Myelomeningocele Repair Randomized Trial (U01-HD41665)

描述（由申请方提供）：脊髓脊膜膨出管理研究（MOMS）是一项在三个临床中心进行的200例患者的多中心非混合随机临床试验，这三个临床中心是加利福尼亚大学-旧金山弗朗西斯科分校、费城儿童医院和范德比尔特大学医学中心，以及作为数据和研究协调中心的乔治华盛顿大学生物统计中心。该试验的主要目的是确定在妊娠190至256周时对胎儿脊髓脊膜膨出进行宫内修复是否能改善结局，通过1）1岁时死亡或需要心室减压分流，以及2）30个月矫正年龄时的复合神经功能评分进行测量。 只有一小部分脊柱裂患儿出生时膀胱和括约肌的神经支配正常，泌尿系统后遗症是终身的。产前手术治疗脊髓脊膜膨出对随后泌尿系统功能的影响尚不清楚，但确定这一点至关重要。该提案是对MOMS试验的补充，旨在对试验中的婴儿进行更严格的泌尿科随访。预计120名婴儿将在研究期间的三个时间点接受肾超声检查、视频尿动力学和尿分析、培养和敏感性检查：分娩后新生儿出院前、12个月和30个月矫正年龄。该补充的目的是确定开放性脊髓脊膜膨出的宫内治疗是否影响尿路感染的结局，以及是否需要间歇性导尿。一个独立的儿科泌尿科医生委员会将审查医疗记录和成像研究，以确定婴儿是否符合清洁间歇导尿的客观标准。 乔治华盛顿大学生物统计中心将作为本补充的数据和研究协调中心。我们将在研究设计、修订方案和操作手册、准备病例报告表、安排宣传、更新和维护数据管理系统以及研究内部和外部网站方面进行合作。我们将继续作为中心筛选和转诊中心，并将协调集中结局审查以及在三家中心进行的长期随访访视。

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.