Treatment Trials

2 Clinical Trials for Various Conditions

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      WITHDRAWN
      A Natural History Study in Pediatric Participants With Hearing Loss Due to OTOF, GJB2, or GJB2/GJB6 Mutations
      Description

      This is an observational study to examine the characteristics of gene-related hearing loss in pediatric participants with biallelic otoferlin (OTOF) Mutations, Gap Junction Beta 2 (GJB2) Mutations, or Digenic GJB2/Gap Junction Beta 6 (GJB6) Mutations. This study will follow the participant for 4 years with annual visits each year.

      Conditions
      Congenital Hearing Loss Secondary to Biallelic Mutations in the Otoferlin Gene (OTOF)Biallelic Mutations in the Gap Junction Beta 2 (GJB2) GeneDigenic Mutations in GJB2/Gap Junction Beta 6 (GJB6) Genes
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      RECRUITING
      A Study of DB-OTO, an Adeno-associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
      Description

      Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat children who have hearing loss due to changes in the otoferlin gene. The purpose of this study is to: * Learn about the safety of DB-OTO * Determine how well DB-OTO is tolerated (does not cause ongoing discomfort) * Evaluate the efficacy of DB-OTO (how well DB-OTO works)

      Conditions
      Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
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