BridgeMD
New treatment option for Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations...
Official title A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
ClinicalTrials.gov ID: NCT07288580
What this study is testing
What is EHT102 Injection?
EHT102 Injection is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for treatment of congenital hearing loss secondary to biallelic mutations of the otoferlin gene (otof).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 17
You may be able to join if
- Prior to study participation, participants and/or their legal guardians must provide informed consent for this trial, voluntarily sign the written...
- Participants must be able to communicate effectively with investigators and comply with study requirements, with guardian assistance if needed. For...
- Participants and/or their legal guardians must demonstrate adequate comprehension of the trial's nature and maintain realistic expectations regarding...
- 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;
You likely can't join if
- Have other types of hearing loss ineligible for otologic surgery, including but not limited to: Middle/inner ear malformations or developmental...
- Have pre-existing otologic conditions deemed by the investigator to potentially compromise the planned surgery or interfere with study endpoint...
- 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.
See the full eligibility 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...
- 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...
- 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...
- DPOAE testing shows present response.
- 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...
- 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...
- 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: Positive HIV antibody (HIV Ab) test; Congenital or acquired immunodeficiency (investigator-determined contraindication to...
- 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...
- Have participated in gene therapy trials within 6 months prior to screening, plan to participate in other treatment clinical trials within one year post-treatment, or have received investigational drugs within 5...
- 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...
- 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...
- Any other condition that, at the discretion of the investigator, renders the participant unsuitable for enrollment.
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.