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.
• 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.