NCT06025032撤回早期 1 期
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 个研究点 分布在 1 个国家目标入组 6 人开始时间: 2023年3月30日最近更新:
适应症
试验速览
- 阶段
- 早期 1 期
- 状态
- 撤回
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Incidence of otological and systemic adverse events
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
结局指标
主要结局
Incidence of otological and systemic adverse events
时间窗: 26 weeks
Number of AE(Adverse events),SAE(Serious Adverse Events),DLT(Dose Limiting Toxicities)
次要结局
- 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)
研究者
研究点 (1)
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