A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

NCTID	NCT05788536 (View at clinicaltrials.gov)
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) (Show More)
Development Status	Active
Indication	Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Disease Ontology Term	DOID:0110535
Compound Name	DB-OTO
Compound Description	Myo15-OTOF-N, OTOF-C
Sponsor	Regeneron Pharmaceuticals
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	30 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	OTOF
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracochlear
Drug Product Type	Viral vector
Target Tissue/Cell	Hair cell
Delivery System	Viral transduction
Vector Type	dual AAV1
Editor Type	none
Dose 1	7.2E12 vg/ear (one or both ears); concentration: 3E13 vg/ml
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	15
Locations	United States,United Kingdom,Germany,Spain

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Regeneron planning regulatory submission by year end 2025