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 range1 Year – 17 Years
SexALL
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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;
✓. 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;
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
✕. 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: