A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- Akouos, Inc.
- 入组人数
- 22
- 试验地点
- 17
- 主要终点
- Frequency of Adverse Events (AEs)
研究概览
简要总结
Part A of this trial will evaluate the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos Delivery Device, together with the Precision Delivery Mechanism, to safely achieve the intended product performance.
详细描述
The following two dose levels will be tested:
- Up to 4.1E11 total vg/cochlea
- Up to 8.1E11 total vg/cochlea
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Criteria for Inclusion:
- •Participants may be of any age, based on Cohort Criteria
- •At least two mutations in the otoferlin gene
- •Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
- •Preserved distortion product otoacoustic emissions (DPOAEs)
- •Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial
- •Criteria for
排除标准
- •Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy
- •Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
- •Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial
研究组 & 干预措施
Cohort 1a and Cohort 1b
Cohort 1a: Two participants aged 7 to 17 years old (inclusive) to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device
Cohort 1b: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age
干预措施: AAVAnc80-hOTOF via Akouos Delivery Device (Combination Product)
Cohort 2
Cohort 2: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age
干预措施: AAVAnc80-hOTOF via Akouos Delivery Device (Combination Product)
Bilateral Expansion
Bilateral Expansion: up to eight eligible participants of any age, and no cochlear implants, may be eligible for enrollment into the Bilateral Expansion Cohort (simultaneous bilateral administration of AAVAnc80-hOTOF).
干预措施: AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism (Combination Product)
结局指标
主要结局
Frequency of Adverse Events (AEs)
时间窗: through trial completion, approximately 1 year
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system)
次要结局
- Auditory Brainstem Response (ABR) Threshold(through trial completion, approximately 1 year)
- Performance of the Akouos Delivery Device and Precision Delivery Mechanism(through trial completion, approximately 1 year)