An Evaluation of the Long Term Safety and Efficacy of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- Akouos, Inc.
- 入组人数
- 30
- 试验地点
- 5
- 主要终点
- Long term safety of AAVAnc80-hOTOF, including the incidence and frequency of late-occurring adverse events (AEs)
研究概览
简要总结
This multi-center, observational, post-intervention long term follow-up (LTFU) study will monitor for safety and efficacy of AAVAnc80-hOTOF in individuals with sensorineural hearing loss due to otoferlin gene mutations who have previously received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.
详细描述
After completing one year of initial safety and efficacy assessments following an intracochlear administration of AAVAnc80-hOTOF in AK-OTOF-101, participants will continue to be observed for an additional approximately four years of safety and efficacy follow-up in this LTFU study, for a total of approximately five years of safety and efficacy observations after vector administration. For each participant, evaluations in the LTFU study will occur at approximately one and a half years and two years after vector administration, followed by annual visits through Year 5; the duration in this LTFU study is approximately four years, with the aim of understanding and mitigating any risks related to delayed adverse events, as well as characterizing the durability of effect of the AAVAnc80-hOTOF.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial
- •Legally authorized representative and/or participant willingness to comply with all study requirements, as evidenced by successful completion of the informed consent process or permission and participant assent process (where appropriate)
排除标准
- •Any condition that would not allow the potential participant to complete follow-up examinations during the course of the study and/or, in the opinion of the Investigator, makes the potential participant unsuitable for the study
研究组 & 干预措施
Study Participants
Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial
结局指标
主要结局
Long term safety of AAVAnc80-hOTOF, including the incidence and frequency of late-occurring adverse events (AEs)
时间窗: Through study completion, approximately four years.
The following adverse events will be collected: 1. The development of new or exacerbation of malignancies (oncologic), neurologic, rheumatologic or other autoimmune, hematologic disorders, or new infections (if the infection is potentially related to AAVAnc80-hOTOF 2. AEs that are related to AAVAnc80-hOTOF and/or the administration procedure 3. AEs of special interest 4. All serious adverse events regardless of severity or of relationship to participation in the AK-OTOF-101 Clinical Trial
次要结局
- Auditory Brainstem Response (ABR) Threshold(Through study completion, approximately four years.)