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New treatment option for DFNB9
Official title A Study of EH002 Gene Therapy for Otoferlin Gene Mutation-mediated Hearing Loss
ClinicalTrials.gov ID: NCT06722170
What this study is testing
What is EH002 administration?
EH002 administration is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for dfnb9.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- The participant and/or their legal guardian must provide informed consent before the study, voluntarily sign the written informed consent form, and...
- The participant must communicate effectively with the investigator and comply with the investigator's requirements, with the assistance of a legal...
- 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...
You likely can't join if
- 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...
- Pre-existing otological conditions that would interfere with the planned surgery or the interpretation of study endpoints, such as acute or chronic...
- A history of substance abuse, or treatment with any known ototoxic drugs (e.g., aminoglycosides, cisplatin, loop diuretics) within the last 6 months...
- Individuals with compromised immunity or a history of immunodeficiency, including HIV-positive status, other acquired or congenital immunodeficiency...
- Patients with severe systemic diseases or severe acute illnesses, such as tuberculosis, active hepatitis B or C infection, active herpes zoster...
See the full eligibility criteria
- 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...
- 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 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...
- 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...
- 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...
- 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...
- Individuals with compromised immunity or a history of immunodeficiency, including HIV-positive status, other acquired or congenital immunodeficiency disorders, or a history of organ transplantation.
- Patients with severe systemic diseases or severe acute illnesses, such as tuberculosis, active hepatitis B or C infection, active herpes zoster infection, pancreatitis, renal insufficiency, or gastrointestinal ulcers.
- Patients with surgical or anesthetic contraindications as determined by the surgeon, anesthesiologist, or designated personnel. This includes those who have had a cardiovascular or cerebrovascular event within the last...
- Currently participating in or planning to participate in any treatment clinical trial involving drugs or devices within the next year or having received the last dose of another clinical trial within 5 half-lives.
- Presence of an implant in the ear (on the side intended for surgery) at the time of screening, such as a cochlear implant.
- Neutralizing antibody titer against AAV1 \>1:2000.
- Presence of other severe congenital disorders.
- A history of definite neurological or psychiatric disorders, including epilepsy or dementia.
- Patients requiring long-term anticoagulant therapy that cannot be temporarily discontinued.
- A history of chemotherapy or radiation therapy.
- Any other condition that, in the opinion of the investigator, makes the participant unsuitable for this clinical study.
The study team makes the final eligibility decision.
Where it's taking place
- Zhengzhou, Henan, China
- Shanghai, Shanghai Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Zhengzhou, Henan, China; Shanghai, Shanghai Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.