An Investigator Initiated Study for OTOV101N+OTOV101C Injection (NCT05901480) | Clinical Trial Compass
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An Investigator Initiated Study for OTOV101N+OTOV101C Injection
China25 participantsStarted 2023-06-26
Plain-language summary
This study is an investigator initiated study to evaluate the safety, tolerability, and efficacy of OTOV101N+OTOV101C injection in treating patients with OTOF mutation-related deafness. The enrolled subjects who meet the inclusion and exclusion criteria will receive the gene therapy of OTOV101N+OTOV101C injection via intracochlear injection. All participants will return to the hospital for safety and efficacy evaluations at predetermined time points defined by protocol during the study (Week 1 ± 1 Day, Week 2 ± 3 Days, Week 3 ± 3 Days, Month 1 ± 3 Days, Month 2 ± 3 Days, Month 4 ± 6 Days, Month 6 ± 6 Days, Month 9 ± 6 Days, Month 12 ± 6 Days/EOS (end of study)/unscheduled visit).
Who can participate
Age range
1 Year
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
. Age ≥ 1 years old at the time of signing the informed consent form (ICF); both male and female are eligible.
. Diagnostic criteria for OTOF-related hearing loss are:
. The hearing test and auditory brainstem response (ABR) examination show the presence of hearing loss (based on the testing report conducted within one month prior to signing the informed consent form).
. Genetic testing confirms the presence of OTOF gene homozygous or compound heterozygous mutations.
. Hearing loss: severe (65 dB ≤ hearing threshold \< 80 dB) or profound (80 dB ≤ hearing threshold \< 95 dB) or total (hearing threshold ≥ 95 dB) hearing loss in both ears (If the testing result of ABR is "waveform is not obtained", the subjects with bilateral hearing threshold \<65 dB will be enrolled as determined by the investigator).
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 and severity of adverse events (AEs)
Timeframe: Up to 12 months after unilateral cochlear injection
2
Drug-relatedness of adverse events (AEs)
Timeframe: Up to 12 months after unilateral cochlear injection
3
Safety assessment by physical examination
Timeframe: Up to 12 months after unilateral cochlear injection
4
Safety assessment by whole blood count
Timeframe: Up to 12 months after unilateral cochlear injection
5
Safety assessment by urinalysis
Timeframe: Up to 12 months after unilateral cochlear injection
6
Safety assessment by blood biochemistry testing
Timeframe: Up to 12 months after unilateral cochlear injection
7
Safety assessment by coagulation function testing
Timeframe: Up to 12 months after unilateral cochlear injection
. Vital signs, physical examination, laboratory tests (including whole blood count, blood biochemistry, urinalysis, coagulation function, etc.), and 12-lead electrocardiogram are all normal, or any abnormalities judged by the investigator are clinically non-significant.
. The subjects and their guardians sign the informed consent form.
Exclusion criteria
. Subjects who have had a severe allergic reaction (NCICTCAE5.0 ≥ 3 Grade) to any drug or its components used in this study in the past;
. Subjects who have received any gene therapy in the past, or have high levels of neutralizing antibodies (\>1:128) in their blood;
. Subjects who have systemic diseases or are receiving related treatments that may affect hearing or surgical operations;
. Subjects who cannot tolerate anesthesia;
. Subjects with inner ear malformations;
. Subjects who have undergone bilateral cochlear implantation or have a history of major inner ear surgery (as determined by the investigator)(not include unilateral cochlear implantation);
. Subjects with other genetic mutations causing deafness that may affect the effectiveness of OTOF gene therapy;
. Subjects with Meniere's disease;
8
Safety assessment by vital signs
Timeframe: Up to 12 months after unilateral cochlear injection
9
Safety assessment by electrocardiogram
Timeframe: Up to 12 months after unilateral cochlear injection
10
Safety assessment by cranial MRI (Magnetic Resonance Imaging)
Timeframe: Up to 12 months after unilateral cochlear injection
11
Safety assessment by neutralizing antibodies in peripheral blood
Timeframe: Up to 12 months after unilateral cochlear injection
12
Safety assessment by Adeno-Associated Virus (AAV) in peripheral blood
Timeframe: Up to 12 months after unilateral cochlear injection
13
Safety assessment by CT (Computed Tomography)
Timeframe: Up to 12 months after unilateral cochlear injection