A Phase 1/2 Trial of AAVAnc80-antiVEGF Gene Therapy in Individuals With Unilateral Vestibular Schwannoma
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- Akouos, Inc.
- 入组人数
- 27
- 试验地点
- 8
- 主要终点
- Frequency of Adverse Events (AEs)
研究概览
简要总结
This trial will evaluate the safety and tolerability of a single unilateral administration of one of three dose levels of AAVAnc80-antiVEGF and will evaluate the Akouos delivery device to safely achieve the intended product performance.
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
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Criteria for Inclusion:
- •Presence of unilateral, progressive vestibular schwannoma.
- •Vestibular schwannoma larger than 2 mm.
- •Profound hearing loss, defined by pure-tone audiometry thresholds or word recognition score, in the affected ear.
- •Able and willing to comply with all trial requirements, including willingness to participate in a separate long term follow-up study after completion of this trial.
- •Criteria for
排除标准
- •Prior diagnosis of NF2 and/or bilateral vestibular schwannoma.
- •Prior surgery or radiation therapy for vestibular schwannoma.
- •Clinical history consistent with endolymphatic hydrops (documented fluctuating sensorineural hearing loss and/or episodic vertigo) in the affected ear.
- •Profound hearing loss, defined by pure-tone audiometry thresholds or word recognition score, in the unaffected ear.
- •Prior participation in a clinical trial with an investigational drug within six months prior to administration (Day 0), or any prior participation in a gene therapy clinical trial.
研究组 & 干预措施
Cohort 1
Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 1) in the study ear using a sterile, one-time use investigational medical device.
干预措施: AAVAnc80-antiVEGF via Akouos Delivery Device (Combination Product)
Cohort 2
Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 2) in the study ear using a sterile, one-time use investigational medical device.
干预措施: AAVAnc80-antiVEGF via Akouos Delivery Device (Combination Product)
Cohort 3
Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 3) in the study ear using a sterile, one-time use investigational medical device
干预措施: AAVAnc80-antiVEGF via Akouos Delivery Device (Combination Product)
结局指标
主要结局
Frequency of Adverse Events (AEs)
时间窗: through trial completion, approximately one year
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)
次要结局
- Performance of Akouos Delivery Device(through trial completion, approximately one year)
- Tumor Volume(through trial completion, approximately one year)