Actively Recruiting
A Study on the Safety, Tolerability, and Preliminary Efficacy of EH002 in the Treatment of DFNB9 Congenital Deafness
Led by Yilai Shu · Updated on 2025-07-25
24
Participants Needed
2
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and early effectiveness of EH002 gene therapy for treating congenital deafness caused by mutations in the OTOF gene, known as DFNB9. This study focuses on participants with severe or profound hearing loss confirmed by genetic testing. The research aims to understand how EH002 may help restore hearing by addressing the genetic cause of this type of deafness. Participants may receive one or two doses of EH002 delivered directly into one or both ears through a surgical injection into the cochlea. The study includes dose escalation groups with varying amounts of EH002 administered. Eligibility requires passing imaging tests like CT or MRI to confirm suitable ear anatomy for surgery, with follow-up visits scheduled to monitor participants closely. During the 26-week study period, researchers will track any serious side effects or dose-related toxicities and assess changes in hearing levels compared to baseline. Participants will undergo genetic testing, audiological assessments, imaging scans, and safety monitoring throughout. The study will also evaluate hearing improvements and any adverse events to better understand the therapy's impact.
CONDITIONS
Brief Title
A Study of EH002 Gene Therapy for Otoferlin Gene Mutation-mediated Hearing Loss
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant or legal guardian provides informed consent and agrees to follow-up visits
- Participant can communicate and comply with study requirements, with guardian help if needed
- Participant or guardian understands the study and has appropriate expectations
- Participant is at least 6 months old, any gender
- Diagnosed with DFNB9 congenital deafness confirmed by genetic testing showing OTOF mutations
- Severe or profound hearing loss (≥65 dB)
- Meets surgical requirements: no middle- or inner-ear malformations, no vestibulocochlear nerve abnormalities, no ear inflammation as confirmed by CT or MRI within 3 months or at screening
- Eligible for surgery as determined by the study team
You will not qualify if you...
- Genetic diagnosis does not show OTOF mutation
- Deafness types unsuitable for surgery: ear malformations, vestibulocochlear nerve abnormalities, conductive or mixed hearing loss, malformation syndromes
- Pre-existing ear conditions interfering with surgery or study results, such as otitis media, Meniere's disease, acoustic neuroma, unrecovered sudden sensorineural hearing loss
- History of substance abuse or use of ototoxic drugs in last 6 months, antiviral or immunotherapy in last 3 months, or vaccination in last month
- Compromised immunity or immunodeficiency, including HIV or organ transplant history
- Severe systemic or acute illnesses such as tuberculosis, active hepatitis B or C, herpes zoster, pancreatitis, renal insufficiency, gastrointestinal ulcers
- Surgical or anesthetic contraindications including recent cardiovascular or cerebrovascular events or allergies to planned medications
- Participation in other interventional clinical trials within the last year or recent dosing
- Presence of ear implant on side planned for surgery
- Neutralizing antibody titer against AAV1 greater than 1:2000
- Other severe congenital disorders
- History of neurological or psychiatric disorders including epilepsy or dementia
- Need for long-term anticoagulant therapy that cannot be stopped temporarily
- History of chemotherapy or radiation therapy
- Any condition making participant unsuitable as judged by investigator
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) to assess eligibility including genetic testing and imaging
Duration - Up to 26 weeks
Participants receive EH002 gene therapy administered via intracochlear injection into one or both ears.
Multiple visits for treatment administration and monitoring over 26 weeks
Duration - Up to 26 weeks
Participants are monitored for safety, tolerability, and changes in hearing level after treatment.
Regular visits for safety and hearing assessments during follow-up period
Trial Site Locations
Total: 2 locations
1
The First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, China
Actively Recruiting
2
Eye & ENT Hospital of Fudan University
Shanghai, Shanghai Municipality, China, 200031
Actively Recruiting
Research Team
Y
Yilai Shu, M.D. & Ph.D.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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