Myelomeningocele Repair Randomized Trial (U01-HD41665)

描述（由申请人提供）：髓脊膜膨出的管理研究（mom）是一项多中心、200名患者的非混合随机临床试验，正在三个临床中心进行，分别是加州大学旧金山分校、费城儿童医院和范德比尔特大学医学中心，乔治华盛顿大学生物统计中心作为数据和研究协调中心。该试验的主要目的是确定在妊娠190 ~ 256周时宫内修复胎儿脊膜膨出是否能改善预后，通过以下指标来衡量：(1)死亡或1年的心室减压分流需求；(2)矫正年龄30个月时的复合神经功能评分。

DESCRIPTION (provided by applicant): The Management of Myelomeningocele Study (MOMS) is a multi-center unblended randomized clinical trial of 200 patients that is underway at three clinical centers, the University of California-San Francisco, The Children's Hospital of Philadelphia, and Vanderbilt University Medical Center, and the George Washington University Biostatistics Center as the Data and Study Coordinating Center. The primary objective of the trial is to determine whether intrauterine repair of fetal myelomeningocele at 190 to 256 weeks gestation improves outcome, as measured by 1) death or the need for ventricular decompressive shunting by one year of life, and 2) a composite neurologic function score at 30 months corrected age. Only a small percentage of children with spina bifida are born with a normal innervation of their bladder and sphincter muscles, with urologic sequelae that are life-long. The effect of prenatal surgery for myelomeningocele on subsequent urologic function is unknown, but vitally important to determine. This proposal is for a supplement to the MOMS trial to initiate a more rigorous urologic follow-up of the infants in the trial. It is anticipated that 120 infants will receive renal sonography, video urodynamics and urinalysis, culture and sensitivity at three time points during the study: before neonatal discharge following delivery, at 12 months, and 30 months corrected age. The aim of the supplement is to determine whether in utero treatment of open myelomeningocele affects the outcome with respect to urinary tract infection, and the need for intermittent catheterization. A committee of independent pediatric urologists will review medical records and imaging studies to determine whether infants meet objective criteria for clean intermittent catheterization. The George Washington University Biostatistics Center will serve as the Data and Study Coordinating Center for this supplement. We will collaborate on study design, revise the protocol and manual of operations, prepare case report forms, arrange publicity, update and maintain the data management system and study internal and external internet sites. We will continue to serve as the central screening and referral site, and we will coordinate the centralized outcome review, as well as the long term follow-up visits conducted at the three centers.