DFNB9NCT05901480
An Investigator Initiated Study for OTOV101N+OTOV101C Injection
A study testing OTOV101N+OTOV101C Injections in people with DFNB9, run by Otovia Therapeutics at 23 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- DFNB9
- What is being tested?
- OTOV101N+OTOV101C Injections
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 23 sitesChina
- Study dates
- 26 Jun 2023to 18 Feb 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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).
- 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.
Who cannot join
- 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);
Show all 19Show fewer
- Subjects with other genetic mutations causing deafness that may affect the effectiveness of OTOF gene therapy;
- Subjects with Meniere's disease;
- Subjects who routinely use ototoxic drugs for other medical conditions;
- Subjects with congenital deafness caused by non-genetic factors related to birth;
- Subjects who are currently receiving or may receive immunosuppressive therapy other than this study;
- Subjects who are allergic or intolerant to glucocorticoid treatment;
- Subjects with a history of malignant tumors or meningitis;
- Subjects with a persistent or active infection, positive for hepatitis B surface antigen (HBsAg) with peripheral blood HBV DNA titers higher than the detection limit, positive for hepatitis C virus (HCV) antibodies with peripheral blood HCV RNA titers higher than the detection limit, positive for human immunodeficiency virus (HIV) antibodies, or with other immune deficiency diseases, or positive for syphilis;
- Subjects of childbearing potential who refuse to take effective contraceptive measures (hormonal or barrier methods or abstinence) from the time of signing the informed consent form until 12 months after receiving AAV injection;
- Female subjects of childbearing age who have a positive blood pregnancy test result, or are currently pregnant or breastfeeding;
- Subjects who have participated in any other clinical trial and have received treatment or medication within 4 weeks prior to the first administration (excluding non-interventional studies);
- Subjects who are unwilling or unable to comply with this study protocol;
- Subjects whom the investigator believes are unable to participate in this study due to any medical condition or who are unable to complete the follow-up study.
Trial locations and contacts
23 sites in 1 country.
23 sites in 1 country
China23 sites, 23 recruiting
- Not yet recruiting
Beijing Tongren Hospital
Beijing
Yongxin Li
- Not yet recruiting
Beijing Union Hospital
Beijing
Xiaowei Chen
- Not yet recruiting
Chinese PLA Genreal Hospital
Beijing
Shiming Yang
Shiming Yang
- Not yet recruiting
The Third Bethune Hospital of Jilin University
Changchun
Dongdong Zhu
- Not yet recruiting
The Second Xiangya Hospital of Central South University
Changsha
Zi'an Xiao
- Not yet recruiting
Sichuan Provincial People Hospital
Chengdu
Jiangang Fan
- Not yet recruiting
Chongqing Municipal People Hospital
Chongqing
Wei Yuan
- Not yet recruiting
The First Affiliated Hospital of Fujian Medical University
Fuzhou
Chang Lin
- Not yet recruiting
Guangdong Provincial People Hospital
Guangzhou
Peina Wu
- Not yet recruiting
The First Affiliated Hospital of Harbin Medical University
Harbin
Tianhong Zhang
- Not yet recruiting
The First Affiliated Hospital of USTC
Hefei
Jingwu Sun
- Not yet recruiting
The Second Affiliated Hospital of Anhui Medical University
Hefei
Jianming Yang
- Recruiting
Shandong Second Provincial General Hospital
Jinan, Shandong, 250023
Lei Xu, +8615168860532, sdphxl@126.com
- Not yet recruiting
Yunnan Provincial First People Hospital
Kunming
Min Guo
- Not yet recruiting
Dongnan University Zhongda Hospital
Nanjing
Xiaoqiong Ding
- Recruiting
Nanjing Drum Tower Hospital
Nanjing
Xia Gao
- Not yet recruiting
The Second Hospital of Ningbo
Ningbo
Kai Wang
- Not yet recruiting
Shengjing Hospital Of China Medical University
Shenyang
Yaodong Dong
- Not yet recruiting
The First Affiliated Hospital of Wenzhou Medical University
Wenzhou
Yideng Huang
- Recruiting
Union Hospital of Tongji Medical College of Huazhong University of Science and Technology
Wuhan
Yu Sun
- Not yet recruiting
Zhongnan Hospital of Wuhan University
Wuhan
Xiong Chen
- Not yet recruiting
Xijing Hospital
Xi'an
Dingjun Zha
- Not yet recruiting
The First Affiliated Hospital of Zhengzhou University
Zhengzhou
Fanglei Ye
About this study
Official title: An Investigator Initiated Study Evaluating the Safety, Tolerability, and Efficacy of OTOV101N+OTOV101C Injection in Treating Patients With OTOF Mutation-related Deafness
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).
Read the detailed description
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.
Subjects who are successfully enrolled will visit the hospital on Day -3 to initiate the glucocorticoid treatment, then be hospitalized on Day -3~-1 to prepare for inner ear gene therapy. On the day of surgery (Day 0), subjects will undergo intracochlear injection of gene therapy products after skin preparation and disinfection of the surgical area and general anesthesia. The round window will be exposed through the tympanic membrane route. Each subject will receive adeno-associated virus (AAV) injection at a dose of 15~30μL of each AAV, mixed at 1: 1 ratio with total volume of 30~60 μL/ear. For subjects without any cochlear implantation, bilateral or unilateral intracochlear injection could be conducted as decided by investigators. For intracochlear injection, the investigators will decide if the second intracochlear injection should be conducted based on dose of the first injection by considering anatomical structure of artificial cochlea and drug loss. The timing of the second injection will be decided by recovery status of the first injection. Subjects will be in hospital for 3 days for observation after receiving the intracochlear injection or follow the routine hospitalization timing of diagnosis/treatment of site, then be discharged after recovery from the surgical operation and receive 1 year follow-up visits.
All subjects will return to the hospital (except in case of non-resistance) for safety and efficacy assessments during the study at the established time points in the protocol (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) unless encountering force majeure.
- Conditions
- DFNB9
- Being tested
- OTOV101N+OTOV101C Injections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OTOV101N+OTOV101C InjectionsGenetic
The gene therapy of OTOV101N+OTOV101C injection via intracochlear injection.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | Patients with OTOF mutation-related deafness Receives: Genetic: OTOV101N+OTOV101C Injections |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events (AEs)
Incidence and severity of AEs are assessed by NCI-CTCAE 5.0.
Measured: Up to 12 months after unilateral cochlear injection
Drug-relatedness of adverse events (AEs)
Drug-relatedness of AEs include definitely relevant, probably relevant, possible relevant, possible irrelevant, and definitely irrelevant.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by physical examination
Number and percentage of participants with abnormal physical examination findings with clinical significance.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by whole blood count
Number and percentage of participants with abnormal laboratory test results (whole blood count) with clinical significance.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by urinalysis
Number and percentage of participants with abnormal laboratory test results (urinalysis) with clinical significance.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by blood biochemistry testing
Number and percentage of participants with abnormal laboratory test results (blood biochemistry testing) with clinical significance.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by coagulation function testing
Number and percentage of participants with abnormal laboratory test results (coagulation function) with clinical significance.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by vital signs
Number and percentage of participants with abnormal vital signs with clinical significance.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by electrocardiogram
Number and percentage of participants with abnormal ECG readings with clinical significance.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by cranial MRI (Magnetic Resonance Imaging)
Changes in cranial MRI relative to baseline, to observe possible signs of infection after gene therapy on inner ear. The MRI conduction is decided by investigator.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by neutralizing antibodies in peripheral blood
Changes in neutralizing antibodies relative to baseline in peripheral blood collections. Concentrations of neutralizing antibodies are analyzed by cell-mediated assay in vitro.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by Adeno-Associated Virus (AAV) in peripheral blood
Changes in AAV signals relative to baseline in peripheral blood collections. AAV signals are analyzed by real-time PCR assay in vitro.
Measured: Up to 12 months after unilateral cochlear injection
Safety assessment by CT (Computed Tomography)
Changes in cranial CT relative to baseline, to observe possible signs of infection after gene therapy on inner ear. The CT conduction is decided by investigator.
Measured: Up to 12 months after unilateral cochlear injection
Secondary outcomes (6)
Efficacy assessment by Behavioral audiometry testing
Changes in hearing assessment by behavioral audiometry relative to baseline, to observe the improvement of hearing functions after gene therapy on inner ear. Behavioral audiometry assessments are to measure the hearing threshold at different frequencies (pitches) after treatment compared to its baseline values.
Measured: Up to 12 months after unilateral cochlear injection
Efficacy assessment by ABR (Auditory Brainstem Response) testing
Changes in hearing assessment by ABR relative to baseline, to observe the improvement of hearing functions after gene therapy on inner ear. ABR assessments are to measure the electrical response evoked by acoustic stimuli as sound signal is processed along the auditory pathway. Mean ABR air and bone conduction threshold are assessed.
Measured: Up to 12 months after unilateral cochlear injection
Efficacy assessment by ASSR (Auditory Steady-state Response) testing
Changes in hearing assessment by ASSR relative to baseline, to observe the improvement of hearing functions after gene therapy on inner ear. ASSR assessments are to measure the Steady-State electroencephalic response evoked by acoustic stimuli as sound signal is processed along the auditory pathway. Mean ASSR air and bone conduction threshold are assessed.
Measured: Up to 12 months after unilateral cochlear injection
Efficacy assessment by DPOAE (Distortion Product Otoacoustic Emission) testing
Changes in hearing assessment by DPOAE relative to baseline, to observe the improvement of hearing functions after gene therapy on inner ear. DPOAE is defined as sound generated within the cochlear by stimulating the ear with two simultaneous tones of different frequency. DPOAEs serve as an objective measure of hearing sensitivity. Tones will be played from low to high frequencies at soft to moderate levels to assess responses at different regions of the inner ear. DP levels will be recorded for each frequency and ear. Higher DP levels indicate more sensitive hearing. Change from baseline values will be calculated as the reported DP level value minus the baseline value. A negative change from baseline indicates less sensitive hearing.
Measured: Up to 12 months after unilateral cochlear injection
Efficacy assessment by CM (Cochlear Microphonic) potential
Changes in hearing assessment of CM potentials relative to baseline, to observe the improvement of hearing functions after gene therapy on inner ear. CM potentials are measured by Cochlear Response Telemetry System.
Measured: Up to 12 months after unilateral cochlear injection
Efficacy assessment by tympanometry
Changes in hearing assessment of tympanometry relative to baseline, to observe the improvement of hearing functions after gene therapy on inner ear. Tympanometry are used to assess the mobility of the eardrum. Compliance, middle ear pressure and ear canal volume will be recorded and checked to measure function of middle ear and eustachian tube.
Measured: Up to 12 months after unilateral cochlear injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2023
- Study start26 Jun 2023
- Primary completion6 Dec 2024 (estimated)
- Study completion18 Feb 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for DFNB9Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Otovia Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shanzhong Zhang, MD PhD, Study director, Otovia Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05901480
- Sponsor's protocol ID
- OTOV101-IIT-101
- Status last verified
- Aug 2023
- First submitted
- 9 May 2023
- Last updated
- 8 May 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Qi J, Zhang L, Lu L, Tan F, Cheng C, Lu Y, Dong W, Zhou Y, Fu X, Jiang L, Tan C, Zhang S, Sun S, Song H, Duan M, Zha D, Sun Y, Gao X, Xu L, Zeng FG, Chai R. AAV gene therapy for autosomal recessive deafness 9: a single-arm trial. Nat Med. 2025 Sep;31(9):2917-2926. doi: 10.1038/s41591-025-03773-w. Epub 2025 Jul 2.
PubMed 40603731
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigator Initiated Study Evaluating the Safety, Tolerability, and Efficacy of OTOV101N+OTOV101C Injection in Treating Patients With OTOF Mutation-related Deafness
- ClinicalTrials.gov ID
- NCT05901480
- Acronym / sponsor ID
- OTOV101-IIT-101
- Conditions
- DFNB9
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Otovia TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 26 Jun 2023 (actual)
- Primary completion
- 6 Dec 2024 (estimated)
- Study completion
- 18 Feb 2025 (estimated)
- First posted
- 13 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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.
Subjects who are successfully enrolled will visit the hospital on Day -3 to initiate the glucocorticoid treatment, then be hospitalized on Day -3~-1 to prepare for inner ear gene therapy. On the day of surgery (Day 0), subjects will undergo intracochlear injection of gene therapy products after skin preparation and disinfection of the surgical area and general anesthesia. The round window will be exposed through the tympanic membrane route. Each subject will receive adeno-associated virus (AAV) injection at a dose of 15~30μL of each AAV, mixed at 1: 1 ratio with total volume of 30~60 μL/ear. For subjects without any cochlear implantation, bilateral or unilateral intracochlear injection could be conducted as decided by investigators. For intracochlear injection, the investigators will decide if the second intracochlear injection should be conducted based on dose of the first injection by considering anatomical structure of artificial cochlea and drug loss. The timing of the second injection will be decided by recovery status of the first injection. Subjects will be in hospital for 3 days for observation after receiving the intracochlear injection or follow the routine hospitalization timing of diagnosis/treatment of site, then be discharged after recovery from the surgical operation and receive 1 year follow-up visits.
All subjects will return to the hospital (except in case of non-resistance) for safety and efficacy assessments during the study at the established time points in the protocol (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) unless encountering force majeure.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Experimental | Genetic: OTOV101N+OTOV101C InjectionsPatients with OTOF mutation-related deafness |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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:
- 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).
- 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.
Key exclusion criteria (19 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OTOV101N+OTOV101C InjectionsThe gene therapy of OTOV101N+OTOV101C injection via intracochlear injection. | Genetic | Treatment Arm |
Primary outcome
Incidence and severity of adverse events (AEs)
Time frame: Up to 12 months after unilateral cochlear injection
Incidence and severity of AEs are assessed by NCI-CTCAE 5.0.
Drug-relatedness of adverse events (AEs)
Time frame: Up to 12 months after unilateral cochlear injection
Drug-relatedness of AEs include definitely relevant, probably relevant, possible relevant, possible irrelevant, and definitely irrelevant.
Safety assessment by physical examination
Time frame: Up to 12 months after unilateral cochlear injection
Number and percentage of participants with abnormal physical examination findings with clinical significance.
Safety assessment by whole blood count
Time frame: Up to 12 months after unilateral cochlear injection
Number and percentage of participants with abnormal laboratory test results (whole blood count) with clinical significance.
Safety assessment by urinalysis
Time frame: Up to 12 months after unilateral cochlear injection
Number and percentage of participants with abnormal laboratory test results (urinalysis) with clinical significance.
Safety assessment by blood biochemistry testing
Time frame: Up to 12 months after unilateral cochlear injection
Number and percentage of participants with abnormal laboratory test results (blood biochemistry testing) with clinical significance.
Safety assessment by coagulation function testing
Time frame: Up to 12 months after unilateral cochlear injection
Number and percentage of participants with abnormal laboratory test results (coagulation function) with clinical significance.
Safety assessment by vital signs
Time frame: Up to 12 months after unilateral cochlear injection
Number and percentage of participants with abnormal vital signs with clinical significance.
Safety assessment by electrocardiogram
Time frame: Up to 12 months after unilateral cochlear injection
Number and percentage of participants with abnormal ECG readings with clinical significance.
Safety assessment by cranial MRI (Magnetic Resonance Imaging)
Time frame: Up to 12 months after unilateral cochlear injection
Changes in cranial MRI relative to baseline, to observe possible signs of infection after gene therapy on inner ear. The MRI conduction is decided by investigator.
Safety assessment by neutralizing antibodies in peripheral blood
Time frame: Up to 12 months after unilateral cochlear injection
Changes in neutralizing antibodies relative to baseline in peripheral blood collections. Concentrations of neutralizing antibodies are analyzed by cell-mediated assay in vitro.
Safety assessment by Adeno-Associated Virus (AAV) in peripheral blood
Time frame: Up to 12 months after unilateral cochlear injection
Changes in AAV signals relative to baseline in peripheral blood collections. AAV signals are analyzed by real-time PCR assay in vitro.
Safety assessment by CT (Computed Tomography)
Time frame: Up to 12 months after unilateral cochlear injection
Changes in cranial CT relative to baseline, to observe possible signs of infection after gene therapy on inner ear. The CT conduction is decided by investigator.
6 secondary outcomes
Efficacy assessment by Behavioral audiometry testing
Time frame: Up to 12 months after unilateral cochlear injection
Efficacy assessment by ABR (Auditory Brainstem Response) testing
Time frame: Up to 12 months after unilateral cochlear injection
Efficacy assessment by ASSR (Auditory Steady-state Response) testing
Time frame: Up to 12 months after unilateral cochlear injection
Efficacy assessment by DPOAE (Distortion Product Otoacoustic Emission) testing
Time frame: Up to 12 months after unilateral cochlear injection
Efficacy assessment by CM (Cochlear Microphonic) potential
Time frame: Up to 12 months after unilateral cochlear injection
Efficacy assessment by tympanometry
Time frame: Up to 12 months after unilateral cochlear injection
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2024.
- Derived from this study: PubMed 40603731Qi J, Zhang L, Lu L, Tan F, Cheng C, Lu Y, Dong W, Zhou Y, Fu X, Jiang L, Tan C, Zhang S, Sun S, Song H, Duan M, Zha D, Sun Y, Gao X, Xu L, Zeng FG, Chai R. AAV gene therapy for autosomal recessive deafness 9: a single-arm trial. Nat Med. 2025 Sep;31(9):2917-2926. doi: 10.1038/s41591-025-03773-w. Epub 2025 Jul 2.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shanzhong Zhang, MD PhD study director
Otovia Therapeutics
23 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing OTOV101N+OTOV101C Injections in people with DFNB9, run by Otovia Therapeutics at 23 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Shanzhong Zhang, MD PhD (+86 18616595944, zhangshanzhong@fosunpharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events (AEs) (time frame: Up to 12 months after unilateral cochlear injection). The study lists 13 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05901480, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 May 2024.