A Study of EH002 Gene Therapy for Otoferlin Gene Mutation-mediated Hearing Loss
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age6+
SponsorYilai Shu
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
The genetic diagnosis does not indicate an OTOF mutation.
Other types of deafness that are unsuitable for otological surgery, such as middle- or inner-ear developmental abnormalities or malformations, vestibulocochlear nerve abnormalities, conductive hearing loss, mixed hearing loss, or malformation syndromes, as detected by CT or MRI scans within 3 months.
Pre-existing otological conditions that would interfere with the planned surgery or the interpretation of study endpoints, such as acute or chronic otitis media, Meniere's disease, acoustic neuroma, or unrecovered sudden sensorineural hearing loss.
A history of substance abuse, or treatment with any known ototoxic drugs (e.g., aminoglycosides, cisplatin, loop diuretics) within the last 6 months, or antiviral medications or immunotherapy within the last 3 months, or vaccination within the last month.
Trial design
Design model
Sequential
Treatments tested in this trial
EH002 administration
GeneticEH002 was administered into one or both ears via intracochlear injection.
Treatment groups
24 Participants
are divided into 4 treatment groupsGroup A: Dose escalation: 50-μl dose groupExperimental treatment 1 intervention
Group B: Dose escalation: 100-μl dose groupExperimental treatment 1 intervention
Group C: Dose escalation: 150-μl dose groupExperimental treatment 1 intervention
Group D: Dose escalation: 25-μl dose groupExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
The incidence of dose-limited toxicity and the incidence of serious adverse events or adverse events
Time frame
26 weeksSecondary outcomes
1
Changes in hearing level relative to baseline level
Time frame
26 weeksSponsors and contacts
Click on the lead sponsor to view all of their trials.
Yilai Shu
Lead sponsor
Eye & ENT Hospital of Fudan University
Sponsor institution
Source ClinicalTrials.gov