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。这种缺陷的特点是 肋骨软骨畸形导致胸骨凹陷手术矫正需要 手术，住院控制疼痛，以及60，000至80，000美元的费用。磁性迷你- Mover手术（3 MP）是一种门诊手术，使用磁耦合植入物， 将胸骨向前拉，逐渐重塑变形的肋软骨。以不间断 FDA申办的I-II期研究（R 01 FD 003341），我们正在研究 3 MP在10例年龄8-14岁的患者中的疗效，其中胸部严重指数> 3.50（正常= 3.50）。 2.56）。中期分析表明，3 MP是一种安全的，门诊，微创， 漏斗胸的经济有效的治疗方法，即支架佩戴依从性为10-21 小时/天（平均16.8小时/天），而胸部畸形改善更快， 青春期患者 此外，最初的I-II期试验使钛包裹的 磁性植入物（Magnimplant）和外部磁性支架的主要改进 （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力，每10分钟记录一次力和温度 分钟，并通过互联网安全地下载数据。与FDA协商 表明，关于安全性和可能疗效的进一步数据， 程序可以安全地在其他中心使用，允许申请人道主义援助。 器械豁免（HDE）。我们现在将在第三阶段研究中扩展初步试验的结果 它结合了第一次试验的改进，在第二代磁性迷你- 移动程序（G 090006; 2009年1月2日）。我们建议再招募15名8岁的患者- 12例患者在3个中心的胸大肌严重指数> 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.