A Phase I/II, Open-ended, Adaptative, Open Label Dose Escalation and Expansion Clinical Trial to Evaluate the Efficacy and Safety of Unilateral Intracochlear Injection of SENS-501 Using an Injection System in Children With Severe to Profound Hearing Loss Due to Otoferlin Gene Mutations
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Sensorion
- 入组人数
- 12
- 试验地点
- 3
- 主要终点
- Safety and tolerability of SENS-501
研究概览
简要总结
This study intends to assess safety, tolerability, and efficacy of SENS-501 in children between the ages of 6-31 months with pre-lingual hearing loss due to a mutation in the Otoferlin gene.
详细描述
It is a multicenter, adaptive open-label, non-randomized, dose-escalation, and expansion study to assess safety, tolerability, and efficacy following intracochlear administration of SENS-501.
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
- 6 Months 至 31 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children (male or female) ≥ 6 to ≤ 31 months at the time of inclusion
- •Severe to profound hearing loss assessed by auditory brainstem response (ABR)
- •Biallelic mutation in the Otoferlin gene
- •Presence of Otoacoustic emissions (OAEs)
- •Documented normal cochlea and internal auditory canals
- •Patients with intact vestibular function
排除标准
- •History of chronic, acute, or major disease, or unspecified reasons, that in the opinion of the Investigator, makes the participant unsuitable for participation in the study or constitutes an unacceptable risk.
- •Have been dosed in a previous gene therapy clinical trial
- •Patients with a prior or current cochlear implant
- •Any contraindication to the surgery determined by the surgeon or anesthesia determined by the anesthesiologist, or designee, or history of therapy known as ototoxic (e.g., cisplatin, high dose and long treatment with aminoglycosides, etc.) for an extended period (more than 2 weeks).
- •Participation in any other interventional clinical trial
- •Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful completion of the study or might interfere with the evaluation of study treatment
- •Anticipated noncompliance with the protocol requirements
研究组 & 干预措施
Dose escalation - Low dose group
Intracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system
干预措施: SENS-501 administration (Combination Product)
Dose escalation - High dose group
Intracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system
干预措施: SENS-501 administration (Combination Product)
Dose expansion group
Intracochlear administration of SENS-501 in one ear, with a dedicated administration system, at the dose recommended following the Dose escalation phase
干预措施: SENS-501 administration (Combination Product)
结局指标
主要结局
Safety and tolerability of SENS-501
时间窗: 5 years
Number of study-related adverse events (AEs) and serious adverse events (SAEs).
Efficacy of SENS-501 assessed by ABR
时间窗: 5 years
Improvement of hearing threshold measured by auditory brainstem response (ABR)
次要结局
- Safety and tolerability of SENS-501(5 years)
- Efficacy of SENS-501 assessed by ABR(5 years)
- Efficacy of SENS-501 assessed by PTA(5 years)
- Clinical performance of the administration system(1 day)
- Safety of the administration system(5 years)
- Usability of the administration system(1 day)