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)
Indication	Congenital Hearing Loss
Compound Name	DB-OTO, Myo15 promotor
Sponsor	Regeneron Pharmaceuticals
Funder Type	Industry
Status	Recruiting
Enrollment Count	22

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
Dose 1	3 x 10^13 vg/ml
Dose 2	Undisclosed high dose
Dose 3	Undisclosed expansion dose
Dose 4
Dose 5

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

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