Phase III Multicenter Study of Magnetic Mini-Mover for Pectus Excavatum G090006 1

摘要 漏斗胸是最常见的先天性胸部畸形。这一缺陷的特征是 由畸形的肋软骨导致胸骨凹陷。手术矫正需要较大的 手术，止痛住院，以及60,000至80,000美元的费用。磁性迷你- Mover Procedure(3MP)是一种门诊手术，它使用磁性耦合植入物来 将胸骨向前拉，逐渐重塑变形的肋软骨。在一个正在进行的 FDA赞助的I-II阶段研究(R01 FD003341)，我们正在研究 10例8~14岁胸腔积液严重程度指数为3.50(正常= 2.56)。中期分析表明，3MP是一种安全、门诊、微创和 经济有效的漏斗胸治疗，支具佩戴依从性为10-21 4小时/天(平均16.8小时/天)，胸廓畸形改善较快。 青春期患者。 此外，这项最初的I-II期试验在钛包裹的 磁性植入物(Magnimplant)及外磁体支架的主要改进 (Magnatract)。第二代Magnatract允许患者调整施加的力， 通过留置压力传感器测量压力，每隔10分钟记录力和温度 分钟，并通过互联网安全地下载数据。与FDA进行咨询 建议进一步的关于安全性和可能疗效的数据，并证明 可以在其他中心安全使用的程序将允许申请人道主义 设备豁免(HDE)。我们现在将在第三阶段研究中延长初步试验的结果 这结合了第二代磁力迷你第一次试验的改进- 移动者程序(G090006；2009年1月2日)。我们建议再招收15名8岁的患者- 12，三个中心的胸腔严重程度指数为3.50。

ABSTRACT Pectus excavatum is the most common congenital chest deformity. This defect is characterized by malformed costal cartilages causing a depressed sternum. Surgical correction requires major surgery, hospitalization for pain control, and costs of $60,000 to $80,000. The Magnetic Mini- Mover Procedure (3MP) is an outpatient procedure that uses a magnetically coupled implant to pull the sternum forward and gradually remodel the deformed costal cartilage. In an ongoing FDA-sponsored phase I-II study (R01 FD003341), we are studying the safety and probable efficacy of the 3MP in 10 patients ages 8-14 years with Pectus Severity Index > 3.50 (normal = 2.56). Interim analysis demonstrates that the 3MP is a safe, outpatient, minimally-invasive, and cost-effective treatment for pectus excavatum, that brace-wear compliance was 10-21 hours/day (mean 16.8 h/day), and that the pectus deformity improved more rapidly in pre- pubertal patients. In addition, this initial phase I-II trial has led to minor improvements in the titanium-encased magnetic implant (Magnimplant) and major improvements in the external magnet brace (Magnatract). The second generation Magnatract allows the patient to adjust the force applied, measures the force by an indwelling pressure sensor, logs force and temperature every 10 minutes, and downloads the data securely over the Internet. Consultation with the FDA suggested that further data on safety and probable efficacy and demonstration that the procedure can be safely used at other centers would allow application for a Humanitarian Device Exemption (HDE). We will now extend the findings of the initial trial in a phase III study that incorporates the improvements from the first trial in the second generation Magnetic Mini- Mover Procedure (G090006; 02 Jan 2009). We propose enrolling 15 additional patients ages 8- 12 with Pectus Severity Index > 3.50 at three centers.