 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Safety and tolerability of SENS-501 [ Time Frame: 5 years ]
Number of study-related adverse events (AEs) and serious adverse events (SAEs).
Efficacy of SENS-501 assessed by ABR [ Time Frame: 5 years ]
Improvement of hearing threshold measured by auditory brainstem response (ABR)

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

No Changes Posted

Current Secondary Outcome Measures ^ICMJE

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

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Pre-specified Outcome Measures

Not Provided

Original Other Pre-specified Outcome Measures

Not Provided

Descriptive Information

Brief Title ^ICMJE

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 ^ICMJE

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

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 Type ^ICMJE

Interventional

Study Phase ^ICMJE

Phase 1
Phase 2

Study Design ^ICMJE

Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment

Condition ^ICMJE

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

Intervention ^ICMJE

Combination Product: SENS-501 administration

Administration of SENS-501 with a dedicated administration system

Study Arms ^ICMJE

Experimental: Dose escalation - Low dose group
Intracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system

Intervention: Combination Product: SENS-501 administration
Experimental: Dose escalation - High dose group
Intracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system

Intervention: Combination Product: SENS-501 administration
Experimental: Dose expansion group
Intracochlear administration of SENS-501 in one ear, with a dedicated administration system, at the dose recommended following the Dose escalation phase

Intervention: Combination Product: SENS-501 administration

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Not yet recruiting

Estimated Enrollment ^ICMJE

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date ^ICMJE

April 2031

Estimated Primary Completion Date

April 2027 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Sex/Gender ^ICMJE

Sexes Eligible for Study:

All

Ages ^ICMJE

6 Months to 31 Months (Child)

Accepts Healthy Volunteers ^ICMJE

Contacts ^ICMJE

Contact: Lionel HOVSEPIAN, MD	+33786311376	lionel.hovsepian@sensorion-pharma.com
Contact: Géraldine HONNET, MD		geraldine.honnet@sensorion-pharma.com

Listed Location Countries ^ICMJE

France

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT06370351

Other Study ID Numbers ^ICMJE

SENS-501-101
2023-504466-28-00 ( EU Trial (CTIS) Number )

Has Data Monitoring Committee

Yes

U.S. FDA-regulated Product

Studies a U.S. FDA-regulated Drug Product:	No
Studies a U.S. FDA-regulated Device Product:	No

IPD Sharing Statement ^ICMJE

Plan to Share IPD:

Current Responsible Party

Sensorion

Original Responsible Party

Same as current

Current Study Sponsor ^ICMJE

Sensorion

Original Study Sponsor ^ICMJE

Same as current

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

Natalie LOUNDON, Pr

Hopital Necker Enfants Malades

PRS Account

Sensorion

Verification Date

April 2024

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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