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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-06-21

Study Completion Date

2031-07-31

Brief Summary

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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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Detailed Description

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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

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OTOF Gene Mutation DFNB9 Congenital Deafness Hearing Disorders Ear Diseases Otorhinolaryngologic Diseases Deafness Hearing Loss, Sensorineural

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose escalation - Low dose group

Intracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system

Group Type EXPERIMENTAL

SENS-501 administration

Intervention Type COMBINATION_PRODUCT

Administration of SENS-501 with a dedicated administration system

Dose escalation - High dose group

Intracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system

Group Type EXPERIMENTAL

SENS-501 administration

Intervention Type COMBINATION_PRODUCT

Administration of SENS-501 with a dedicated administration system

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

Group Type EXPERIMENTAL

SENS-501 administration

Intervention Type COMBINATION_PRODUCT

Administration of SENS-501 with a dedicated administration system

Interventions

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SENS-501 administration

Administration of SENS-501 with a dedicated administration system

Intervention Type COMBINATION_PRODUCT

Eligibility Criteria

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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

Minimum Eligible Age

6 Months

Maximum Eligible Age

31 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No