NCT06025032WithdrawnEarly Phase 1
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
HuidaGene Therapeutics Co., Ltd.1 site in 1 country6 target enrollmentStarted: March 30, 2023Last updated:
Conditions
Trial Snapshot
- Phase
- Early Phase 1
- Status
- Withdrawn
- Sponsor
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Incidence of otological and systemic adverse events
Study Overview
Brief 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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 1 Year to 16 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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.
Exclusion Criteria
- •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.
Outcomes
Primary Outcomes
Incidence of otological and systemic adverse events
Time Frame: 26 weeks
Number of AE(Adverse events),SAE(Serious Adverse Events),DLT(Dose Limiting Toxicities)
Secondary Outcomes
- Change from baseline in ABR(Auditory Brainstem Response) intensity threshold (decibels normal hearing level [dB nHL])(26 weeks)
- Change from baseline in hearing performance by behavioral audiometry with pure-tone audiometry(26 weeks)
Investigators
Study Sites (1)
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