Status:

RECRUITING

Natural History in Children Up to 16 Years with Mild to Profound Hearing Loss Due to Mutations in GJB2 / OTOF Genes

Lead Sponsor:

Sensorion

Conditions:

Sensorineural Hearing Loss, Bilateral

AUNB1

Eligibility:

All Genders

Up to 16 years

Brief Summary

The purpose of this study is to follow the natural history of non-syndromic hearing loss caused by mutations in two genes (GJB2 or OTOF) in children up to 16 years of age.

Detailed Description

The study aims to: * better describe the prevalence of cases of DFNB1A and DFNB9, including the type of mutations, and to assess the clinical course of the disease in children up to 16 years of age w...

Eligibility Criteria

Inclusion

  • Main
  • Participants meeting all the following main inclusion criteria will be eligible to participate in the study:
  • Aged ≤ 16 years on the date of signed informed consent for cohort 1 and ≤ 10 years for cohort 2;
  • With a diagnosis of non-syndromic, bilateral, mild to profound, sensorineural hearing loss (according to the American Speech Language-Hearing Association);
  • With documented genotyping results showing mutation(s) in GJB2 or OTOF genes;
  • Written informed consent as required by local regulations.
  • Either without Cochlear Implant, or with unilateral or bilateral Cochlear Implant(s)

Exclusion

  • Participants presenting with any of the following main exclusion criteria will not be included in the study
  • Other type of deafness, such as unilateral deafness, persistent conductive deafness, malformation syndrome, syndromic deafness, known familial deafness linked to mutations in other genes than OTOF or GJB2;
  • Documented genotyping results showing pathogenic mutation(s) in other gene(s) than GJB2 or OTOF genes in the tested panel;
  • Unable and/or unwilling to comply with all the protocol requirements and/or study procedures.

Key Trial Info

Start Date :

November 18 2022

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

November 18 2028

Estimated Enrollment :

180 Patients enrolled

Trial Details

Trial ID

NCT05402813

Start Date

November 18 2022

End Date

November 18 2028

Last Update

February 14 2025

Active Locations (1)

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Page 1 of 1 (1 locations)

1

Necker Hospital

Paris, France