Biallelik hOTOF Mutasyonları Olan Çocuk Hastalarda EHT102 Enjeksiyonu Klinik Çalışması

Sade dilde özet

Bu çalışma, her iki OTOF gen kopyasında (biallelik) mutasyon bulunan ve buna bağlı DFNB9 tipi doğuştan işitme kaybı (congenital hearing loss) olan çocuklarda EHT102 adlı bir gen terapisini inceliyor. Çalışmaya, genetik testle tanısı doğrulanmış, ağır veya çok ağır işitme kaybı olan ve kulak ameliyatına uygun 1-17 yaş arası çocuklar katılabilmektedir. Faz 1/Faz 2 aşamasında yürütülmektedir ve halen katılımcı almaktadır.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

PHASE1/PHASE2

Durum

Katılımcı alıyor

Sponsor

Shanghai Euhearing Therapeutics Co., Ltd

Müdahale

EHT102 Injection

Başlangıç

02.04.2026

Tahmini bitiş

30.12.2033

Ülkeler

China

İlgili genler

Merkezler

• Shanghai, China

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Key Inclusion Criteria:

1. 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;
2. 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;
3. Participants and/or their legal guardians must demonstrate adequate comprehension of the trial's nature and maintain realistic expectations regarding potential benefits.
4. Pediatric patients (male or female) aged ≥ 1 and ≤ 17 years at the time of inclusion ;
5. Genetic testing report indicates DFNB9 congenital deafness with Biallelic mutations in the Otoferlin gene;
6. Severe or profound hearing loss (≥65 dB) assessed by ABR, with the sentinel participant having an ABR \>90 dB;
7. 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;
8. DPOAE testing shows present response.

Exclusion Criteria:

1. 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.
2. 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.
3. Have a history of drug abuse.
4. Have a history of receiving any known ototoxic medications (e.g., aminoglycosides, cisplatin, loop diuretics) within the past 6 months.
5. Antiviral/immunotherapy within 3 months prior to screening.
6. Administration of any live-attenuated vaccines within 30 days prior to screening.
7. 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.
8. Have severe systemic diseases or acute conditions, including but not limited to:

   Active tuberculosis; Active herpes zoster infection; Pancreatitis; Renal insufficiency; Gastrointestinal ulcers.
9. 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.
10. 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.
11. Have implantable devices (e.g., cochlear implants) in the target ear at screening.
12. Have other severe congenital disorders.
13. Have a history of neurological/psychiatric disorders (e.g., epilepsy, dementia).
14. Have chronic anticoagulant therapy that cannot be temporarily discontinued.
15. Have a history of radiotherapy/chemotherapy deemed by investigators to potentially affect trial outcomes.
16. 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).
17. 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.
18. Any other condition that, at the discretion of the investigator, renders the participant unsuitable for enrollment.