NCT06370351

Brief 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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
63mo left

Started Jun 2024

Longer than P75 for phase_1

Geographic Reach
2 countries

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress27%
Jun 2024Jul 2031

First Submitted

Initial submission to the registry

April 9, 2024

Completed
8 days until next milestone

First Posted

Study publicly available on registry

April 17, 2024

Completed
2 months until next milestone

Study Start

First participant enrolled

June 21, 2024

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2027

Expected
4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2031

Last Updated

September 26, 2024

Status Verified

September 1, 2024

Enrollment Period

3 years

First QC Date

April 9, 2024

Last Update Submit

September 23, 2024

Conditions

OTOF Gene MutationDFNB9Congenital DeafnessHearing DisordersEar DiseasesOtorhinolaryngologic DiseasesDeafnessHearing Loss, Sensorineural

Keywords

OTOF Gene MutationHearing impairmentDeafnessotoferlinAUDIOGENEHearing LossDFNB9Hearing disorderCochlear implantSENS-501Congenital Hearing lossGene therapy

Outcome Measures

Primary Outcomes (2)

  • 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 Outcomes (6)

  • Safety and tolerability of SENS-501

    5 years

  • Efficacy of SENS-501 assessed by ABR

    5 years

  • Efficacy of SENS-501 assessed by PTA

    5 years

  • Clinical performance of the administration system

    1 day

  • Safety of the administration system

    5 years

  • +1 more secondary outcomes

Study Arms (3)

Dose escalation - Low dose group

EXPERIMENTAL

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

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

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

Combination Product: SENS-501 administration

Interventions

SENS-501 administrationCOMBINATION_PRODUCT

Administration of SENS-501 with a dedicated administration system

Dose escalation - High dose groupDose escalation - Low dose groupDose expansion group

Eligibility Criteria

Age6 Months - 31 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Childrens Hospital Westmead

Westmead, NSW 2145, Australia

RECRUITING

Hopital Necker Enfants Malades

Paris, 75015, France

RECRUITING

MeSH Terms

Conditions

Hearing DisordersEar DiseasesOtorhinolaryngologic DiseasesDeafnessHearing Loss, SensorineuralHearing LossDeafness, Autosomal Recessive 9

Condition Hierarchy (Ancestors)

Sensation DisordersNeurologic ManifestationsNervous System DiseasesSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Officials

  • Natalie LOUNDON, Pr

    Hopital Necker Enfants Malades

    STUDY CHAIR

Central Study Contacts

Lionel HOVSEPIAN, MD

CONTACT

+33 7 86 31 13 76lionel.hovsepian@sensorion-pharma.com

GĂ©raldine HONNET, MD

CONTACT

geraldine.honnet@sensorion-pharma.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 9, 2024

First Posted

April 17, 2024

Study Start

June 21, 2024

Primary Completion (Estimated)

July 1, 2027

Study Completion (Estimated)

July 1, 2031

Last Updated

September 26, 2024

Record last verified: 2024-09

Data Sharing

IPD Sharing
Will not share

Locations

AU(1)FR(1)