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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
Sponsor:
Information provided by (Responsible Party):
Sensorion

Study Description
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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.

Condition or disease Intervention/treatment Phase
OTOF Gene Mutation DFNB9 Congenital Deafness Hearing Disorders Ear Diseases Otorhinolaryngologic Diseases Deafness Hearing Loss, Sensorineural Combination Product: SENS-501 administration Phase 1 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Study Start Date : April 30, 2024
Estimated Primary Completion Date : April 2027
Estimated Study Completion Date : April 2031

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Dose escalation - Low dose group
Intracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system
 Combination Product: SENS-501 administration
Administration of SENS-501 with a dedicated administration system
Experimental: Dose escalation - High dose group
Intracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system
 Combination Product: SENS-501 administration
Administration of SENS-501 with a dedicated administration system
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
 Combination Product: SENS-501 administration
Administration of SENS-501 with a dedicated administration system


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability of SENS-501 [ Time Frame: 5 years ]
    Number of study-related adverse events (AEs) and serious adverse events (SAEs).

  2. Efficacy of SENS-501 assessed by ABR [ Time Frame: 5 years ]
    Improvement of hearing threshold measured by auditory brainstem response (ABR)


Secondary Outcome Measures :
  1. Safety and tolerability of SENS-501 [ Time Frame: 5 years ]
    Number of study-related adverse events (AEs) and serious adverse events (SAEs).

  2. Efficacy of SENS-501 assessed by ABR [ Time Frame: 5 years ]
    Improvement of hearing threshold measured by auditory brainstem response (ABR)

  3. Efficacy of SENS-501 assessed by PTA [ Time Frame: 5 years ]
    Improvement of hearing threshold measured by Pure-tone average (PTA)

  4. Clinical performance of the administration system [ Time Frame: 1 day ]
    Administration success ; Users' feedback questionnaire on the administration and use of the devices

  5. Safety of the administration system [ Time Frame: 5 years ]
    Adverse device effects and Device deficiencies, Procedure complications

  6. Usability of the administration system [ Time Frame: 1 day ]
    Usability questionnaire


Eligibility Criteria
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Ages Eligible for Study:   6 Months to 31 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06370351


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

Locations
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France
Hopital Necker Enfants Malades
Paris, France, 75015
Contact: Natalie LOUNDON, Pr    +33(0)171396782    natalie.loundon@aphp.fr   
Principal Investigator: Natalie LOUNDON, Pr         
Sponsors and Collaborators
Sensorion
Investigators
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Principal Investigator: Natalie LOUNDON, Pr Hopital Necker Enfants Malades
More Information
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Responsible Party: Sensorion
ClinicalTrials.gov Identifier: NCT06370351    
Other Study ID Numbers: SENS-501-101
2023-504466-28-00 ( EU Trial (CTIS) Number )
First Posted: April 17, 2024    Key Record Dates
Last Update Posted: April 17, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sensorion:
Gene therapy
OTOF Gene Mutation
Hearing impairment
Deafness
otoferlin
AUDIOGENE
 Hearing Loss
DFNB9
Hearing disorder
Cochlear implant
SENS-501
Congenital Hearing loss
Additional relevant MeSH terms:
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Hearing Loss
Deafness
Hearing Loss, Sensorineural
Ear Diseases
Hearing Disorders
 Otorhinolaryngologic Diseases
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases