DFNB9NCT07752888
Safety, Tolerability, Efficacy of EA0010 in OTOF Patients With CI
A study testing EA0010 Injection in people with DFNB9 and Cochlear Implantation, run by Shanghai EmayGene Technology Co., Ltd at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- DFNB9, Cochlear Implantation
- What is being tested?
- EA0010 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteChina
- Study dates
- 23 May 2026to 31 Jul 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
Union Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan, Hubei, 430030
About this study
Official title: The Safety, Tolerability, and Efficacy of EA0010 in Patients With OTOF Mutations-Related Hearing Loss Who Have Received Cochlear Implantation
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 cochlear implant users and administer a single intratympanic injection of EA0010 through the stapes annular ligament into the implanted cochlea. One subject is planned to be enrolled, and post-administration assessments of both safety and efficacy will be performed.
Read the detailed description
This study is a single-center, single-arm, open-label, non-randomized clinical study.
To prevent or mitigate potential immune responses to EA0010, participants will receive daily oral or intravenous dexamethasone (0.3 mg/kg) for nine consecutive days, from 3 days before to 5 days after administration (day -3 to day 5). Investigators may adjust the duration and dosage based on clinical requirements. Participants may be admitted to hospital between Day -3 and Day -1. On the day of surgery (Day 0), EA0010 will be administered via intratympanic injection through the stapes annular ligament into the cochlea under aseptic conditions. Participants will receive adeno-associated virus (AAV) at a dose of 2.0×1011 vg/ear/total virus via unilateral cochlear injection (EA0010 Injection is supplied as Solution A and Solution B, each with a titer of 2.0×1013 vg/mL or subject to the actual labeled titer; the two solutions will be mixed at a 1:1 ratio prior to cochlear injection, with a total volume of 10-40 μL); Participants will be closely monitored for 10 days after cochlear injection, may return home after surgical recovery, and will then undergo a 52-week follow-up; the clinical study will end after the last follow-up visit. Subsequent annual follow-ups may be conducted as needed.
Participants will return to the hospital for safety and efficacy assessments at the time points specified in the protocol (D10±3d, W4±3d, W13±7d, W26±14d, W52±14d/EOS/unscheduled) (excluding circumstances of force majeure) during the study period.
- Conditions
- DFNB9, Cochlear Implantation
- Being tested
- EA0010 Injection
- Keywords
- OTOF, DFNB9, EA0010
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EA0010 InjectionGenetic
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 intratympanic injection via the stapes annular ligament.
Pre-medication: Systemic glucocorticoid therapy will be initiated 3 days prior to surgery (Day -3) to mitigate potential inflammation.
| Study group | Role | What they receive |
|---|---|---|
| EA0010 Injection Arm | Experimental | Not described Receives: Genetic: EA0010 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Day 0 (injection) through Week 26
Secondary outcomes (3)
Incidence and severity of AEs, SAEs, and DLTs of EA0010 injection to Week 52
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.
Measured: Week 27 through Week 52
Efficacy of EA0010 on hearing levels as measured by pure-tone or behavioral audiometry
Efficacy of EA0010 on hearing recovery, assessed by PTA or age-appropriate behavioral audiometry.
Measured: 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
Objective electrophysiological assessment of auditory pathway response following EA0010 injection.
Measured: Baseline, Day 10, Week 4, Week 13, Week 26, Week 52, and annually through Year 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2026
- Study start23 May 2026
- Primary completion31 May 2027 (estimated)
- Study completion31 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DFNB9Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shanghai EmayGene Technology Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07752888
- Sponsor's protocol ID
- GSEA0011
- Status last verified
- Aug 2026
- First submitted
- 28 Jul 2026
- Last updated
- 12 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data (IPD) will not be shared. The data generated in this study are proprietary. Aggregate safety and efficacy data will be reported in clinical study reports and potential publications.
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Safety, Tolerability, and Efficacy of EA0010 in Patients With OTOF Mutations-Related Hearing Loss Who Have Received Cochlear Implantation
- ClinicalTrials.gov ID
- NCT07752888
- Acronym / sponsor ID
- GSEA0011
- Conditions
- DFNB9, Cochlear Implantation
- Keywords
- OTOF, DFNB9, EA0010
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Shanghai EmayGene Technology Co., LtdIndustry (pharma, biotech, devices)
- Collaborators
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
- Study start
- 23 May 2026 (actual)
- Primary completion
- 31 May 2027 (estimated)
- Study completion
- 31 Jul 2027 (estimated)
- First posted
- 7 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 cochlear implant users and administer a single intratympanic injection of EA0010 through the stapes annular ligament into the implanted cochlea. One subject is planned to be enrolled, and post-administration assessments of both safety and efficacy will be performed.
Detailed description
This study is a single-center, single-arm, open-label, non-randomized clinical study.
To prevent or mitigate potential immune responses to EA0010, participants will receive daily oral or intravenous dexamethasone (0.3 mg/kg) for nine consecutive days, from 3 days before to 5 days after administration (day -3 to day 5). Investigators may adjust the duration and dosage based on clinical requirements. Participants may be admitted to hospital between Day -3 and Day -1. On the day of surgery (Day 0), EA0010 will be administered via intratympanic injection through the stapes annular ligament into the cochlea under aseptic conditions. Participants will receive adeno-associated virus (AAV) at a dose of 2.0×1011 vg/ear/total virus via unilateral cochlear injection (EA0010 Injection is supplied as Solution A and Solution B, each with a titer of 2.0×1013 vg/mL or subject to the actual labeled titer; the two solutions will be mixed at a 1:1 ratio prior to cochlear injection, with a total volume of 10-40 μL); Participants will be closely monitored for 10 days after cochlear injection, may return home after surgical recovery, and will then undergo a 52-week follow-up; the clinical study will end after the last follow-up visit. Subsequent annual follow-ups may be conducted as needed.
Participants will return to the hospital for safety and efficacy assessments at the time points specified in the protocol (D10±3d, W4±3d, W13±7d, W26±14d, W52±14d/EOS/unscheduled) (excluding circumstances of force majeure) during the study period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| EA0010 Injection Arm | Experimental | Genetic: EA0010 Injection |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Subjects must meet all of the following inclusion criteria to be enrolled in the study:
Key exclusion criteria (20 listed)
- Subjects who meet any of the following criteria are not eligible for enrollment:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EA0010 InjectionEA0010 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 intratympanic injection via the stapes annular ligament. Pre-medication: Systemic glucocorticoid therapy will be initiated 3 days prior to surgery (Day -3) to mitigate potential inflammation. | Genetic | EA0010 Injection Arm |
Primary outcome
Incidence and severity of Adverse Events (AEs), Serious Adverse Events (SAEs), and Dose-Limiting Toxicities (DLTs) to Week 26
Time frame: Day 0 (injection) through 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.
3 secondary outcomes
Incidence and severity of AEs, SAEs, and DLTs of EA0010 injection to Week 52
Time frame: Week 27 through Week 52
Efficacy of EA0010 on hearing levels as measured by pure-tone or behavioral audiometry
Time frame: 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
Time frame: Baseline, Day 10, Week 4, Week 13, Week 26, Week 52, and annually through Year 5
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2026.
- Individual participant dataSharing planned: No. Individual participant data (IPD) will not be shared. The data generated in this study are proprietary. Aggregate safety and efficacy data will be reported in clinical study reports and potential publications.
Related trials for DFNB9
Enrolling by invitationObservational study
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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
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Auditory Restoration and Outcomes After Gene Therapy and Cochlear Implantation: a PRospective Cohort Analysis (AURORA)
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Genetic Study of OTOF- Related Auditory Neuropathy Spectrum Disorder
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A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing EA0010 Injection in people with DFNB9 and Cochlear Implantation, run by Shanghai EmayGene Technology Co., Ltd at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 12 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of Adverse Events (AEs), Serious Adverse Events (SAEs), and Dose-Limiting Toxicities (DLTs) to Week 26 (time frame: Day 0 (injection) through Week 26). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2026.