A Study of EH002 Gene Therapy for Otoferlin Gene Mutation-mediated Hearing Loss

NCTID	NCT06722170 (View at clinicaltrials.gov)
Description	The study is designed to evaluate the safety, tolerability, and preliminary efficacy of EH002 for the treatment of congenital deafness caused by mutations in the OTOF gene. Participants may receive one or two injections of the EH002 gene therapy in one or both ears. (Show More)
Development Status	Active
Indication	DFNB9, Congenital Hearing Loss
Disease Ontology Term	DOID:0110535
Compound Name	EH002
Compound Description	AAV1-Myo15-hOTOF NT + AAV1-hOTOF CT
Sponsor	Yilai Shu
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	18
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
Dose 1	9E11 vg/ear (unilateral)
Dose 2	1.5E12 vg/ear (unilateral)
Dose 3	1.5E12 vg/ear (bilateral)
Dose 4
Dose 5

No.of Trial Sites	2
Locations	China