Phase I Pediatric Auditory Brainstem Implant Clinical Trial

描述(由申请人提供)：多通道听性脑干植入物(ABI)现已在全球1000多名不适合植入人工耳蜗(CI)的聋人中使用，并取得了一定的疗效。多年来，ABI主要用于2型神经纤维瘤病(NF2)致聋的成年人。在过去的十年里，ABI手术已经在美国以外的非NF2儿童身上安全地进行了手术，结果显示出了巨大的希望。这一第一阶段临床试验拨款的总体目标是评估10名儿童受试者的安全性，这些受试者由于耳蜗神经缺陷、耳蜗性发育不全或耳蜗骨化而不是传统人工耳蜗术的候选者。这些类型的病理阻碍了听觉信息在耳蜗和脑干之间的正常传输。豪斯研究所提议在美国进行第一个儿科、非NF2 ABI临床试验。主要目的是描述装置植入手术的安全性，以及在10名儿童患者中使用ABI 12个月，这些患者患有严重的双侧听力损失，不适合其他治疗。如果手术安全性分析显示与手术相关的严重不良事件的数量没有超过接受开颅手术的非NF2成人(无论有无ABI)的常见数量，并且在12个月的随访中这10名受试者观察到的与设备相关的意外严重不良事件不超过4例，那么将建议进行一项有更多受试者的II期研究。二级目标将确定该设备是否在交流技能发展的关键时期为患者提供声音(阈值为50dBHL)。效能在这里被定义为在已知与言语相关的水平和频率范围内获得声音的机会。这项第一阶段临床试验的成功结果将为计划第二阶段研究提供必要的信息。

DESCRIPTION (provided by applicant): The multichannel auditory brainstem implant (ABI) has now been used with some benefit in more than 1000 deaf patients worldwide who were not candidates for a cochlear implant (CI). For many years, the ABI was used primarily in adults deafened by Neurofibromatosis-Type 2 (NF2). During the past decade, ABI surgery has been safely performed on non-NF2 children outside of the United States, and results have shown great promise. The overall objective of this Phase I Clinical Trial grant is to evaluate safety in ten pediatric subjects who are not candidates for traditional cochlear implants due to cochlear nerve deficiency, cochlear aplasia, or cochlear ossification. These types of pathologies prevent normal transmission of auditory information between the cochlea and the brainstem. The House Research Institute proposes the first pediatric, non-NF2 ABI clinical trial in the United States. The Primary Aim is to describe the safety aspects of device implant surgery and 12- month use of an ABI in 10 pediatric patients with profound bilateral hearing loss not amenable to other treatments. If the surgical safety analysis shows no greater number of serious surgery-related adverse events than is commonly observed in non-NF2 adults undergoing craniotomy with or without an ABI and no more than four unexpected serious device-related adverse events are observed per these 10 subjects during 12 months of follow-up; then a Phase II study with a larger number of subjects will be proposed. A Secondary Aim will determine whether or not the device provides patients with access to sound (thresholds of 50 dB HL) during a critical period for communication skills development. Efficacy is here defined as access to sound at a level and within the frequency range, known to be associated with speech. Successful outcomes from this Phase I Clinical Trial will provide the necessary information for planning a Phase II investigation.