Actively Recruiting
A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
Led by Shanghai Euhearing Therapeutics Co., Ltd · Updated on 2026-04-14
30
Participants Needed
1
Research Sites
404 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study is a multicenter, single-arm, open-label Phase I/II clinical trial, which is designed to evaluate the safety, tolerability and efficacy of EHT102 injection in treating congenital hearing loss secondary to biallelic mutations of OTOF (DFNB9).Up to 30 pediatric participants (A maximum of 15 participants will be enrolled in each of the United States and China) will be enrolled and dosed with EHT102. The dose-escalation phase (Phase I) includes two predefined dose cohorts (3 participants per cohort), with sequential enrollment from low to high dose. During dose escalation, each participant will receive a unilateral EHT102 injection followed by safety observation.
CONDITIONS
Official Title
A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants and/or guardians must provide informed consent and agree to complete all study visits
- Participants must be able to communicate and cooperate with study requirements, with guardian help if needed
- Participants and/or guardians must understand the trial and have realistic expectations of benefits
- Pediatric patients aged 1 to 17 years at enrollment
- Genetic testing confirming DFNB9 congenital deafness with biallelic Otoferlin gene mutations
- Severe or profound hearing loss (65 dB or worse) by auditory brainstem response testing
- Eligibility for ear surgery confirmed by imaging showing no malformations or nerve abnormalities
- Present response on distortion product otoacoustic emissions testing
You will not qualify if you...
- Presence of middle or inner ear malformations or developmental abnormalities on imaging
- Hearing loss caused by cochleovestibular nerve abnormalities
- Conductive or mixed hearing loss
- Syndromic deafness with malformations
- Otologic conditions that could affect surgery or study results, such as otitis media, M�e9ni�e8re's disease, acoustic neuroma, or unresolved sudden sensorineural hearing loss
- History of drug abuse
- Use of ototoxic medications within 6 months prior to screening
- Use of antiviral or immunotherapy within 3 months prior to screening
- Live-attenuated vaccines within 30 days prior to screening
- Immunocompromised status or immunodeficiency disorders
- Severe systemic or acute diseases like active tuberculosis, herpes zoster, pancreatitis, renal insufficiency, or gastrointestinal ulcers
- Contraindications to surgery or anesthesia including recent cardiovascular or cerebrovascular events
- Participation in gene therapy or other interventional trials recently
- Presence of implantable devices like cochlear implants in the target ear
- Other severe congenital disorders
- History of neurological or psychiatric disorders
- Chronic anticoagulant therapy that cannot be stopped temporarily
- History of radiotherapy or chemotherapy affecting trial outcomes
- Positive tests for hepatitis B, hepatitis C, HIV, or active syphilis
- Pregnancy or unwillingness/inability to use contraception as required
- Any other condition deemed unsuitable by the investigator
AI-Screening
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Trial Site Locations
Total: 1 location
1
Eye & ENT Hospital of Fudan University
Shanghai, Shanghai Municipality, China, 200032
Actively Recruiting
Research Team
M
Minghui Huang, M.D
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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