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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

PHASE1PHASE2RECRUITING

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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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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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

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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

Condition: OTOF Gene Mutation, DFNB9 and more
COMBINATION_PRODUCT: SENS-501 administration
Westmead, Not Specified, Australia
Sponsor: Sensorion

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

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Find a site

Closest Location:

Childrens Hospital Westmead

Research sites nearby

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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 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

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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You may be eligible to participate in this trial based on your search.Apply for study

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References

Clinical Trials Gov: A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations