Phase I Pediatric Auditory Brainstem Implant Clinical Trial

描述（由申请人提供）：多通道听觉脑干植入体（ABI）目前已在全球1000多名不适合人工耳蜗植入体（CI）的耳聋患者中使用，并取得了一定的获益。多年来，ABI主要用于2型神经纤维瘤病（NF 2）的成年人。在过去的十年中，ABI手术已经安全地在美国以外的非NF 2儿童中进行，结果显示出很大的希望。该I期临床试验资助的总体目标是评价10名因耳蜗神经缺陷、耳蜗发育不全或耳蜗骨化而不适合传统人工耳蜗植入的儿童受试者的安全性。这些类型的病理阻止听觉信息在耳蜗和脑干之间的正常传输。众议院研究所提出了美国第一个儿科非NF 2 ABI临床试验。主要目的是描述10例无法接受其他治疗的重度双侧听力损失儿童患者的器械植入手术和12个月ABI使用的安全性。如果手术安全性分析显示严重手术相关不良事件的数量不超过在接受开颅术（伴或不伴ABI）的非NF 2成人中常见的数量，并且在12个月随访期间，每10例受试者观察到的非预期严重器械相关不良事件不超过4起;则将提议开展一项受试者数量更多的II期研究。次要目的将确定器械是否在沟通技能发展的关键时期为患者提供声音（阈值50 dB HL）。功效在这里被定义为在已知与语音相关联的水平和频率范围内获得声音。本I期临床试验的成功结果将为II期研究的计划提供必要的信息。

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.