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

NCT07752888 | Active Not Recruiting

• 1 other identifier: GSEA0011
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

DFNB9; Cochlear Implantation

Keywords

OTOF; DFNB9; EA0010

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of Adverse Events (AEs), Serious Adverse Events (SAEs), and Dose-Limiting Toxicities (DLTs) to Week 26

  AEs, SAEs, and DLTs will be evaluated according to NCI-CTCAE v5.0. Safety evaluation includes: physical examination (including cranial nerve function), laboratory indicators (routine blood test, blood biochemistry, urinalysis, coagulation function), vital signs, ECG, peripheral blood neutralizing antibody testing, and peripheral blood AAV shedding testing.

  Day 0 (injection) through Week 26

Secondary Outcomes (3)

• Incidence and severity of AEs, SAEs, and DLTs of EA0010 injection to Week 52

  Week 27 through Week 52

• Efficacy of EA0010 on hearing levels as measured by pure-tone or behavioral audiometry

  Baseline, Week 4, Week 13, Week 26, Week 52, and annually through Year 5

• Efficacy of EA0010 on auditory function as measured by ABR (Auditory Brainstem Response) testing

  Baseline, Day 10, Week 4, Week 13, Week 26, Week 52, and annually through Year 5

Study Arms (1)

EA0010 Injection Arm

EXPERIMENTAL

Genetic: EA0010 Injection

Interventions

EA0010 Injection GENETIC

EA0010 is an investigational gene therapy product comprising two adeno-associated virus (AAV) vectors carrying OTOF gene sequences. The two components (Solution A and Solution B) are mixed at a 1:1 ratio immediately prior to administration. Dose and Administration: Participants will receive a single dose of 2.0 × 10\^11 viral genomes (vg) per ear. The total injection volume will range from 10 to 40 μL, adjusted based on the actual viral titer of the manufactured batch. Administration: Single unilateral intracochlear injection via the round window. Pre-medication: Systemic glucocorticoid therapy will be initiated 3 days prior to surgery (Day -3) to mitigate potential inflammation.

EA0010 Injection Arm

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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;

Sponsors & Collaborators

• Shanghai EmayGene Technology Co., Ltd: lead
• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: collaborator

Study Sites (1)

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, Hubei, 430030, China

Location

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2026
• First Posted: August 7, 2026
• Study Start: May 23, 2026
• Primary Completion (Estimated): May 31, 2027
• Study Completion (Estimated): July 31, 2027
• Last Updated: August 7, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)