A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations (NCT07288580) | Clinical Trial Compass
RecruitingPhase 1/2
A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
China30 participantsStarted 2026-04-02
Plain-language 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 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.
Who can participate
Age range
1 Year – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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;
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of Treatment-Related Adverse Events as Assessed by CTCAE v5.0
Timeframe: Up to week 52
2
Proportion of Participants Achieving a Hearing Sensitivity Threshold of ≤70 dB by behavioral audiometry
. 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.
Exclusion criteria
. Have other types of hearing loss ineligible for otologic surgery, including but not limited to:
. 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:
. 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.
. 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:
. Have severe systemic diseases or acute conditions, including but not limited to: