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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06370351
Registration number
NCT06370351
Ethics application status
Date submitted
9/04/2024
Date registered
17/04/2024
Date last updated
26/09/2024
Titles & IDs
Public title
A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
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Scientific 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
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Secondary ID [1]
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2023-504466-28-00
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Secondary ID [2]
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SENS-501-101
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Universal Trial Number (UTN)
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Trial acronym
AUDIOGENE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
OTOF Gene Mutation
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DFNB9
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Congenital Deafness
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Hearing Disorders
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Ear Diseases
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Otorhinolaryngologic Diseases
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Deafness
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Hearing Loss, Sensorineural
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Condition category
Condition code
Ear
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Deafness
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Ear
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Other ear disorders
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Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - SENS-501 administration
Experimental: Dose escalation - Low dose group - Intracochlear administration of a Low dose of SENS-501 in one ear, 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
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
Other interventions: SENS-501 administration
Administration of SENS-501 with a dedicated administration system
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Safety and tolerability of SENS-501
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Assessment method [1]
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Number of study-related adverse events (AEs) and serious adverse events (SAEs).
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Timepoint [1]
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5 years
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Primary outcome [2]
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Efficacy of SENS-501 assessed by ABR
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Assessment method [2]
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Improvement of hearing threshold measured by auditory brainstem response (ABR)
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Timepoint [2]
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5 years
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Secondary outcome [1]
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Safety and tolerability of SENS-501
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Assessment method [1]
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Number of study-related adverse events (AEs) and serious adverse events (SAEs).
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Timepoint [1]
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5 years
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Secondary outcome [2]
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Efficacy of SENS-501 assessed by ABR
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Assessment method [2]
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Improvement of hearing threshold measured by auditory brainstem response (ABR)
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Timepoint [2]
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5 years
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Secondary outcome [3]
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Efficacy of SENS-501 assessed by PTA
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Assessment method [3]
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Improvement of hearing threshold measured by Pure-tone audiometry (PTA)
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Timepoint [3]
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5 years
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Secondary outcome [4]
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Clinical performance of the administration system
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Assessment method [4]
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Administration success ; Users' feedback questionnaire on the administration and use of the devices
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Timepoint [4]
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1 day
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Secondary outcome [5]
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Safety of the administration system
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Assessment method [5]
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Adverse device effects and Device deficiencies, Procedure complications
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Timepoint [5]
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5 years
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Secondary outcome [6]
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Usability of the administration system
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Assessment method [6]
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Usability questionnaire
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Timepoint [6]
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1 day
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Eligibility
Key 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
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Minimum age
6
Months
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Maximum age
31
Months
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
21/06/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/07/2031
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Actual
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Sample size
Target
12
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Childrens Hospital Westmead - Westmead
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Recruitment postcode(s) [1]
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NSW 2145 - Westmead
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Recruitment outside Australia
Country [1]
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France
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State/province [1]
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Paris
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Sensorion
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT06370351
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Natalie LOUNDON, Pr
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Address
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Hopital Necker Enfants Malades
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Lionel HOVSEPIAN, MD
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Address
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Country
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Phone
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+33 7 86 31 13 76
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06370351
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