2023-504466-28-00招募中2 期
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
- 2 期
- 状态
- 招募中
- 发起方
- Sensorion
- 入组人数
- 6
- 试验地点
- 3
- 主要终点
- Part 1: Number of study-related adverse events and serious adverse events (through trial completion)
研究概览
简要总结
Part 1: To assess safety, tolerability of SENS-501 following unilateral injection into the cochlea. Part 2: To assess the efficacy following unilateral injection SENS-501 into the cochlea.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Part 2
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Children (male or female) ≥ 6 to ≤ 31 months at the time of inclusion.
- •Severe to profound hearing loss assessed by ABR
- •Biallelic mutation in the Otoferlin gene
- •Presence of 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 (in accordance with the definition in Art. 2.2(1) of REGULATION (EU) No 536/2014).
- •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
结局指标
主要结局
Part 1: Number of study-related adverse events and serious adverse events (through trial completion)
Part 1: Number of study-related adverse events and serious adverse events (through trial completion)
Part 2: Number of patients who demonstrate an improvement of hearing threshold measured by ABR (through trial completion)
Part 2: Number of patients who demonstrate an improvement of hearing threshold measured by ABR (through trial completion)
次要结局
- Part 1: Number of patients who demonstrate an improvement of hearing threshold measured by ABR (through trial completion)
- Part 2 : Number of study-related adverse events and serious adverse events (through trial completion)
- Part 1 and 2: Number of patients who demonstrate an improvement of hearing threshold measured by PTA (through trial completion)
- Part 1 and 2: Clinical performance and usability of SPHYNX Pump and Catheter (SPHYNX pump injection log, user feedback questionnaire, usability questionnaire - through trial completion)
研究者
Lionel HOVSEPIAN
Scientific
Sensorion
研究点 (3)
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