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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

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
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
Secondary ID [1] 0 0
2023-504466-28-00
Secondary ID [2] 0 0
SENS-501-101
Universal Trial Number (UTN)
Trial acronym
AUDIOGENE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
OTOF Gene Mutation 0 0
DFNB9 0 0
Congenital Deafness 0 0
Hearing Disorders 0 0
Ear Diseases 0 0
Otorhinolaryngologic Diseases 0 0
Deafness 0 0
Hearing Loss, Sensorineural 0 0
Condition category
Condition code
Ear 0 0 0 0
Deafness
Ear 0 0 0 0
Other ear disorders
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability of SENS-501
Timepoint [1] 0 0
5 years
Primary outcome [2] 0 0
Efficacy of SENS-501 assessed by ABR
Timepoint [2] 0 0
5 years
Secondary outcome [1] 0 0
Safety and tolerability of SENS-501
Timepoint [1] 0 0
5 years
Secondary outcome [2] 0 0
Efficacy of SENS-501 assessed by ABR
Timepoint [2] 0 0
5 years
Secondary outcome [3] 0 0
Efficacy of SENS-501 assessed by PTA
Timepoint [3] 0 0
5 years
Secondary outcome [4] 0 0
Clinical performance of the administration system
Timepoint [4] 0 0
1 day
Secondary outcome [5] 0 0
Safety of the administration system
Timepoint [5] 0 0
5 years
Secondary outcome [6] 0 0
Usability of the administration system
Timepoint [6] 0 0
1 day

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
Minimum age
6 Months
Maximum age
31 Months
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/06/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2031
Actual
Sample size
Target
12
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Childrens Hospital Westmead - Westmead
Recruitment postcode(s) [1] 0 0
NSW 2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
France
State/province [1] 0 0
Paris

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sensorion
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Natalie LOUNDON, Pr
Address 0 0
Hopital Necker Enfants Malades
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Lionel HOVSEPIAN, MD
Address 0 0
Country 0 0
Phone 0 0
+33 7 86 31 13 76
Fax 0 0
Email 0 0
lionel.hovsepian@sensorion-pharma.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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