NCT05572073招募中不适用
A Natural History Study in Individuals With Otoferlin Gene-mediated Hearing Loss
适应症
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Akouos, Inc.
- 入组人数
- 150
- 试验地点
- 18
- 主要终点
- ABR
研究概览
简要总结
This is a retrospective and prospective longitudinal study in participants with Otoferlin Gene-Mediated Hearing Loss.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Other
入排标准
- 年龄范围
- — 至 44 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical presentation of bilateral sensorineural hearing loss (SNHL), including auditory neuropathy (AN) / auditory neuropathy spectrum disorder (ANSD) phenotype or medical history of AN / ANSD phenotype earlier in life
- •Mutation(s) in the otoferlin gene
- •Able and willing to comply with all study requirements, as evidenced by successful completion of the informed consent (and assent, if applicable) process
- •Additional Criteria for Inclusion in the Prospective Phase:
- •Presence of OAE / CM and absent / abnormal ABRs in at least one ear (that does not have a cochlear implant) within 12 months prior to or at the Month 0 visit
排除标准
- •Unwillingness or inability of the potential participant and/or legally authorized representative to comply with all protocol requirements
- •Presence of cochlear nerve deficiency and/or cochlear nerve dysplasia
- •Additional Criteria for Exclusion from the Prospective Phase:
- •Presence of bilateral cochlear implants at the time of record review or planned within the next 6 months
- •Presence of middle ear or auditory brainstem implant(s) at the time of record review or planned within the next 6 months
- •Any condition that would not allow the potential participant to complete follow-up assessments during the course of the study and/or, in the opinion of the Investigator, makes the potential participant unsuitable for the study
- •Note: Potential participants will not be excluded based on their sex, gender, race, or ethnicity
研究组 & 干预措施
Retrospective
干预措施: Natural History Study (Other)
Prospective
干预措施: Natural History Study (Other)
结局指标
主要结局
ABR
时间窗: First audiologic data in participant medical record through five-year prospective follow up
Auditory Brainstem Response
次要结局
- OAE(First audiologic data in participant medical record through the five-year prospective follow up)
研究者
研究点 (18)
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