Safety, Tolerability, Efficacy of EA0010 in OTOF Patients With CI

Running, not enrolling · Not applicable

Conditions studied: DFNB9, Cochlear Implantation

In brief

This study will evaluate the safety, tolerability, and efficacy of EA0010 injection in patients with OTOF-related hearing loss who have already undergone cochlear implantation. Conventional gene therapy generally excludes cochlear implant recipients, based on the concern that the electrode array may compromise the reparative potential of inner ear cells. To further address this clinical issue, the present study is designed to enroll bilaterally implanted cochlear implant users and administer a single-ear, unilateral injection of EA0010 via the round window. One subject is planned to be enrolled, and post-administration assessments of both safety and efficacy will be performed.

Key facts

Study ID

NCT07752888

Run by

Shanghai EmayGene Technology Co., Ltd

People needed

1

Starts

2026-05-23

Expected to finish

2027-07-31

Last updated by the study team

2026-08-07

Who can join

Age: 1 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects must meet all of the following inclusion criteria to be enrolled in the study:
• Age between 1 year and 17 years (inclusive) at the time of signing the ICF, male or female;
• Confirmed homozygous or compound heterozygous mutation in the OTOF gene as documented in a report issued by a qualified genetic testing institution;
• Audiometric testing (reports from within 6 months prior to signing the ICF are acceptable): Severe or profound deafness (Click ABR ≥ 80 dBnHL);
• Has received cochlear implantation and has reasonable expectations;
• Vital signs, physical examination, laboratory tests (complete blood count, blood biochemistry, coagulation function, urinalysis, etc.), and 12-lead ECG are all normal or show abnormalities judged by the investigator to be clinically insignificant;
• Subject and/or their legal guardian signs the informed consent form.

You may not qualify if…

• Subjects who meet any of the following criteria are not eligible for enrollment:
• Presence of other definite genetic mutations causing deafness other than the OTOF gene that may affect the judgment of the treatment effect of the OTOF investigational drug;
• History of severe allergic reactions to any drug or its components in this study;
• Prior gene therapy and/or oligonucleotide drug treatment in the ear that has undergone cochlear implantation;
• Blood AAV2 neutralizing antibody titer > 1:2000;
• Presence of systemic diseases or receipt of related treatments that may affect hearing or surgical procedures;
• Inability to undergo general anesthesia;
• History of major inner ear surgery (judged by the investigator as inappropriate for gene therapy);
• Other types of deafness unsuitable for otologic surgery, such as deafness caused by middle-inner ear developmental abnormalities or malformations, vestibulocochlear nerve abnormalities, conductive hearing loss, mixed hearing loss, or syndromic malformations as detected by CT/MRI;
• Otologic diseases that may interfere with the planned surgery or interpretation of study endpoints, such as acute-chronic otitis media, Meniere's disease, acoustic neuroma, sudden deafness with no hearing recovery, etc.;
• History of drug abuse, or receipt of any known ototoxic drug therapy within 6 months (e.g., aminoglycosides, cisplatin, loop diuretics, etc.), or receipt of antiviral and immunosuppressive therapy within 3 months, or vaccination within 1 month;
• Subjects currently receiving or likely to receive immunosuppressive therapy other than that required for this study;
• Allergy or intolerance to glucocorticoids;
• History of malignant tumors or meningitis;
• Presence of persistent or active infection; subjects who are HBsAg positive with peripheral blood HBV DNA titer above the lower limit of detection; subjects who are HCV antibody positive with peripheral blood HCV RNA titer above the lower limit of detection; subjects who are HIV antibody positive or have other immunodeficiency diseases; subjects with positive syphilis serology;
• Subjects of childbearing potential who refuse to use effective contraceptive measures (hormonal, barrier methods, or abstinence) from the time of signing the ICF through 12 months after AAV injection;
• Female subjects of childbearing potential with a positive serum pregnancy test, or who are currently pregnant or breastfeeding;
• Subjects who have participated in any other clinical study within 4 weeks prior to the first dose and have already received study drug or treatment (including drug and device clinical studies, excluding non-interventional studies);
• Unwilling or unable to comply with this study protocol;
• Subjects whom the investigator judges to have any medical condition that renders them unable to participate in this study or complete the subsequent follow-up.

Where it is running

• Union Hospital Tongji Medical College Huazhong University of Science and Technology — Wuhan, Hubei, China

Full record on ClinicalTrials.gov

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