A Study on the Safety, Tolerability, and Preliminary Efficacy of EH002 in the Treatment of DFNB9 Congenital Deafness
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.
• The participant and/or their legal guardian must provide informed consent before the study, voluntarily sign the written informed consent form, and be willing to attend follow-up visits as scheduled by the study.
• The participant must communicate effectively with the investigator and comply with the investigator's requirements, with the assistance of a legal guardian if necessary. Young children without mature language skills must be able to cooperate and comply with the investigator's requirements with the help of a legal guardian.
• The participant and/or legal guardian must understand the study correctly and have appropriate expectations regarding the potential benefits.
• The participant must be at least 6 months old, with no restrictions on gender.
• The participant must be diagnosed with DFNB9 congenital deafness, confirmed by genetic testing showing homozygous or compound heterozygous mutations in the OTOF gene.
• Audiological inclusion criteria: Severe or profound hearing loss (≥65 dB).
• The participant must meet the surgical requirements, including the absence of middle- or inner-ear malformations, vestibulocochlear nerve developmental abnormalities, and ear inflammation, as confirmed by CT or MRI scans within 3 months or at the time of screening. Additionally, the participant must be deemed eligible for surgery.