Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)Phase 1/2NCT07288580
A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
A Phase 1/2 study testing EHT102 Injection in people with Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF), run by Shanghai Euhearing Therapeutics Co., Ltd at 1 site in China.
Trial summary
This study is a multicenter, single-arm, open-label Phase I/II clinical trial, which is designed to evaluate the safety, tolerability and efficacy of EHT102 injection in treating congenital hearing loss secondary to biallelic mutations of OTOF (DFNB9).Up to 30 pediatric participants (A maximum of 15 participants will… Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
- What is being tested?
- EHT102 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 2 Apr 2026to 30 Dec 2033
- Phase
- Phase 1/2
- 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
- Prior to study participation, participants and/or their legal guardians must provide informed consent for this trial, voluntarily sign the written informed consent form (ICF), and commit to completing all protocol-specified follow-up visits;
- Participants must be able to communicate effectively with investigators and comply with study requirements, with guardian assistance if needed. For young children without developed language skills, guardians must ensure cooperation with investigator instructions;
- Participants and/or their legal guardians must demonstrate adequate comprehension of the trial's nature and maintain realistic expectations regarding potential benefits.
- Pediatric patients (male or female) aged ≥ 1 and ≤ 17 years at the time of inclusion ;
- Genetic testing report indicates DFNB9 congenital deafness with Biallelic mutations in the Otoferlin gene;
- Severe or profound hearing loss (≥65 dB) assessed by ABR, with the sentinel participant having an ABR >90 dB;
- Meet eligibility criteria for otologic surgery: Absence of middle/inner ear malformations, cochleovestibular nerve abnormalities, or active otologic inflammation as confirmed by computed tomography (CT) and/or magnetic resonance imaging (MRI) within 3 months or during screening period, with surgical suitability determined by the investigator;
- DPOAE testing shows present response.
Who cannot join
- Have other types of hearing loss ineligible for otologic surgery, including but not limited to:
- Middle/inner ear malformations or developmental abnormalities identified by CT/MRI within 3 months; Hearing loss caused by cochleovestibular nerve abnormalities; Conductive hearing loss ; Mixed hearing loss; Syndromic deafness with malformations.
- Have pre-existing otologic conditions deemed by the investigator to potentially compromise the planned surgery or interfere with study endpoint evaluation, including but not limited to:
- Acute/chronic otitis media;; Ménière's disease; Acoustic neuroma; Unresolved sudden sensorineural hearing loss.
- Have a history of drug abuse.
- Have a history of receiving any known ototoxic medications (e.g., aminoglycosides, cisplatin, loop diuretics) within the past 6 months.
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- Antiviral/immunotherapy within 3 months prior to screening.
- Administration of any live-attenuated vaccines within 30 days prior to screening.
- Have immunocompromised status or immunodeficiency disorders, including but not limited to:
- Positive HIV antibody (HIV Ab) test; Congenital or acquired immunodeficiency (investigator-determined contraindication to immunosuppressants) ; History of organ transplantation.
- Have severe systemic diseases or acute conditions, including but not limited to:
- Active tuberculosis; Active herpes zoster infection; Pancreatitis; Renal insufficiency; Gastrointestinal ulcers.
- Have contraindications to surgery or anesthesia as determined by the surgeon, anesthesiologist, or designated personnel, including but not limited to:
- History of cardiovascular or cerebrovascular events within the past 6 months (e.g., myocardial infarction, heart failure, angina, stroke, or transient ischemic attack) Any other cardiac conditions deemed unsuitable for study participation by the investigator; Known hypersensitivity to the investigational drug.
- Have participated in gene therapy trials within 6 months prior to screening, plan to participate in other interventional clinical trials within one year post-treatment, or have received investigational drugs within 5 half-lives of the last dose from previous trials.
- Have implantable devices (e.g., cochlear implants) in the target ear at screening.
- Have other severe congenital disorders.
- Have a history of neurological/psychiatric disorders (e.g., epilepsy, dementia).
- Have chronic anticoagulant therapy that cannot be temporarily discontinued.
- Have a history of radiotherapy/chemotherapy deemed by investigators to potentially affect trial outcomes.
- Have tested positive for hepatitis B surface antigen (HBsAg), or positive for hepatitis C virus (HCV) antibody with HCV RNA positive, or positive for human immunodeficiency virus (HIV) antibody, or have active syphilis (TPPA positive and RPR positive).
- Females of childbearing potential with positive pregnancy tests prior to dosing; Females of childbearing potential and non-sterilized males with fertile partners unwilling/unable to use effective contraception from ICF signing until ≥24 months post-dosing.
- Any other condition that, at the discretion of the investigator, renders the participant unsuitable for enrollment.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Eye & ENT Hospital of Fudan University
Shanghai, Shanghai Municipality, 200032
Yilai Shu, M.D. & Ph.D., +86 021 64377134, yilai_shu@fudan.edu.cn
About this study
Official title: A Phase Ⅰ/Ⅱ, Multicenter, Single-arm, Open-label Clinical Trial to Evaluate the Safety, Tolerability and Preliminary Efficacy of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
This study is a multicenter, single-arm, open-label Phase I/II clinical trial, which is designed to evaluate the safety, tolerability and efficacy of EHT102 injection in treating congenital hearing loss secondary to biallelic mutations of OTOF (DFNB9).Up to 30 pediatric participants (A maximum of 15 participants will be enrolled in each of the United States and China) will be enrolled and dosed with EHT102. The dose-escalation phase (Phase I) includes two predefined dose cohorts (3 participants per cohort), with sequential enrollment from low to high dose. During dose escalation, each participant will receive a unilateral EHT102 injection followed by safety observation.
Read the detailed description
The low-dose and high-dose cohorts will be followed by a 28-day DLT observation period.
- Conditions
- Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
- Being tested
- EHT102 Injection
- Keywords
- OTOF
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EHT102 InjectionDrug
EHT102 injection is a dual-vector gene therapy product developed for the treatment of OTOF-related hearing loss.
| Study group | Role | What they receive |
|---|---|---|
| The low-dose cohort | Experimental | Participants will receive a single unilateral or bilateral administration of EHT102 at the defined low dose level Receives: Drug: EHT102 Injection |
| The high-dose cohort | Experimental | Participants will receive a single unilateral or bilateral administration of EHT102 at the defined high dose level. Receives: Drug: EHT102 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Related Adverse Events as Assessed by CTCAE v5.0
Safety will be evaluated by summarizing the number and percentage of participants experiencing treatment-related adverse events (TEAEs) and serious adverse events (SAEs). Adverse events will be coded using the Medical Dictionary for Regulatory Activities (MedDRA) and graded for severity according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
Measured: Up to week 52
Proportion of Participants Achieving a Hearing Sensitivity Threshold of ≤70 dB by behavioral audiometry
This endpoint evaluates the efficacy of EHT102 injection by calculating the percentage of participants who achieve a hearing sensitivity threshold of ≤70 dB in the treated ear, as assessed by behavioral audiometry.
Measured: Up to week 26
Secondary outcomes (4)
Change from Baseline in Auditory Brainstem Response (ABR) Thresholds
The change from baseline in ABR thresholds will be assessed in the treated ear. ABR thresholds are measured in decibels (dB). The metric reported will be the change in the average threshold across frequencies (0.5, 1, 2, and 4 kHz) from baseline to each post-treatment time point.
Measured: Week 4, Week 13, Week 26, and Week 52
Change from baseline in auditory steady state response (ASSR) thresholds
This endpoint assesses the change from baseline in Auditory Steady-State Response (ASSR) thresholds in the treated ear. The metric reported will be the mean change in threshold (in dB) across available frequencies from baseline to each post-treatment time point.
Measured: Week 4, 13, 26, and 52
Proportion of Participants of a hearing sensitivity threshold of ≤70 dB assessed by behavioral audiometry
This endpoint evaluates the efficacy of EHT102 injection at additional timepoints by calculating the percentage of participants who achieve a hearing sensitivity threshold of ≤70 dB in the treated ear, as assessed by behavioral audiometry.
Measured: Week 4, 13 and 52
Proportion of Participants of a hearing sensitivity threshold of ≤45 dB assessed by behavioral audiometry at Week 26 post injection
This endpoint evaluates a higher efficacy threshold by calculating the percentage of participants who achieve a hearing sensitivity threshold of ≤45 dB in the treated ear at Week 26, as assessed by behavioral audiometry
Measured: Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2025
- Study start2 Apr 2026
- Primary completion30 Jun 2028 (estimated)
- Study completion30 Dec 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)Studies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- EHT102 will be administered as a single intracochlear injection into one or both ears. If bilateral injections are required, they will be performed in a single surgical session
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Euhearing Therapeutics Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yilai Shu, M.D. & Ph.D., Principal investigator, Eye & ENT Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07288580
- Sponsor's protocol ID
- EHT102USCL01
- Other IDs
- EHT102CNCL02
- Status last verified
- Apr 2026
- First submitted
- 5 Dec 2025
- Last updated
- 14 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data (IPD) will not be shared. The data generated in this Phase I/II 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 14 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ⅰ/Ⅱ, Multicenter, Single-arm, Open-label Clinical Trial to Evaluate the Safety, Tolerability and Preliminary Efficacy of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
- ClinicalTrials.gov ID
- NCT07288580
- Acronym / sponsor ID
- EHT102USCL01
- Conditions
- Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
- Keywords
- OTOF
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Shanghai Euhearing Therapeutics Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 2 Apr 2026 (actual)
- Primary completion
- 30 Jun 2028 (estimated)
- Study completion
- 30 Dec 2033 (estimated)
- First posted
- 17 Dec 2025 (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 is a multicenter, single-arm, open-label Phase I/II clinical trial, which is designed to evaluate the safety, tolerability and efficacy of EHT102 injection in treating congenital hearing loss secondary to biallelic mutations of OTOF (DFNB9).Up to 30 pediatric participants (A maximum of 15 participants will be enrolled in each of the United States and China) will be enrolled and dosed with EHT102. The dose-escalation phase (Phase I) includes two predefined dose cohorts (3 participants per cohort), with sequential enrollment from low to high dose. During dose escalation, each participant will receive a unilateral EHT102 injection followed by safety observation.
Detailed description
The low-dose and high-dose cohorts will be followed by a 28-day DLT observation period.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- EHT102 will be administered as a single intracochlear injection into one or both ears. If bilateral injections are required, they will be performed in a single surgical session
| Arm | Type | Receives |
|---|---|---|
| The low-dose cohort | Experimental | Drug: EHT102 InjectionParticipants will receive a single unilateral or bilateral administration of EHT102 at the defined low dose level |
| The high-dose cohort | Experimental | Drug: EHT102 InjectionParticipants will receive a single unilateral or bilateral administration of EHT102 at the defined high dose level. |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Prior to study participation, participants and/or their legal guardians must provide informed consent for this trial, voluntarily sign the written informed consent form (ICF), and commit to completing all protocol-specified follow-up visits;
- Participants must be able to communicate effectively with investigators and comply with study requirements, with guardian assistance if needed. For young children without developed language skills, guardians must ensure cooperation with investigator instructions;
- Participants and/or their legal guardians must demonstrate adequate comprehension of the trial's nature and maintain realistic expectations regarding potential benefits.
- Pediatric patients (male or female) aged ≥ 1 and ≤ 17 years at the time of inclusion ;
- Genetic testing report indicates DFNB9 congenital deafness with Biallelic mutations in the Otoferlin gene;
Key exclusion criteria (23 listed)
- Have other types of hearing loss ineligible for otologic surgery, including but not limited to:
- Middle/inner ear malformations or developmental abnormalities identified by CT/MRI within 3 months; Hearing loss caused by cochleovestibular nerve abnormalities; Conductive hearing loss ; Mixed hearing loss; Syndromic deafness with malformations.
- Have pre-existing otologic conditions deemed by the investigator to potentially compromise the planned surgery or interfere with study endpoint evaluation, including but not limited to:
- Acute/chronic otitis media;; Ménière's disease; Acoustic neuroma; Unresolved sudden sensorineural hearing loss.
- Have a history of drug abuse.
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 |
|---|---|---|
| EHT102 InjectionEHT102 injection is a dual-vector gene therapy product developed for the treatment of OTOF-related hearing loss. | Drug | The high-dose cohort, The low-dose cohort |
Primary outcome
Incidence of Treatment-Related Adverse Events as Assessed by CTCAE v5.0
Time frame: Up to week 52
Safety will be evaluated by summarizing the number and percentage of participants experiencing treatment-related adverse events (TEAEs) and serious adverse events (SAEs). Adverse events will be coded using the Medical Dictionary for Regulatory Activities (MedDRA) and graded for severity according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
Proportion of Participants Achieving a Hearing Sensitivity Threshold of ≤70 dB by behavioral audiometry
Time frame: Up to week 26
This endpoint evaluates the efficacy of EHT102 injection by calculating the percentage of participants who achieve a hearing sensitivity threshold of ≤70 dB in the treated ear, as assessed by behavioral audiometry.
4 secondary outcomes
Change from Baseline in Auditory Brainstem Response (ABR) Thresholds
Time frame: Week 4, Week 13, Week 26, and Week 52
Change from baseline in auditory steady state response (ASSR) thresholds
Time frame: Week 4, 13, 26, and 52
Proportion of Participants of a hearing sensitivity threshold of ≤70 dB assessed by behavioral audiometry
Time frame: Week 4, 13 and 52
Proportion of Participants of a hearing sensitivity threshold of ≤45 dB assessed by behavioral audiometry at Week 26 post injection
Time frame: Week 26
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 14 Apr 2026.
- Individual participant dataSharing planned: No. Individual participant data (IPD) will not be shared. The data generated in this Phase I/II study are proprietary. Aggregate safety and efficacy data will be reported in clinical study reports and potential publications.
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Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
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.
Yilai Shu, M.D. & Ph.D. principal investigator
Eye & ENT Hospital of Fudan University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing EHT102 Injection in people with Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF), run by Shanghai Euhearing Therapeutics 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 8 inclusion and 23 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: looking for participants now, as of the sponsor's last update on 14 Apr 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Minghui Huang, M.D (+86 18017315362, minghui.huang@euhearing.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Related Adverse Events as Assessed by CTCAE v5.0 (time frame: Up to week 52). The study lists 2 primary outcomes in all. 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 14 Apr 2026.