Final clinical studies for submission of a pre-market approval application to the FDA for a Bionic Pancreas that automates type 1 diabetes management

 描述(由申请人提供)：我们已经建立了我们的生物激素仿生胰腺(BP)，并在1型糖尿病(T1D)患者的住院和现实世界门诊研究中进行了广泛的测试。通过波士顿大学(BU)和马萨诸塞州总医院(MGH)长达八年的合作，我们已经完成了三项为期1天和2天的住院研究，测试笔记本电脑版本的BP，以及3项为期5天的门诊研究，测试患有T1D的成年人(在波士顿市中心)、患有T1D的青少年(在糖尿病营地)和青少年前期的T1D(在糖尿病营地)。在波士顿大学、麻省理工学院、马萨诸塞大学医学中心、斯坦福大学和北卡罗来纳大学教堂山分校的合作下，我们完成了一项为期11天的成人仿生胰腺多中心门诊研究，T1D在家里和工作场所测试iPhone版本的BP，不限制活动或锻炼。我们最近按照III类医疗器械标准构建了一款新的双激素BP设备平台，该平台集成了一款微精度双输液泵，该泵可(1)分别输送胰岛素和胰高血糖素，(2)充当FDA认可的连续血糖监测仪(CGM)的接收器，以及(3)包括我们经过临床测试的一套数学控制算法，它可以根据CGM血糖数据自动确定和控制胰岛素和胰高血糖素的剂量。这一新的设备平台提供了一个交钥匙解决方案，将标准化、简化和改进T1D治疗。在这里，我们建议进行仿生胰腺枢纽试验(BPPT)，它将招募480名患有T1D的儿童和成人(8岁及以上，HbA1c&lt；11%)，以测试和鉴定完全整合的BP版本，并向FDA提供上市前批准(PMA)申请所需的所有临床数据。我们建议通过以下特殊fic目标来实现这一目标：(1)评估BP在人为因素研究中的可用性，必要时基于研究结果重新fi设备和用户界面，并在小型门诊可行性研究(仿生胰腺桥研究)中鉴定其功能；以及(2)进行BPPT以测试fifiNal BP系统在控制血糖方面的安全性和有效性(具有HbA1c的共同初级结果和CGM血糖测量和lt的百分比；60 mg/dl)，以评估我们的技术相对于常规护理的行为和心理社会影响，并为该设备的新使用适应症提供关于长期给予fi的安全性的数据。

 DESCRIPTION (provided by applicant): We have built and extensively tested our bihormonal bionic pancreas (BP) in inpatient and real-world outpatient studies in subjects with type 1 diabetes (T1D). Through an eight-year collaboration between Boston University (BU) and the Massachusetts General Hospital (MGH), we have completed three 1- and 2-day inpatient studies testing a laptop version of the BP and three 5-day outpatient studies testing an iPhone version of the BP in adults with T1D (in downtown Boston), adolescents with T1D (in a diabetes camp), and pre-adolescents with T1D (in a diabetes camp). In collaboration among BU, MGH, the University of Massachusetts Medical Center, Stanford University, and the University of North Carolina, Chapel Hill, we completed an 11-day Bionic Pancreas Multi-Center outpatient study in adults with T1D testing the iPhone version of the BP at home and in the workplace without restrictions on activity or exercise. We have recently built a new bihormonal BP device platform in accordance with Class III medical device standards that integrates a microprecision dual-infusion pump that (1) separately delivers insulin and glucagon, (2) acts as a receiver for an FDA-cleared continuous glucose monitor (CGM), and (3) includes our clinically tested suite of mathematical control algorithms that autonomously determines and commands doses of insulin and glucagon based on CGM glucose data. This new device platform provides a turnkey solution that will standardize, simplify, and improve T1D therapy. Here we propose to conduct the Bionic Pancreas Pivotal Trial (BPPT), which will enroll 480 children and adults with T1D (ages 8 and older, HbA1c <11%) to test and qualify the fully integrated version of the BP and to provide all of the clinical data necessary for a pre-market approval (PMA) application to the FDA. We propose to achieve this objective with the following specific aims: (1) to evaluate the usability of the BP in a human factors study, to refine the device and user interface, if necessary, based on the study results, and to qualify its functionality in a small outpatient feasibility study (the Bionic Pancreas Bridging Study); and (2) to conduct the BPPT to test the safety and efficacy of the final BP system in controlling glycemia (with co-primary outcomes of HbA1c and percentage of CGM glucose measurements < 60 mg/dl), to evaluate the behavioral and psychosocial impact of our technology relative to usual care, and to provide data on the safety of chronic glucagon administration sufficient for a new use indication for this device.