Myelomeningocele Repair Randomized Trial (U01-HD41665)

脊髓脊膜膨出修复随机试验 (U01-HD41665)

• 批准号: 6822220
• 负责人: ELIZABETH ANN THOM
• 金额: $ 10.26万
• 依托单位: GEORGE WASHINGTON UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-04-11 至 2007-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6822220
• 关键词: Internet; biomedical facility; clinical research; clinical trials; cooperative study; data management; data quality /integrity; embryo /fetus surgery; experimental designs; human pregnant subject; human therapy evaluation; infant mortality; information dissemination; longitudinal human study; medical outreach /case finding; meningomyelocele; neurosurgery; patient oriented research; spina bifida; statistics /biometry

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.

描述(由申请人提供)：脊髓脊膜膨出管理研究(MOMS)是一项多中心非混合随机临床试验，有200名患者参加，目前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，以及乔治华盛顿大学生物统计中心作为数据和研究协调中心。该试验的主要目标是确定怀孕190到256周的胎儿脊髓脊膜膨出的宫内修复是否改善了结局，衡量标准是1)死亡或需要一年的脑室减压术，以及2)校正年龄30个月时的综合神经功能评分。 在患有脊柱裂的儿童中，只有一小部分出生时膀胱和括约肌的神经正常，并有终生的泌尿系统后遗症。脊髓脊膜膨出的产前手术对随后的泌尿系功能的影响尚不清楚，但至关重要的是要确定。这项建议是对MOMS试验的补充，以启动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脏超声、视频尿流动力学和尿液分析、培养和敏感性检查：新生儿分娩后出院前、12个月和30个月校正年龄。补充的目的是确定开放性脊髓膜脑膨出的宫内治疗是否会影响尿路感染的预后，以及是否需要间歇性导尿。一个由独立的儿科泌尿科医生组成的委员会将审查医疗记录和影像研究，以确定婴儿是否符合清洁间歇导尿术的客观标准。 乔治华盛顿大学生物统计中心将作为本增刊的数据和研究协调中心。我们将合作进行研究设计，修改操作规程和手册，准备病例报告表格，安排宣传，更新和维护数据管理系统，并研究内部和外部网站。我们将继续作为中央筛查和转介网站，并将协调集中结果审查，以及在三个中心进行的长期跟踪访问。