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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

    Study type

    TREATMENT

    phase

    Phase

      PHASE1

      PHASE2

    trial

    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

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    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/ArmIntervention/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 MeasurePrimary Outcome DescriptionPrimary Outcome Time Frame
    Safety and tolerability of SENS-501Number of study-related adverse events (AEs) and serious adverse events (SAEs).5 years
    Efficacy of SENS-501 assessed by ABRImprovement of hearing threshold measured by auditory brainstem response (ABR)5 years

    Secondary outcome

    Secondary Outcome MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
    Safety and tolerability of SENS-501Number of study-related adverse events (AEs) and serious adverse events (SAEs).5 years
    Efficacy of SENS-501 assessed by ABRImprovement of hearing threshold measured by auditory brainstem response (ABR)5 years
    Efficacy of SENS-501 assessed by PTAImprovement of hearing threshold measured by Pure-tone audiometry (PTA)5 years
    Clinical performance of the administration systemAdministration success ; Users' feedback questionnaire on the administration and use of the devices1 day
    Safety of the administration systemAdverse device effects and Device deficiencies, Procedure complications5 years
    Usability of the administration systemUsability questionnaire1 day

    Frequently Asked Questions

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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

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