Congenital Hearing LossEarly Phase 1NCT06025032
a Study in Subjects With Otoferlin Mutation-related Hearing Loss Using RNA Base-eDiting Therapy(SOUND)
A Early Phase 1 study testing HG205 in people with Congenital Hearing Loss, run by HuidaGene Therapeutics Co., Ltd. at 1 site in China.
Trial summary
The purpose of the study is to determine whether HG205 as CRISPR/Cas13 RNA base-editing therapy is safe and effective for the treatment of hearing loss caused by p.Q829X mutation in OTOF gene. Read more about this study
- Who is it for?
- Ages 1 year to 16 years
- Conditions the study is for
- Congenital Hearing Loss
- What is being tested?
- HG205
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteChina
- Study dates
- 30 Mar 2023to 8 Apr 2025
- Phase
- Early Phase 1
- 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 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or females between 1 and 16 years of age at the time the subject/parent/legal guardian signs the informed consent form.
- Willing to adhere to the protocol as evidenced by written informed consent or parental permission and subject assent.
- Molecular diagnosis of biallelic mutations in the OTOF gene with at least one mutation being p.Q829X through collected blood samples at screening;
- Based on auditory brainstem response (ABR), clinically diagnosed sensorineural hearing loss (SNHL) with the following hearing thresholds: severe (65 dB ≤ hearing threshold < 80 dB) or profound (80 dB ≤ hearing threshold < 95 dB) or complete (hearing threshold ≥ 95 dB) hearing loss in both ears.
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
Who cannot join
- Pre-existing other hearing-loss conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or complications of surgery.
- Presence of cochlear implants in the study ear.
- Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
- Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter hearing function.
- Prior participation in clinical study with an investigational drug within the past six months.
- Prior gene therapy treatments.
- Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-label, Multiple-cohort, Dose-finding, Investigator-initiated Trial to Evaluate the Safety, Tolerability, and Efficacy of HG205 RNA Base-Editing Therapy in Subjects With OTOF-p.Q829X Mutation-associated Hearing Loss
The purpose of the study is to determine whether HG205 as CRISPR/Cas13 RNA base-editing therapy is safe and effective for the treatment of hearing loss caused by p.Q829X mutation in OTOF gene.
- Conditions
- Congenital Hearing Loss
- Being tested
- HG205
- Keywords
- Congenital hearing loss, OTOF, Q829X, HG205, CRISPR
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HG205Genetic
The study will enroll up to 2 cohorts, evaluating a starting dose plus a higher or lower dose
| Study group | Role | What they receive |
|---|---|---|
| HG205 | Experimental | Method of Administration: Once Unilateral intracochlear injection/subject; The duration of the study for each subject includes a screening period, enrollment visit, treatment visit, a 26-week follow-up period, and a 5-year long-term safety follow-up after the injection Receives: Genetic: HG205 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of otological and systemic adverse events
Number of AE(Adverse events),SAE(Serious Adverse Events),DLT(Dose Limiting Toxicities)
Measured: 26 weeks
Secondary outcomes (2)
Change from baseline in ABR(Auditory Brainstem Response) intensity threshold (decibels normal hearing level [dB nHL])
ABR intensity threshold (decibels normal hearing level [dB nHL]) measurement
Measured: 26 weeks
Change from baseline in hearing performance by behavioral audiometry with pure-tone audiometry
Behavioral audiometry and pure-tone audiometry measurement
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Sep 2023
- Study start30 Mar 2023
- Primary completion8 Apr 2025
- Study completion8 Apr 2025
Why it stopped: Huidagene stopped this program as lack of patients in China
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Hearing LossStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- HuidaGene Therapeutics Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Eye & ENT Hospital of Fudan University
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, HuidaGene Therapeutics Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06025032
- Sponsor's protocol ID
- HG20501
- Status last verified
- Mar 2025
- First submitted
- 21 Aug 2023
- Last updated
- 10 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multiple-cohort, Dose-finding, Investigator-initiated Trial to Evaluate the Safety, Tolerability, and Efficacy of HG205 RNA Base-Editing Therapy in Subjects With OTOF-p.Q829X Mutation-associated Hearing Loss
- ClinicalTrials.gov ID
- NCT06025032
- Acronym / sponsor ID
- HG20501
- Conditions
- Congenital Hearing Loss
- Keywords
- Congenital hearing loss, OTOF, Q829X, HG205, CRISPR
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Huidagene stopped this program as lack of patients in China
- Enrolment
- 0 participants (actual)
- Lead sponsor
- HuidaGene Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Eye & ENT Hospital of Fudan University
- Study start
- 30 Mar 2023 (actual)
- Primary completion
- 8 Apr 2025 (actual)
- Study completion
- 8 Apr 2025 (actual)
- First posted
- 6 Sep 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.
The purpose of the study is to determine whether HG205 as CRISPR/Cas13 RNA base-editing therapy is safe and effective for the treatment of hearing loss caused by p.Q829X mutation in OTOF gene.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HG205 | Experimental | Genetic: HG205Method of Administration: Once Unilateral intracochlear injection/subject; The duration of the study for each subject includes a screening period, enrollment visit, treatment visit, a 26-week follow-up period, and a 5-year long-term safety follow-up after the injection |
Who may qualify
- Ages
- 1 year to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Male or females between 1 and 16 years of age at the time the subject/parent/legal guardian signs the informed consent form.
- Willing to adhere to the protocol as evidenced by written informed consent or parental permission and subject assent.
- Molecular diagnosis of biallelic mutations in the OTOF gene with at least one mutation being p.Q829X through collected blood samples at screening;
- Based on auditory brainstem response (ABR), clinically diagnosed sensorineural hearing loss (SNHL) with the following hearing thresholds: severe (65 dB ≤ hearing threshold < 80 dB) or profound (80 dB ≤ hearing threshold < 95 dB) or complete (hearing threshold ≥ 95 dB) hearing loss in both ears.
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
Key exclusion criteria (7 listed)
- Pre-existing other hearing-loss conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or complications of surgery.
- Presence of cochlear implants in the study ear.
- Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
- Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter hearing function.
- Prior participation in clinical study with an investigational drug within the past six months.
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 |
|---|---|---|
| HG205The study will enroll up to 2 cohorts, evaluating a starting dose plus a higher or lower dose | Genetic | HG205 |
Primary outcome
Incidence of otological and systemic adverse events
Time frame: 26 weeks
Number of AE(Adverse events),SAE(Serious Adverse Events),DLT(Dose Limiting Toxicities)
2 secondary outcomes
Change from baseline in ABR(Auditory Brainstem Response) intensity threshold (decibels normal hearing level [dB nHL])
Time frame: 26 weeks
Change from baseline in hearing performance by behavioral audiometry with pure-tone audiometry
Time frame: 26 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2025.
- Individual participant dataSharing planned: No
Related trials for Congenital Hearing Loss
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Recruiting
Pitch Perception and Memory: Deficits and Training
Cochlear Implants
RecruitingPhase 1/2
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Primary Photoreceptor Disease, Retinitis Pigmentosa (RP)
RecruitingPhase 3
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD)
RecruitingPhase 3
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
Retinitis Pigmentosa (RP), Usher Syndrome
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.
Study Director study director
HuidaGene Therapeutics Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing HG205 in people with Congenital Hearing Loss, run by HuidaGene Therapeutics Co., Ltd. at 1 site 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 5 inclusion and 7 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: withdrawn, as of the sponsor's last update on 10 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of otological and systemic adverse events (time frame: 26 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Apr 2025.