OTOF Gene MutationPhase 1/2AUDIOGENENCT06370351
A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
A Phase 1/2 study testing SENS-501 administration in people with OTOF Gene Mutation and DFNB9, run by Sensorion at 2 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 6 to 31 months
- Conditions the study is for
- OTOF Gene Mutation, DFNB9, Congenital Deafness
- What is being tested?
- SENS-501 administration
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 21 Jun 2024to Jul 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 31 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
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
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.
Read the 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.
- Conditions
- OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders, Ear Diseases, Otorhinolaryngologic Diseases
- Being tested
- SENS-501 administration
- Keywords
- OTOF Gene Mutation, Hearing impairment, Deafness, otoferlin, AUDIOGENE, Hearing Loss, DFNB9, Hearing disorder, Cochlear implant, SENS-501, Congenital Hearing loss, Gene therapy
- Medical subject headings
- Hearing Disorders, Ear Diseases, Otorhinolaryngologic Diseases, Deafness, Hearing Loss, Sensorineural, Hearing Loss, Deafness, Autosomal Recessive 9
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SENS-501 administrationCombination product
Administration of SENS-501 with a dedicated administration system
| Study group | Role | What they receive |
|---|---|---|
| Dose escalation - Low dose group | Experimental | Intracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system Receives: Combination Product: SENS-501 administration |
| Dose escalation - High dose group | Experimental | Intracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system Receives: Combination Product: SENS-501 administration |
| Dose expansion group | Experimental | Intracochlear administration of SENS-501 in one ear, with a dedicated administration system, at the dose recommended following the Dose escalation phase Receives: Combination Product: SENS-501 administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of SENS-501
Number of study-related adverse events (AEs) and serious adverse events (SAEs).
Measured: 5 years
Efficacy of SENS-501 assessed by ABR
Improvement of hearing threshold measured by auditory brainstem response (ABR)
Measured: 5 years
Secondary outcomes (6)
Safety and tolerability of SENS-501
Number of study-related adverse events (AEs) and serious adverse events (SAEs).
Measured: 5 years
Efficacy of SENS-501 assessed by ABR
Improvement of hearing threshold measured by auditory brainstem response (ABR)
Measured: 5 years
Efficacy of SENS-501 assessed by PTA
Improvement of hearing threshold measured by Pure-tone audiometry (PTA)
Measured: 5 years
Clinical performance of the administration system
Administration success ; Users' feedback questionnaire on the administration and use of the devices
Measured: 1 day
Safety of the administration system
Adverse device effects and Device deficiencies, Procedure complications
Measured: 5 years
Usability of the administration system
Usability questionnaire
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2024
- Study start21 Jun 2024
- Primary completionJul 2027 (estimated)
- Study completionJul 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for OTOF Gene MutationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sensorion
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Natalie LOUNDON, Pr, Study chair, Hopital Necker Enfants Malades
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06370351
- Sponsor's protocol ID
- SENS-501-101
- Other IDs
- 2023-504466-28-00, 2023
- Acronym
- AUDIOGENE
- Status last verified
- Jul 2026
- First submitted
- 9 Apr 2024
- Last updated
- 8 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06370351
- Acronym / sponsor ID
- AUDIOGENE / SENS-501-101
- Conditions
- OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders, Ear Diseases, Otorhinolaryngologic Diseases
- Keywords
- OTOF Gene Mutation, Hearing impairment, Deafness, otoferlin, AUDIOGENE, Hearing Loss, DFNB9, Hearing disorder
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- SensorionIndustry (pharma, biotech, devices)
- Study start
- 21 Jun 2024 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Jul 2031 (estimated)
- First posted
- 17 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose escalation - Low dose group | Experimental | Combination Product: SENS-501 administrationIntracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system |
| Dose escalation - High dose group | Experimental | Combination Product: SENS-501 administrationIntracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system |
| Dose expansion group | Experimental | Combination Product: SENS-501 administrationIntracochlear administration of SENS-501 in one ear, with a dedicated administration system, at the dose recommended following the Dose escalation phase |
Who may qualify
- Ages
- 6 to 31 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SENS-501 administrationAdministration of SENS-501 with a dedicated administration system | Combination product | Dose escalation - High dose group, Dose escalation - Low dose group, Dose expansion group |
Primary outcome
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)
6 secondary outcomes
Safety and tolerability of SENS-501
Time frame: 5 years
Efficacy of SENS-501 assessed by ABR
Time frame: 5 years
Efficacy of SENS-501 assessed by PTA
Time frame: 5 years
Clinical performance of the administration system
Time frame: 1 day
Safety of the administration system
Time frame: 5 years
Usability of the administration system
Time frame: 1 day
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for OTOF Gene Mutation
Enrolling by invitationObservational study
Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
Otoferlin Gene-mediated Hearing Loss
RecruitingPhase 3
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Sensorineural Hearing Loss, Bilateral
Not yet recruitingObservational study
Genetic Study of OTOF- Related Auditory Neuropathy Spectrum Disorder
Auditory Neuropathy Spectrum Disorder, Auditory Neuropathy
RecruitingPhase 1/2
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
RecruitingObservational study
Natural History in Children up to 16 Years With Mild to Profound Hearing Loss Due to Mutations in GJB2 / OTOF Genes
Sensorineural Hearing Loss, Bilateral, AUNB1
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Natalie LOUNDON, Pr study chair
Hopital Necker Enfants Malades
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SENS-501 administration in people with OTOF Gene Mutation and DFNB9, run by Sensorion at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of SENS-501 (time frame: 5 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2026.