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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
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.
Study details:
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.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 6 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2024-06-21
Primary completion: 2027-07-01
Study completion finish: 2031-07-01
Study type
TREATMENT
Phase
PHASE1
PHASE2
Trial ID
NCT06370351
Intervention or treatment
COMBINATION_PRODUCT: SENS-501 administration
Conditions
- • OTOF Gene Mutation
- • DFNB9
- • Congenital Deafness
- • Hearing Disorders
- • Ear Diseases
- • Otorhinolaryngologic Diseases
- • Deafness
- • Hearing Loss, Sensorineural
Find a site
Closest Location:
Childrens Hospital Westmead
Research sites nearby
Select from list below to view details:
Childrens Hospital Westmead
Westmead, Not Specified, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: Dose escalation - Low dose group
| COMBINATION_PRODUCT: SENS-501 administration
|
EXPERIMENTAL: Dose escalation - High dose group
| COMBINATION_PRODUCT: SENS-501 administration
|
EXPERIMENTAL: Dose expansion group
| COMBINATION_PRODUCT: SENS-501 administration
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
Safety and tolerability of SENS-501 | Number of study-related adverse events (AEs) and serious adverse events (SAEs). | 5 years |
Efficacy of SENS-501 assessed by ABR | Improvement of hearing threshold measured by auditory brainstem response (ABR) | 5 years |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Safety and tolerability of SENS-501 | Number of study-related adverse events (AEs) and serious adverse events (SAEs). | 5 years |
Efficacy of SENS-501 assessed by ABR | Improvement of hearing threshold measured by auditory brainstem response (ABR) | 5 years |
Efficacy of SENS-501 assessed by PTA | Improvement of hearing threshold measured by Pure-tone audiometry (PTA) | 5 years |
Clinical performance of the administration system | Administration success ; Users' feedback questionnaire on the administration and use of the devices | 1 day |
Safety of the administration system | Adverse device effects and Device deficiencies, Procedure complications | 5 years |
Usability of the administration system | Usability questionnaire | 1 day |
Frequently Asked Questions
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