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
Enrollment Count	22
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	3E13 vg/ml (unilateral)
Dose 2	Undisclosed high dose (unilateral)
Dose 3	Undisclosed expansion dose (bilateral)
Dose 4
Dose 5

No.of Trial Sites	12
Locations	United States,United Kingdom,Spain

Has US IND	True
FDA Designations
Recent Updates	Phase I/II interim data presented at ASGCT 2024 on 2 patients